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href="#In_vivo_versus_ex_vivo_therapies"> <div class="vector-toc-text"> 2.4 In vivo versus ex vivo therapies </div> </a> <ul id="toc-In_vivo_versus_ex_vivo_therapies-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Gene_editing" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Gene_editing"> <div class="vector-toc-text"> 2.5 Gene editing </div> </a> <ul id="toc-Gene_editing-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-Vectors" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Vectors"> <div class="vector-toc-text"> 3 Vectors </div> </a> <button aria-controls="toc-Vectors-sublist" class="cdx-button cdx-button--weight-quiet cdx-button--icon-only vector-toc-toggle"> Toggle Vectors subsection </button> <ul id="toc-Vectors-sublist" class="vector-toc-list"> <li id="toc-Viruses" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Viruses"> <div class="vector-toc-text"> 3.1 Viruses </div> </a> <ul id="toc-Viruses-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Non-viral" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Non-viral"> <div class="vector-toc-text"> 3.2 Non-viral </div> </a> <ul id="toc-Non-viral-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-Treatment" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Treatment"> <div class="vector-toc-text"> 4 Treatment </div> </a> <button aria-controls="toc-Treatment-sublist" class="cdx-button cdx-button--weight-quiet cdx-button--icon-only vector-toc-toggle"> Toggle Treatment subsection </button> <ul id="toc-Treatment-sublist" class="vector-toc-list"> <li id="toc-Cancer" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Cancer"> <div class="vector-toc-text"> 4.1 Cancer </div> </a> <ul id="toc-Cancer-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Genetic_diseases" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Genetic_diseases"> <div class="vector-toc-text"> 4.2 Genetic diseases </div> </a> <ul id="toc-Genetic_diseases-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Infectious_diseases" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Infectious_diseases"> <div class="vector-toc-text"> 4.3 Infectious diseases </div> </a> <ul id="toc-Infectious_diseases-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-List_of_gene_therapies_for_treatment_of_disease" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#List_of_gene_therapies_for_treatment_of_disease"> <div class="vector-toc-text"> 4.4 List of gene therapies for treatment of disease </div> </a> <ul id="toc-List_of_gene_therapies_for_treatment_of_disease-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-Adverse_effects,_contraindications_and_hurdles_for_use" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Adverse_effects,_contraindications_and_hurdles_for_use"> <div class="vector-toc-text"> 5 Adverse effects, contraindications and hurdles for use </div> </a> <button aria-controls="toc-Adverse_effects,_contraindications_and_hurdles_for_use-sublist" class="cdx-button cdx-button--weight-quiet cdx-button--icon-only vector-toc-toggle"> Toggle Adverse effects, contraindications and hurdles for use subsection </button> <ul id="toc-Adverse_effects,_contraindications_and_hurdles_for_use-sublist" class="vector-toc-list"> <li id="toc-Deaths" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#Deaths"> <div class="vector-toc-text"> 5.1 Deaths </div> </a> <ul id="toc-Deaths-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-Regulations" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Regulations"> <div class="vector-toc-text"> 6 Regulations </div> </a> <button aria-controls="toc-Regulations-sublist" class="cdx-button cdx-button--weight-quiet cdx-button--icon-only vector-toc-toggle"> Toggle Regulations subsection </button> <ul id="toc-Regulations-sublist" class="vector-toc-list"> <li id="toc-United_States" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#United_States"> <div class="vector-toc-text"> 6.1 United States </div> </a> <ul id="toc-United_States-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-Gene_doping" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Gene_doping"> <div class="vector-toc-text"> 7 Gene doping </div> </a> <ul id="toc-Gene_doping-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Genetic_enhancement" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Genetic_enhancement"> <div class="vector-toc-text"> 8 Genetic enhancement </div> </a> <ul id="toc-Genetic_enhancement-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-History" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#History"> <div class="vector-toc-text"> 9 History </div> </a> <button aria-controls="toc-History-sublist" class="cdx-button cdx-button--weight-quiet cdx-button--icon-only vector-toc-toggle"> Toggle History subsection </button> <ul id="toc-History-sublist" class="vector-toc-list"> <li id="toc-1970s_and_earlier" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#1970s_and_earlier"> <div class="vector-toc-text"> 9.1 1970s and earlier </div> </a> <ul id="toc-1970s_and_earlier-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-1980s" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#1980s"> <div class="vector-toc-text"> 9.2 1980s </div> </a> <ul id="toc-1980s-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-1990s" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#1990s"> <div class="vector-toc-text"> 9.3 1990s </div> </a> <ul id="toc-1990s-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2000s" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#2000s"> <div class="vector-toc-text"> 9.4 2000s </div> </a> <ul id="toc-2000s-sublist" class="vector-toc-list"> <li id="toc-2002" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2002"> <div class="vector-toc-text"> 9.4.1 2002 </div> </a> <ul id="toc-2002-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2003" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2003"> <div class="vector-toc-text"> 9.4.2 2003 </div> </a> <ul id="toc-2003-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2006" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2006"> <div class="vector-toc-text"> 9.4.3 2006 </div> </a> <ul id="toc-2006-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2007" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2007"> <div class="vector-toc-text"> 9.4.4 2007 </div> </a> <ul id="toc-2007-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2008" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2008"> <div class="vector-toc-text"> 9.4.5 2008 </div> </a> <ul id="toc-2008-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2009" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2009"> <div class="vector-toc-text"> 9.4.6 2009 </div> </a> <ul id="toc-2009-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-2010s" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#2010s"> <div class="vector-toc-text"> 9.5 2010s </div> </a> <ul id="toc-2010s-sublist" class="vector-toc-list"> <li id="toc-2010" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2010"> <div class="vector-toc-text"> 9.5.1 2010 </div> </a> <ul id="toc-2010-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2011" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2011"> <div class="vector-toc-text"> 9.5.2 2011 </div> </a> <ul id="toc-2011-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2012" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2012"> <div class="vector-toc-text"> 9.5.3 2012 </div> </a> <ul id="toc-2012-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2013" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2013"> <div class="vector-toc-text"> 9.5.4 2013 </div> </a> <ul id="toc-2013-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2014" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2014"> <div class="vector-toc-text"> 9.5.5 2014 </div> </a> <ul id="toc-2014-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2015" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2015"> <div class="vector-toc-text"> 9.5.6 2015 </div> </a> <ul id="toc-2015-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2016" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2016"> <div class="vector-toc-text"> 9.5.7 2016 </div> </a> <ul id="toc-2016-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2017" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2017"> <div class="vector-toc-text"> 9.5.8 2017 </div> </a> <ul id="toc-2017-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2019" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2019"> <div class="vector-toc-text"> 9.5.9 2019 </div> </a> <ul id="toc-2019-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> <li id="toc-2020s" class="vector-toc-list-item vector-toc-level-2"> <a class="vector-toc-link" href="#2020s"> <div class="vector-toc-text"> 9.6 2020s </div> </a> <ul id="toc-2020s-sublist" class="vector-toc-list"> <li id="toc-2020" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2020"> <div class="vector-toc-text"> 9.6.1 2020 </div> </a> <ul id="toc-2020-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2021" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2021"> <div class="vector-toc-text"> 9.6.2 2021 </div> </a> <ul id="toc-2021-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2022" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2022"> <div class="vector-toc-text"> 9.6.3 2022 </div> </a> <ul id="toc-2022-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-2023" class="vector-toc-list-item vector-toc-level-3"> <a class="vector-toc-link" href="#2023"> <div class="vector-toc-text"> 9.6.4 2023 </div> </a> <ul id="toc-2023-sublist" class="vector-toc-list"> </ul> </li> </ul> </li> </ul> </li> <li id="toc-List_of_gene_therapies" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#List_of_gene_therapies"> <div class="vector-toc-text"> 10 List of gene therapies </div> </a> <ul id="toc-List_of_gene_therapies-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-References" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#References"> <div class="vector-toc-text"> 11 References </div> </a> <ul id="toc-References-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-Further_reading" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#Further_reading"> <div class="vector-toc-text"> 12 Further reading </div> </a> <ul id="toc-Further_reading-sublist" class="vector-toc-list"> </ul> </li> <li id="toc-External_links" class="vector-toc-list-item vector-toc-level-1"> <a class="vector-toc-link" href="#External_links"> <div class="vector-toc-text"> 13 External links </div> </a> <ul id="toc-External_links-sublist" class="vector-toc-list"> </ul> </li> </ul> </div> </div> </nav> </div> </div> <div class="mw-content-container"> <main id="content" class="mw-body"> <header class="mw-body-header vector-page-titlebar"> <nav aria-label="Contents" class="vector-toc-landmark"> <div id="vector-page-titlebar-toc" class="vector-dropdown vector-page-titlebar-toc vector-button-flush-left" > <input type="checkbox" id="vector-page-titlebar-toc-checkbox" role="button" aria-haspopup="true" data-event-name="ui.dropdown-vector-page-titlebar-toc" class="vector-dropdown-checkbox " aria-label="Toggle the table of contents" > <label id="vector-page-titlebar-toc-label" for="vector-page-titlebar-toc-checkbox" class="vector-dropdown-label cdx-button cdx-button--fake-button cdx-button--fake-button--enabled cdx-button--weight-quiet cdx-button--icon-only " aria-hidden="true" > Toggle the table of contents </label> <div class="vector-dropdown-content"> <div id="vector-page-titlebar-toc-unpinned-container" class="vector-unpinned-container"> </div> </div> </div> </nav> <h1 id="firstHeading" class="firstHeading mw-first-heading">Gene therapy</h1> <div id="p-lang-btn" class="vector-dropdown mw-portlet mw-portlet-lang" > <input type="checkbox" id="p-lang-btn-checkbox" role="button" aria-haspopup="true" data-event-name="ui.dropdown-p-lang-btn" class="vector-dropdown-checkbox mw-interlanguage-selector" aria-label="Go to an article in another language. Available in 51 languages" > <label id="p-lang-btn-label" for="p-lang-btn-checkbox" class="vector-dropdown-label cdx-button cdx-button--fake-button cdx-button--fake-button--enabled cdx-button--weight-quiet cdx-button--action-progressive mw-portlet-lang-heading-51" aria-hidden="true" > 51 languages </label> <div class="vector-dropdown-content"> <div class="vector-menu-content"> <ul class="vector-menu-content-list"> <li class="interlanguage-link interwiki-ar mw-list-item"><a href="https://ar.wikipedia.org/wiki/%D8%B9%D9%84%D8%A7%D8%AC_%D8%AC%D9%8A%D9%86%D9%8A" title="علاج جيني – Arabic" lang="ar" hreflang="ar" data-title="علاج جيني" data-language-autonym="العربية" data-language-local-name="Arabic" class="interlanguage-link-target">العربية</a></li><li class="interlanguage-link interwiki-bs mw-list-item"><a href="https://bs.wikipedia.org/wiki/Genska_terapija" title="Genska terapija – Bosnian" lang="bs" hreflang="bs" data-title="Genska terapija" data-language-autonym="Bosanski" data-language-local-name="Bosnian" class="interlanguage-link-target">Bosanski</a></li><li class="interlanguage-link interwiki-ca mw-list-item"><a href="https://ca.wikipedia.org/wiki/Ter%C3%A0pia_g%C3%A8nica" title="Teràpia gènica – Catalan" lang="ca" hreflang="ca" data-title="Teràpia gènica" data-language-autonym="Català" data-language-local-name="Catalan" class="interlanguage-link-target">Català</a></li><li class="interlanguage-link interwiki-cs mw-list-item"><a href="https://cs.wikipedia.org/wiki/Genov%C3%A1_terapie" title="Genová terapie – Czech" lang="cs" hreflang="cs" data-title="Genová terapie" data-language-autonym="Čeština" data-language-local-name="Czech" class="interlanguage-link-target">Čeština</a></li><li class="interlanguage-link interwiki-da mw-list-item"><a href="https://da.wikipedia.org/wiki/Genterapi" title="Genterapi – Danish" lang="da" hreflang="da" data-title="Genterapi" data-language-autonym="Dansk" data-language-local-name="Danish" class="interlanguage-link-target">Dansk</a></li><li class="interlanguage-link interwiki-de mw-list-item"><a href="https://de.wikipedia.org/wiki/Gentherapie" title="Gentherapie – German" lang="de" hreflang="de" data-title="Gentherapie" data-language-autonym="Deutsch" data-language-local-name="German" class="interlanguage-link-target">Deutsch</a></li><li class="interlanguage-link interwiki-et mw-list-item"><a href="https://et.wikipedia.org/wiki/Geeniteraapia" title="Geeniteraapia – Estonian" lang="et" hreflang="et" data-title="Geeniteraapia" data-language-autonym="Eesti" data-language-local-name="Estonian" class="interlanguage-link-target">Eesti</a></li><li class="interlanguage-link interwiki-el mw-list-item"><a href="https://el.wikipedia.org/wiki/%CE%93%CE%BF%CE%BD%CE%B9%CE%B4%CE%B9%CE%B1%CE%BA%CE%AE_%CE%B8%CE%B5%CF%81%CE%B1%CF%80%CE%B5%CE%AF%CE%B1" title="Γονιδιακή θεραπεία – Greek" lang="el" hreflang="el" data-title="Γονιδιακή θεραπεία" data-language-autonym="Ελληνικά" data-language-local-name="Greek" class="interlanguage-link-target">Ελληνικά</a></li><li class="interlanguage-link interwiki-es mw-list-item"><a href="https://es.wikipedia.org/wiki/Terapia_g%C3%A9nica" title="Terapia génica – Spanish" lang="es" hreflang="es" data-title="Terapia génica" data-language-autonym="Español" data-language-local-name="Spanish" class="interlanguage-link-target">Español</a></li><li class="interlanguage-link interwiki-eo mw-list-item"><a href="https://eo.wikipedia.org/wiki/Genterapio" title="Genterapio – Esperanto" lang="eo" hreflang="eo" data-title="Genterapio" data-language-autonym="Esperanto" data-language-local-name="Esperanto" class="interlanguage-link-target">Esperanto</a></li><li class="interlanguage-link interwiki-eu mw-list-item"><a href="https://eu.wikipedia.org/wiki/Terapia_genetiko" title="Terapia genetiko – Basque" lang="eu" hreflang="eu" data-title="Terapia genetiko" data-language-autonym="Euskara" data-language-local-name="Basque" class="interlanguage-link-target">Euskara</a></li><li class="interlanguage-link interwiki-fa mw-list-item"><a href="https://fa.wikipedia.org/wiki/%DA%98%D9%86%E2%80%8C%D8%AF%D8%B1%D9%85%D8%A7%D9%86%DB%8C" title="ژن‌درمانی – Persian" lang="fa" hreflang="fa" data-title="ژن‌درمانی" data-language-autonym="فارسی" data-language-local-name="Persian" class="interlanguage-link-target">فارسی</a></li><li class="interlanguage-link interwiki-fr mw-list-item"><a href="https://fr.wikipedia.org/wiki/Th%C3%A9rapie_g%C3%A9nique" title="Thérapie génique – French" lang="fr" hreflang="fr" data-title="Thérapie génique" data-language-autonym="Français" data-language-local-name="French" class="interlanguage-link-target">Français</a></li><li class="interlanguage-link interwiki-gl mw-list-item"><a href="https://gl.wikipedia.org/wiki/Terapia_x%C3%A9nica" title="Terapia xénica – Galician" lang="gl" hreflang="gl" data-title="Terapia xénica" data-language-autonym="Galego" data-language-local-name="Galician" class="interlanguage-link-target">Galego</a></li><li class="interlanguage-link interwiki-ko mw-list-item"><a href="https://ko.wikipedia.org/wiki/%EC%9C%A0%EC%A0%84%EC%9E%90_%EC%B9%98%EB%A3%8C" title="유전자 치료 – Korean" lang="ko" hreflang="ko" data-title="유전자 치료" data-language-autonym="한국어" data-language-local-name="Korean" class="interlanguage-link-target">한국어</a></li><li class="interlanguage-link interwiki-hy mw-list-item"><a href="https://hy.wikipedia.org/wiki/%D4%B3%D5%A5%D5%B6%D5%A1%D5%B5%D5%AB%D5%B6_%D5%A9%D5%A5%D6%80%D5%A1%D5%BA%D5%AB%D5%A1" title="Գենային թերապիա – Armenian" lang="hy" hreflang="hy" data-title="Գենային թերապիա" data-language-autonym="Հայերեն" data-language-local-name="Armenian" class="interlanguage-link-target">Հայերեն</a></li><li class="interlanguage-link interwiki-hi mw-list-item"><a href="https://hi.wikipedia.org/wiki/%E0%A4%9C%E0%A5%80%E0%A4%A8_%E0%A4%9A%E0%A4%BF%E0%A4%95%E0%A4%BF%E0%A4%A4%E0%A5%8D%E0%A4%B8%E0%A4%BE" title="जीन चिकित्सा – Hindi" lang="hi" hreflang="hi" data-title="जीन चिकित्सा" data-language-autonym="हिन्दी" data-language-local-name="Hindi" class="interlanguage-link-target">हिन्दी</a></li><li class="interlanguage-link interwiki-hr mw-list-item"><a href="https://hr.wikipedia.org/wiki/Genska_terapija" title="Genska terapija – Croatian" lang="hr" hreflang="hr" data-title="Genska terapija" data-language-autonym="Hrvatski" data-language-local-name="Croatian" class="interlanguage-link-target">Hrvatski</a></li><li class="interlanguage-link interwiki-io mw-list-item"><a href="https://io.wikipedia.org/wiki/Gen-terapio" title="Gen-terapio – Ido" lang="io" hreflang="io" data-title="Gen-terapio" data-language-autonym="Ido" data-language-local-name="Ido" class="interlanguage-link-target">Ido</a></li><li class="interlanguage-link interwiki-id mw-list-item"><a href="https://id.wikipedia.org/wiki/Terapi_gen" title="Terapi gen – Indonesian" lang="id" hreflang="id" data-title="Terapi gen" data-language-autonym="Bahasa Indonesia" data-language-local-name="Indonesian" class="interlanguage-link-target">Bahasa Indonesia</a></li><li class="interlanguage-link interwiki-it mw-list-item"><a href="https://it.wikipedia.org/wiki/Terapia_genica" title="Terapia genica – Italian" lang="it" hreflang="it" data-title="Terapia genica" data-language-autonym="Italiano" data-language-local-name="Italian" class="interlanguage-link-target">Italiano</a></li><li class="interlanguage-link interwiki-he mw-list-item"><a href="https://he.wikipedia.org/wiki/%D7%A8%D7%99%D7%A4%D7%95%D7%99_%D7%92%D7%A0%D7%99" title="ריפוי גני – Hebrew" lang="he" hreflang="he" data-title="ריפוי גני" data-language-autonym="עברית" data-language-local-name="Hebrew" class="interlanguage-link-target">עברית</a></li><li class="interlanguage-link interwiki-jv mw-list-item"><a href="https://jv.wikipedia.org/wiki/Terapi_Gen" title="Terapi Gen – Javanese" lang="jv" hreflang="jv" data-title="Terapi Gen" data-language-autonym="Jawa" data-language-local-name="Javanese" 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href="https://no.wikipedia.org/wiki/Genterapi" title="Genterapi – Norwegian Bokmål" lang="nb" hreflang="nb" data-title="Genterapi" data-language-autonym="Norsk bokmål" data-language-local-name="Norwegian Bokmål" class="interlanguage-link-target">Norsk bokmål</a></li><li class="interlanguage-link interwiki-ps mw-list-item"><a href="https://ps.wikipedia.org/wiki/%D8%AC%DB%90%D9%86%DB%90%D9%BC%DB%8C%DA%A9%D9%87_%D8%AF%D8%B1%D9%85%D9%84%D9%86%D9%87" title="جېنېټیکه درملنه – Pashto" lang="ps" hreflang="ps" data-title="جېنېټیکه درملنه" data-language-autonym="پښتو" data-language-local-name="Pashto" class="interlanguage-link-target">پښتو</a></li><li class="interlanguage-link interwiki-pl mw-list-item"><a href="https://pl.wikipedia.org/wiki/Terapia_genowa" title="Terapia genowa – Polish" lang="pl" hreflang="pl" data-title="Terapia genowa" data-language-autonym="Polski" data-language-local-name="Polish" class="interlanguage-link-target">Polski</a></li><li class="interlanguage-link interwiki-pt mw-list-item"><a href="https://pt.wikipedia.org/wiki/Terapia_gen%C3%A9tica" title="Terapia genética – Portuguese" lang="pt" hreflang="pt" data-title="Terapia genética" data-language-autonym="Português" data-language-local-name="Portuguese" class="interlanguage-link-target">Português</a></li><li class="interlanguage-link interwiki-ru mw-list-item"><a href="https://ru.wikipedia.org/wiki/%D0%93%D0%B5%D0%BD%D0%BE%D1%82%D0%B5%D1%80%D0%B0%D0%BF%D0%B8%D1%8F" title="Генотерапия – Russian" lang="ru" hreflang="ru" data-title="Генотерапия" data-language-autonym="Русский" data-language-local-name="Russian" class="interlanguage-link-target">Русский</a></li><li class="interlanguage-link interwiki-sq mw-list-item"><a href="https://sq.wikipedia.org/wiki/Terapia_e_gjeneve" title="Terapia e gjeneve – Albanian" lang="sq" hreflang="sq" data-title="Terapia e gjeneve" data-language-autonym="Shqip" data-language-local-name="Albanian" class="interlanguage-link-target">Shqip</a></li><li 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a{color:var(--color-progressive)!important}}@media screen and (prefers-color-scheme:dark){html.skin-theme-clientpref-os .mw-parser-output .sidebar:not(.notheme) .sidebar-list-title,html.skin-theme-clientpref-os .mw-parser-output .sidebar:not(.notheme) .sidebar-title-with-pretitle{background:transparent!important}html.skin-theme-clientpref-os .mw-parser-output .sidebar:not(.notheme) .sidebar-title-with-pretitle a{color:var(--color-progressive)!important}}@media print{body.ns-0 .mw-parser-output .sidebar{display:none!important}}</style><table class="sidebar nomobile nowraplinks hlist"><tbody><tr><td class="sidebar-pretitle" style="padding-bottom:0.15em;">Part of <a href="/wiki/Category:Genetic_engineering" title="Category:Genetic engineering">a series</a> on</td></tr><tr><th class="sidebar-title-with-pretitle" style="font-size:175%;padding-top:0.2em;font-weight:normal;"><a href="/wiki/Genetic_engineering" title="Genetic engineering">Genetic engineering</a></th></tr><tr><td class="sidebar-image"><a href="/wiki/File:Genetic_engineering_logo.png" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/a/a1/Genetic_engineering_logo.png/150px-Genetic_engineering_logo.png" decoding="async" width="150" height="141" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/a/a1/Genetic_engineering_logo.png/225px-Genetic_engineering_logo.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/a/a1/Genetic_engineering_logo.png/300px-Genetic_engineering_logo.png 2x" data-file-width="459" data-file-height="432" /></a></td></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;;background:none;font-size:40%;line-height:0.4em;">  </td> </tr><tr><th class="sidebar-heading"> <a href="/wiki/Genetically_modified_organism" title="Genetically modified organism">Genetically modified organisms</a></th></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;"> <ul><li><a href="/wiki/Genetically_modified_bacteria" title="Genetically modified bacteria">Bacteria</a></li> <li><a href="/wiki/Genetically_modified_virus" title="Genetically modified virus">Viruses</a></li></ul> <ul><li><a href="/wiki/Genetically_modified_animal" title="Genetically modified animal">Animals</a> <ul><li><a href="/wiki/Genetically_modified_mammal" title="Genetically modified mammal">Mammals</a></li> <li><a href="/wiki/Genetically_modified_fish" title="Genetically modified fish">Fish</a></li> <li><a href="/wiki/Genetically_modified_insect" title="Genetically modified insect">Insects</a></li></ul></li></ul> <ul><li><a href="/wiki/Genetically_modified_plant" title="Genetically modified plant">Plants</a> <ul><li><a href="/wiki/Genetically_modified_maize" title="Genetically modified maize">Maize/corn</a></li> <li><a href="/wiki/Genetically_modified_rice" title="Genetically modified rice">Rice</a></li> <li><a href="/wiki/Genetically_modified_soybean" title="Genetically modified soybean">Soybean</a></li> <li><a href="/wiki/Genetically_modified_potato" title="Genetically modified potato">Potato</a></li></ul></li></ul></td> </tr><tr><th class="sidebar-heading"> History and regulation</th></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;"> <ul><li><a href="/wiki/History_of_genetic_engineering" title="History of genetic engineering">History</a></li></ul> <ul><li><a href="/wiki/Regulation_of_genetic_engineering" title="Regulation of genetic engineering">Regulation</a> <ul><li><a href="/wiki/Substantial_equivalence" title="Substantial equivalence">Substantial equivalence</a></li> <li><a href="/wiki/Cartagena_Protocol_on_Biosafety" title="Cartagena Protocol on Biosafety">Cartagena Protocol on Biosafety</a></li></ul></li></ul></td> </tr><tr><th class="sidebar-heading"> Process</th></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;"> <ul><li><a href="/wiki/Genetic_engineering_techniques" title="Genetic engineering techniques">Techniques</a></li> <li><a href="/wiki/Molecular_cloning" title="Molecular cloning">Molecular cloning</a> <ul><li><a href="/wiki/Recombinant_DNA" title="Recombinant DNA">Recombinant DNA</a></li></ul></li> <li><a href="/wiki/Gene_delivery" title="Gene delivery">Gene delivery</a> <ul><li><a href="/wiki/Transformation_(genetics)" class="mw-redirect" title="Transformation (genetics)">Transformation</a></li> <li><a href="/wiki/Transfection" title="Transfection">Transfection</a></li> <li><a href="/wiki/Transduction_(genetics)" title="Transduction (genetics)">Transduction</a></li></ul></li> <li><a href="/wiki/Genome_editing" title="Genome editing">Genome editing</a> <ul><li><a href="/wiki/TALEN" class="mw-redirect" title="TALEN">TALEN</a></li> <li><a href="/wiki/CRISPR" title="CRISPR">CRISPR</a></li></ul></li></ul></td> </tr><tr><th class="sidebar-heading"> Applications</th></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;"> <ul><li><a href="/wiki/Genetically_modified_crops" title="Genetically modified crops">Genetically modified crops</a> <ul><li><a href="/wiki/Genetically_modified_food" title="Genetically modified food">food</a></li></ul></li> <li><a class="mw-selflink selflink">Gene therapy</a></li> <li><a href="/wiki/Designer_baby" title="Designer baby">Designer baby</a></li></ul></td> </tr><tr><th class="sidebar-heading"> Controversies</th></tr><tr><td class="sidebar-content" style="padding:0.2em 0.4em 0.4em;"> <ul><li><a href="/wiki/Genetically_modified_food_controversies" title="Genetically modified food controversies">Genetically modified food controversies</a></li> <li><a href="/wiki/GMO_conspiracy_theories" title="GMO conspiracy theories">GMO conspiracy theories</a></li> <li><a href="/wiki/Pusztai_affair" title="Pusztai affair">Pusztai affair</a></li> <li><a href="/wiki/S%C3%A9ralini_affair" title="Séralini affair">Séralini affair</a></li> <li><a href="/wiki/StarLink_corn_recall" title="StarLink corn recall">StarLink corn recall</a></li> <li><a href="/wiki/He_Jiankui_affair" title="He Jiankui affair">He Jiankui affair</a></li></ul></td> </tr><tr><td class="sidebar-navbar"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><style data-mw-deduplicate="TemplateStyles:r1239400231">.mw-parser-output .navbar{display:inline;font-size:88%;font-weight:normal}.mw-parser-output .navbar-collapse{float:left;text-align:left}.mw-parser-output .navbar-boxtext{word-spacing:0}.mw-parser-output .navbar ul{display:inline-block;white-space:nowrap;line-height:inherit}.mw-parser-output .navbar-brackets::before{margin-right:-0.125em;content:"[ "}.mw-parser-output .navbar-brackets::after{margin-left:-0.125em;content:" ]"}.mw-parser-output .navbar li{word-spacing:-0.125em}.mw-parser-output .navbar a>span,.mw-parser-output .navbar a>abbr{text-decoration:inherit}.mw-parser-output .navbar-mini abbr{font-variant:small-caps;border-bottom:none;text-decoration:none;cursor:inherit}.mw-parser-output .navbar-ct-full{font-size:114%;margin:0 7em}.mw-parser-output .navbar-ct-mini{font-size:114%;margin:0 4em}html.skin-theme-clientpref-night .mw-parser-output .navbar li a abbr{color:var(--color-base)!important}@media(prefers-color-scheme:dark){html.skin-theme-clientpref-os .mw-parser-output .navbar li a abbr{color:var(--color-base)!important}}@media print{.mw-parser-output .navbar{display:none!important}}</style><div class="navbar plainlinks hlist navbar-mini"><ul><li class="nv-view"><a href="/wiki/Template:Genetic_engineering_sidebar" title="Template:Genetic engineering sidebar"><abbr title="View this template">v</abbr></a></li><li class="nv-talk"><a href="/wiki/Template_talk:Genetic_engineering_sidebar" title="Template talk:Genetic engineering sidebar"><abbr title="Discuss this template">t</abbr></a></li><li class="nv-edit"><a href="/wiki/Special:EditPage/Template:Genetic_engineering_sidebar" title="Special:EditPage/Template:Genetic engineering sidebar"><abbr title="Edit this template">e</abbr></a></li></ul></div></td></tr></tbody></table> Gene therapy is a <a href="/wiki/Health_technology" title="Health technology">medical technology</a> that aims to produce a <a href="/wiki/Therapeutic_effect" title="Therapeutic effect">therapeutic effect</a> through the manipulation of <a href="/wiki/Gene_expression" title="Gene expression">gene expression</a> or through altering the biological properties of living cells.<a href="#cite_note-1">[1]</a><a href="#cite_note-2">[2]</a><a href="#cite_note-3">[3]</a> The first attempt at modifying human <a href="/wiki/DNA" title="DNA">DNA</a> was performed in 1980, by <a href="/wiki/Martin_Cline" title="Martin Cline">Martin Cline</a>, but the first successful nuclear gene transfer in humans, approved by the <a href="/wiki/National_Institutes_of_Health" title="National Institutes of Health">National Institutes of Health</a>, was performed in May 1989.<a href="#cite_note-4">[4]</a> The first therapeutic use of gene transfer as well as the first direct insertion of human DNA into the nuclear genome was performed by <a href="/wiki/French_Anderson" class="mw-redirect" title="French Anderson">French Anderson</a> in a trial starting in September 1990. Between 1989 and December 2018, over 2,900 clinical trials were conducted, with more than half of them in <a href="/wiki/Phases_of_clinical_research" title="Phases of clinical research">phase I</a>.<a href="#cite_note-JGenMed_Database-5">[5]</a> In 2003, <a href="/wiki/Gendicine" title="Gendicine">Gendicine</a> became the first gene therapy to receive regulatory approval. Since that time, further gene therapy drugs were approved, such as <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a> (2012), <a href="/wiki/Strimvelis" title="Strimvelis">Strimvelis</a> (2016), <a href="/wiki/Tisagenlecleucel" title="Tisagenlecleucel">tisagenlecleucel</a> (2017), <a href="/wiki/Voretigene_neparvovec" title="Voretigene neparvovec">voretigene neparvovec</a> (2017), <a href="/wiki/Patisiran" title="Patisiran">patisiran</a> (2018), <a href="/wiki/Onasemnogene_abeparvovec" title="Onasemnogene abeparvovec">onasemnogene abeparvovec</a> (2019), <a href="/wiki/Idecabtagene_vicleucel" title="Idecabtagene vicleucel">idecabtagene vicleucel</a> (2021), <a href="/wiki/Nadofaragene_firadenovec" title="Nadofaragene firadenovec">nadofaragene firadenovec</a>, <a href="/wiki/Valoctocogene_roxaparvovec" title="Valoctocogene roxaparvovec">valoctocogene roxaparvovec</a> and <a href="/wiki/Etranacogene_dezaparvovec" title="Etranacogene dezaparvovec">etranacogene dezaparvovec</a> (all 2022). Most of these approaches utilize <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated viruses</a> (AAVs) and <a href="/wiki/Lentivirus" title="Lentivirus">lentiviruses</a> for performing gene insertions, <a href="/wiki/In_vivo" title="In vivo">in vivo</a> and <a href="/wiki/Ex_vivo" title="Ex vivo">ex vivo</a>, respectively. AAVs are characterized by stabilizing the <a href="/wiki/Viral_capsid" class="mw-redirect" title="Viral capsid">viral capsid</a>, lower immunogenicity, ability to <a href="/wiki/Transduction_(genetics)" title="Transduction (genetics)">transduce</a> both dividing and nondividing cells, the potential to integrate site specifically and to achieve long-term expression in the in-vivo treatment.<a href="#cite_note-Gorell_2014-6">[6]</a> <a href="/wiki/Allele-specific_oligonucleotide" title="Allele-specific oligonucleotide">ASO</a> / <a href="/wiki/Small_interfering_RNA" title="Small interfering RNA">siRNA</a> approaches such as those conducted by <a href="/wiki/Alnylam" class="mw-redirect" title="Alnylam">Alnylam</a> and <a href="/wiki/Ionis_Pharmaceuticals" title="Ionis Pharmaceuticals">Ionis Pharmaceuticals</a> require non-viral delivery systems, and utilize alternative mechanisms for trafficking to liver cells by way of <a href="/wiki/GalNAc" class="mw-redirect" title="GalNAc">GalNAc</a> transporters. Not all medical procedures that introduce alterations to a patient's genetic makeup can be considered gene therapy. <a href="/wiki/Bone_marrow_transplantation" class="mw-redirect" title="Bone marrow transplantation">Bone marrow transplantation</a> and <a href="/wiki/Organ_transplants" class="mw-redirect" title="Organ transplants">organ transplants</a> in general have been found to introduce foreign DNA into patients.<a href="#cite_note-7">[7]</a> <meta property="mw:PageProp/toc" /> <div class="mw-heading mw-heading2"><h2 id="Background">Background</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=1" title="Edit section: Background">edit</a>]</div> Gene therapy was first conceptualized in the 1960s, when the feasibility of adding new genetic functions to <a href="/wiki/Mammal" title="Mammal">mammalian</a> <a href="/wiki/Cell_(biology)" title="Cell (biology)">cells</a> began to be researched. Several methods to do so were tested, including injecting <a href="/wiki/Gene" title="Gene">genes</a> with a micropipette directly into a living mammalian cell, and exposing cells to a precipitate of <a href="/wiki/DNA" title="DNA">DNA</a> that contained the desired genes. Scientists theorized that a <a href="/wiki/Virus" title="Virus">virus</a> could also be used as a vehicle, or vector, to deliver new genes into cells. One of the first scientists to report the successful direct incorporation of functional <a href="/wiki/DNA" title="DNA">DNA</a> into a mammalian cell was <a href="/wiki/Biochemist" title="Biochemist">biochemist</a> Dr. Lorraine Marquardt Kraus (6 September 1922 – 1 July 2016)<a href="#cite_note-The_Commercial_Appeal-8">[8]</a> at the <a href="/wiki/University_of_Tennessee_Health_Science_Center" title="University of Tennessee Health Science Center">University of Tennessee Health Science Center</a> in <a href="/wiki/Memphis,_Tennessee" title="Memphis, Tennessee">Memphis, Tennessee</a>. In 1961, she managed to genetically alter the <a href="/wiki/Hemoglobin" title="Hemoglobin">hemoglobin</a> of <a href="/wiki/Cell_(biology)" title="Cell (biology)">cells</a> from <a href="/wiki/Bone_marrow" title="Bone marrow">bone marrow</a> taken from a patient with <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell anaemia</a>. She did this by incubating the patient's <a href="/wiki/Cell_(biology)" title="Cell (biology)">cells</a> in tissue culture with <a href="/wiki/DNA" title="DNA">DNA</a> extracted from a donor with normal <a href="/wiki/Hemoglobin" title="Hemoglobin">hemoglobin</a>. In 1968, researchers <a href="/wiki/Theodore_Friedmann" title="Theodore Friedmann">Theodore Friedmann</a>, Jay Seegmiller, and John Subak-Sharpe at the <a href="/wiki/National_Institutes_of_Health" title="National Institutes of Health">National Institutes of Health</a> (NIH), Bethesda, in the <a href="/wiki/United_States" title="United States">United States</a> successfully corrected genetic defects associated with <a href="/wiki/Lesch-Nyhan_syndrome" class="mw-redirect" title="Lesch-Nyhan syndrome">Lesch-Nyhan syndrome</a>, a debilitating <a href="/wiki/Neurological_disease" class="mw-redirect" title="Neurological disease">neurological disease</a>, by adding foreign <a href="/wiki/DNA" title="DNA">DNA</a> to cultured cells collected from patients suffering from the disease.<a href="#cite_note-Biotechmet-9">[9]</a> The first attempt, an unsuccessful one, at gene therapy (as well as the first case of medical transfer of foreign genes into humans not counting <a href="/wiki/Organ_transplantation" title="Organ transplantation">organ transplantation</a>) was performed by <a href="/wiki/Geneticist" title="Geneticist">geneticist</a> <a href="/wiki/Martin_Cline" title="Martin Cline">Martin Cline</a> of the <a href="/wiki/University_of_California,_Los_Angeles" title="University of California, Los Angeles">University of California, Los Angeles</a> in <a href="/wiki/California_(state)" class="mw-redirect" title="California (state)">California</a>, <a href="/wiki/United_States" title="United States">United States</a> on 10 July 1980.<a href="#cite_note-10">[10]</a><a href="#cite_note-11">[11]</a> Cline claimed that one of the genes in his patients was active six months later, though he never published this data or had it verified.<a href="#cite_note-12">[12]</a> After extensive research on animals throughout the 1980s and a 1989 bacterial gene tagging trial on humans, the first gene therapy widely accepted as a success was demonstrated in a trial that started on 14 September 1990, when Ashanthi DeSilva was treated for <a href="/wiki/Adenosine_deaminase_deficiency" title="Adenosine deaminase deficiency">ADA</a>-<a href="/wiki/Severe_combined_immunodeficiency" title="Severe combined immunodeficiency">SCID</a>.<a href="#cite_note-Gene_Therapy_Finds_Its_Niche-13">[13]</a> The first somatic treatment that produced a permanent genetic change was initiated in 1993.<a href="#cite_note-14">[14]</a> The goal was to cure malignant brain tumors by using recombinant DNA to transfer a gene making the tumor cells sensitive to a drug that in turn would cause the tumor cells to die.<a href="#cite_note-15">[15]</a> The polymers are either <a href="/wiki/Translation_(biology)" title="Translation (biology)">translated</a> into <a href="/wiki/Proteins" class="mw-redirect" title="Proteins">proteins</a>, interfere with target <a href="/wiki/Gene_expression" title="Gene expression">gene expression</a>, or possibly correct <a href="/wiki/Genetic_mutations" class="mw-redirect" title="Genetic mutations">genetic mutations</a>. The most common form uses <a href="/wiki/DNA" title="DNA">DNA</a> that encodes a functional, therapeutic <a href="/wiki/Gene" title="Gene">gene</a> to replace a <a href="/wiki/Mutate" class="mw-redirect" title="Mutate">mutated</a> gene. The polymer molecule is packaged within a "<a href="/wiki/Vector_(molecular_biology)" title="Vector (molecular biology)">vector</a>", which carries the molecule inside cells.[<a href="/wiki/Wikipedia:Identifying_reliable_sources_(medicine)" title="Wikipedia:Identifying reliable sources (medicine)">medical citation needed</a>] Early clinical failures led to dismissals of gene therapy. Clinical successes since 2006 regained researchers' attention, although as of 2014<a class="external text" href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&action=edit">[update]</a>, it was still largely an experimental technique.<a href="#cite_note-Richards2012-16">[16]</a> These include treatment of <a href="/wiki/Retinal_diseases" class="mw-redirect" title="Retinal diseases">retinal diseases</a> <a href="/wiki/Leber%27s_congenital_amaurosis" class="mw-redirect" title="Leber's congenital amaurosis">Leber's congenital amaurosis</a><a href="#cite_note-Maguire_2008-17">[17]</a><a href="#cite_note-Simonelli_2010-18">[18]</a><a href="#cite_note-Cideciyan_2009-19">[19]</a><a href="#cite_note-Bainbridge_2008-20">[20]</a> and <a href="/wiki/Choroideremia" title="Choroideremia">choroideremia</a>,<a href="#cite_note-BBC2016-21">[21]</a> <a href="/wiki/X-linked_severe_combined_immunodeficiency" title="X-linked severe combined immunodeficiency">X-linked SCID</a>,<a href="#cite_note-22">[22]</a> ADA-SCID,<a href="#cite_note-23">[23]</a><a href="#cite_note-NewScientist1013-24">[24]</a> <a href="/wiki/Adrenoleukodystrophy" title="Adrenoleukodystrophy">adrenoleukodystrophy</a>,<a href="#cite_note-25">[25]</a> <a href="/wiki/Chronic_lymphocytic_leukemia" title="Chronic lymphocytic leukemia">chronic lymphocytic leukemia</a> (CLL),<a href="#cite_note-Porter-26">[26]</a> <a href="/wiki/Acute_lymphocytic_leukemia" class="mw-redirect" title="Acute lymphocytic leukemia">acute lymphocytic leukemia</a> (ALL),<a href="#cite_note-SloanKettering-27">[27]</a> <a href="/wiki/Multiple_myeloma" title="Multiple myeloma">multiple myeloma</a>,<a href="#cite_note-Adaptimmune-28">[28]</a> <a href="/wiki/Haemophilia" title="Haemophilia">haemophilia</a>,<a href="#cite_note-NewScientist1013-24">[24]</a> and <a href="/wiki/Parkinson%27s_disease" title="Parkinson's disease">Parkinson's disease</a>.<a href="#cite_note-29">[29]</a> Between 2013 and April 2014, US companies invested over $600 million in the field.<a href="#cite_note-Forbes-30">[30]</a> The first commercial gene therapy, <a href="/wiki/Gendicine" title="Gendicine">Gendicine</a>, was approved in China in 2003, for the treatment of certain cancers.<a href="#cite_note-Gend-31">[31]</a> In 2011, <a href="/wiki/Neovasculgen" class="mw-redirect" title="Neovasculgen">Neovasculgen</a> was registered in Russia as the first-in-class gene-therapy drug for treatment of <a href="/wiki/Peripheral_artery_disease" title="Peripheral artery disease">peripheral artery disease</a>, including <a href="/wiki/Critical_limb_ischemia" class="mw-redirect" title="Critical limb ischemia">critical limb ischemia</a>.<a href="#cite_note-Neuvasculgen-32">[32]</a> In 2012, <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a>, a treatment for a rare <a href="/wiki/Inherited_disorder" class="mw-redirect" title="Inherited disorder">inherited disorder</a>, <a href="/wiki/Lipoprotein_lipase_deficiency" title="Lipoprotein lipase deficiency">lipoprotein lipase deficiency</a>, became the first treatment to be approved for clinical use in either the European Union or the United States after its endorsement by the <a href="/wiki/European_Commission" title="European Commission">European Commission</a>.<a href="#cite_note-Richards2012-16">[16]</a><a href="#cite_note-Gallagher-33">[33]</a> Following early advances in <a href="/wiki/Genetic_engineering" title="Genetic engineering">genetic engineering</a> of bacteria, cells, and small animals, scientists started considering how to apply it to medicine. Two main approaches were considered – replacing or disrupting defective genes.<a href="#cite_note-34">[34]</a> Scientists focused on diseases caused by single-gene defects, such as <a href="/wiki/Cystic_fibrosis" title="Cystic fibrosis">cystic fibrosis</a>, haemophilia, <a href="/wiki/Muscular_dystrophy" title="Muscular dystrophy">muscular dystrophy</a>, <a href="/wiki/Thalassemia" title="Thalassemia">thalassemia</a>, and <a href="/wiki/Sickle_cell_anemia" class="mw-redirect" title="Sickle cell anemia">sickle cell anemia</a>. <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a> treats one such disease, caused by a defect in <a href="/wiki/Lipoprotein_lipase" title="Lipoprotein lipase">lipoprotein lipase</a>.<a href="#cite_note-Gallagher-33">[33]</a> DNA must be administered, reach the damaged cells, enter the cell and either express or disrupt a protein.<a href="#cite_note-35">[35]</a> Multiple delivery techniques have been explored. The initial approach incorporated DNA into an engineered <a href="/wiki/Virus" title="Virus">virus</a> to deliver the DNA into a <a href="/wiki/Chromosome" title="Chromosome">chromosome</a>.<a href="#cite_note-pmid23015375-36">[36]</a><a href="#cite_note-pmid23435812-37">[37]</a> <a href="/wiki/Naked_DNA" class="mw-redirect" title="Naked DNA">Naked DNA</a> approaches have also been explored, especially in the context of <a href="/wiki/Vaccine" title="Vaccine">vaccine</a> development.<a href="#cite_note-pmid23111168-38">[38]</a> Generally, efforts focused on administering a gene that causes a needed protein to be expressed. More recently, increased understanding of <a href="/wiki/Nucleases" class="mw-redirect" title="Nucleases">nuclease</a> function has led to more direct DNA editing, using techniques such as <a href="/wiki/Zinc_finger_nucleases" class="mw-redirect" title="Zinc finger nucleases">zinc finger nucleases</a> and <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a>. The vector incorporates genes into chromosomes. The expressed nucleases then knock out and replace genes in the chromosome. As of 2014<a class="external text" href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&action=edit">[update]</a> these approaches involve removing cells from patients, editing a chromosome and returning the transformed cells to patients.<a href="#cite_note-pmid20717154-39">[39]</a> <a href="/wiki/Genome_editing" title="Genome editing">Gene editing</a> is a potential approach to alter the human genome to treat genetic diseases,<a href="#cite_note-Bak_600–611-40">[40]</a> viral diseases,<a href="#cite_note-41">[41]</a> and cancer.<a href="#cite_note-42">[42]</a><a href="#cite_note-43">[43]</a> As of 2020<a class="external text" href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&action=edit">[update]</a> these approaches are being studied in clinical trials.<a href="#cite_note-44">[44]</a><a href="#cite_note-45">[45]</a> <div class="mw-heading mw-heading2"><h2 id="Classification">Classification</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=2" title="Edit section: Classification">edit</a>]</div> <div class="mw-heading mw-heading3"><h3 id="Breadth_of_definition">Breadth of definition</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=3" title="Edit section: Breadth of definition">edit</a>]</div> In 1986, a meeting at the <a href="/wiki/Institute_Of_Medicine" class="mw-redirect" title="Institute Of Medicine">Institute Of Medicine</a> defined gene therapy as the addition or replacement of a gene in a targeted cell type. In the same year, the FDA announced that it had jurisdiction over approving "gene therapy" without defining the term. The FDA added a very broad definition in 1993 of any treatment that would 'modify or manipulate the expression of genetic material or to alter the biological properties of living cells'. In 2018 this was narrowed to 'products that mediate their effects by transcription or translation of transferred genetic material or by specifically altering host (human) genetic sequences'.<a href="#cite_note-Sherkow_2018-46">[46]</a> Writing in 2018, in the Journal of Law and the Biosciences, Sherkow et al. argued for a narrower definition of gene therapy than the FDA's in light of new technology that would consist of any treatment that intentionally and permanently modified a cell's genome, with the <a href="/wiki/Genome#definition" title="Genome">definition of genome</a> including episomes outside the nucleus but excluding changes due to episomes that are lost over time. This definition would also exclude introducing cells that did not derive from a patient themselves, but include ex vivo approaches, and would not depend on the vector used.<a href="#cite_note-Sherkow_2018-46">[46]</a> During the <a href="/wiki/COVID-19" title="COVID-19">COVID-19</a> pandemic, some academics insisted that the <a href="/wiki/MRNA_vaccine" title="MRNA vaccine">mRNA vaccines</a> for COVID were not gene therapy to prevent the spread of incorrect information that the vaccine could alter DNA, other academics maintained that the vaccines were a gene therapy because they introduced genetic material into a cell.<a href="#cite_note-47">[47]</a> <a href="/wiki/Fact-checking" title="Fact-checking">Fact-checkers</a>, such as <a href="/wiki/Full_Fact" title="Full Fact">Full Fact</a>,<a href="#cite_note-48">[48]</a> <a href="/wiki/Reuters" title="Reuters">Reuters</a>,<a href="#cite_note-49">[49]</a> <a href="/wiki/PolitiFact" title="PolitiFact">PolitiFact</a>,<a href="#cite_note-50">[50]</a> and <a href="/wiki/FactCheck.org" title="FactCheck.org">FactCheck.org</a><a href="#cite_note-51">[51]</a> said that calling the vaccines a gene therapy was incorrect. Podcast host <a href="/wiki/Joe_Rogan" title="Joe Rogan">Joe Rogan</a> was criticized for calling mRNA vaccines gene therapy as was British politician <a href="/wiki/Andrew_Bridgen" title="Andrew Bridgen">Andrew Bridgen</a>, with fact checker Full Fact calling for Bridgen to be removed from the conservative party for this and other statements.<a href="#cite_note-52">[52]</a><a href="#cite_note-53">[53]</a> <div class="mw-heading mw-heading3"><h3 id="Genes_present_or_added">Genes present or added</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=4" title="Edit section: Genes present or added">edit</a>]</div> Gene therapy encapsulates many forms of adding different <a href="/wiki/Nucleic_acid" title="Nucleic acid">nucleic acids</a> to a cell. Gene augmentation adds a new protein coding gene to a cell. One form of gene augmentiation is gene replacement therapy, a treatment for <a href="/wiki/Monogenic_(genetics)" class="mw-redirect" title="Monogenic (genetics)">monogenic</a> recessive disorders where a single gene is not functional an additional functional gene is added. For diseases caused by multiple genes or a dominant gene, <a href="/wiki/Gene_silencing" title="Gene silencing">gene silencing</a> or gene editing approaches are more appropriate but gene addition, a form of gene augmentation where new gene is added, may improve a cells function without modifying the genes that cause a disorder.<a href="#cite_note-:4-54">[54]</a>: 117  <div class="mw-heading mw-heading3"><h3 id="Cell_types">Cell types</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=5" title="Edit section: Cell types">edit</a>]</div> Gene therapy may be classified into two types by the type of cell it affects: somatic cell and germline gene therapy. In <a href="/wiki/Somatic_cell" title="Somatic cell">somatic cell</a> gene therapy (SCGT), the therapeutic genes are transferred into any cell other than a <a href="/wiki/Gamete" title="Gamete">gamete</a>, <a href="/wiki/Germ_cell" title="Germ cell">germ cell</a>, <a href="/wiki/Gametocyte" title="Gametocyte">gametocyte</a>, or undifferentiated <a href="/wiki/Stem_cell" title="Stem cell">stem cell</a>. Any such modifications affect the individual patient only, and are not inherited by <a href="/wiki/Offspring" title="Offspring">offspring</a>. Somatic gene therapy represents mainstream basic and clinical research, in which therapeutic DNA (either integrated in the <a href="/wiki/Genome" title="Genome">genome</a> or as an external <a href="/wiki/Episome" title="Episome">episome</a> or <a href="/wiki/Plasmid" title="Plasmid">plasmid</a>) is used to treat disease.<a href="#cite_note-55">[55]</a> Over 600 <a href="/wiki/Clinical_trials" class="mw-redirect" title="Clinical trials">clinical trials</a> utilizing SCGT are underway[<a href="/wiki/Wikipedia:Manual_of_Style/Dates_and_numbers#Chronological_items" title="Wikipedia:Manual of Style/Dates and numbers">when?</a>] in the US. Most focus on severe genetic disorders, including <a href="/wiki/Primary_immunodeficiency" title="Primary immunodeficiency">immunodeficiencies</a>, <a href="/wiki/Haemophilia" title="Haemophilia">haemophilia</a>, <a href="/wiki/Thalassemia" title="Thalassemia">thalassaemia</a>, and <a href="/wiki/Cystic_fibrosis" title="Cystic fibrosis">cystic fibrosis</a>. Such single gene disorders are good candidates for somatic cell therapy. The complete correction of a genetic disorder or the replacement of multiple genes is not yet possible. Only a few of the trials are in the advanced stages.<a href="#cite_note-somatic-56">[56]</a>[<a href="/wiki/Wikipedia:Manual_of_Style/Dates_and_numbers#Chronological_items" title="Wikipedia:Manual of Style/Dates and numbers">needs update</a>] In <a href="/wiki/Germline" title="Germline">germline</a> gene therapy (GGT), <a href="/wiki/Germ_cell" title="Germ cell">germ cells</a> (<a href="/wiki/Sperm" title="Sperm">sperm</a> or <a href="/wiki/Egg_cell" title="Egg cell">egg cells</a>) are modified by the introduction of functional genes into their genomes. Modifying a germ cell causes all the organism's cells to contain the modified gene. The change is therefore <a href="/wiki/Heritable" class="mw-redirect" title="Heritable">heritable</a> and passed on to later generations. Australia, Canada, Germany, Israel, Switzerland, and the Netherlands<a href="#cite_note-JHU-57">[57]</a> prohibit GGT for application in human beings, for technical and ethical reasons, including insufficient knowledge about possible risks to future generations<a href="#cite_note-JHU-57">[57]</a> and higher risks versus SCGT.<a href="#cite_note-58">[58]</a> The US has no federal controls specifically addressing human genetic modification (beyond FDA regulations for therapies in general).<a href="#cite_note-JHU-57">[57]</a><a href="#cite_note-germline-59">[59]</a><a href="#cite_note-somatic2-60">[60]</a><a href="#cite_note-germline2-61">[61]</a> <div class="mw-heading mw-heading3"><h3 id="In_vivo_versus_ex_vivo_therapies">In vivo versus ex vivo therapies</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=6" title="Edit section: In vivo versus ex vivo therapies">edit</a>]</div> <figure class="mw-default-size" typeof="mw:File/Thumb"><a href="/wiki/File:ExVivoGeneTherapy.jpg" class="mw-file-description"><img alt="Ex vivo gene therapy" src="//upload.wikimedia.org/wikipedia/commons/thumb/f/f3/ExVivoGeneTherapy.jpg/220px-ExVivoGeneTherapy.jpg" decoding="async" width="220" height="165" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/f/f3/ExVivoGeneTherapy.jpg/330px-ExVivoGeneTherapy.jpg 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/f/f3/ExVivoGeneTherapy.jpg/440px-ExVivoGeneTherapy.jpg 2x" data-file-width="730" data-file-height="549" /></a><figcaption></figcaption></figure> In <a href="/wiki/In_vivo" title="In vivo">in vivo</a> gene therapy, a vector (typically, a virus) is introduced to the patient, which then achieves the desired biological effect by passing the genetic material (e.g. for a missing protein) into the patient's cells. In <a href="/wiki/Ex_vivo" title="Ex vivo">ex vivo</a> gene therapies, such as <a href="/wiki/Chimeric_antigen_receptor_T_cell" class="mw-redirect" title="Chimeric antigen receptor T cell">CAR-T</a> therapeutics, the patient's own cells (autologous) or healthy donor cells (allogeneic) are modified outside the body (hence, ex vivo) using a vector to express a particular protein, such as a chimeric antigen receptor.<a href="#cite_note-62">[62]</a> In vivo gene therapy is seen as simpler, since it does not require the harvesting of <a href="/wiki/Mitosis" title="Mitosis">mitotic</a> cells. However, ex vivo gene therapies are better tolerated and less associated with severe immune responses.<a href="#cite_note-63">[63]</a> The death of <a href="/wiki/Jesse_Gelsinger" title="Jesse Gelsinger">Jesse Gelsinger</a> in a trial of an <a href="/wiki/Adenoviridae" title="Adenoviridae">adenovirus</a>-vectored treatment for <a href="/wiki/Ornithine_transcarbamylase_deficiency" title="Ornithine transcarbamylase deficiency">ornithine transcarbamylase deficiency</a> due to a systemic inflammatory reaction led to a temporary halt on gene therapy trials across the United States.<a href="#cite_note-64">[64]</a> As of 2021<a class="external text" href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&action=edit">[update]</a>, in vivo and ex vivo therapeutics are both seen as safe.<a href="#cite_note-65">[65]</a> <div class="mw-heading mw-heading3"><h3 id="Gene_editing">Gene editing</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=7" title="Edit section: Gene editing">edit</a>]</div> <style data-mw-deduplicate="TemplateStyles:r1236090951">.mw-parser-output .hatnote{font-style:italic}.mw-parser-output div.hatnote{padding-left:1.6em;margin-bottom:0.5em}.mw-parser-output .hatnote i{font-style:normal}.mw-parser-output .hatnote+link+.hatnote{margin-top:-0.5em}@media print{body.ns-0 .mw-parser-output .hatnote{display:none!important}}</style><div role="note" class="hatnote navigation-not-searchable">See also: <a href="/wiki/CRISPR_gene_editing" title="CRISPR gene editing">CRISPR gene editing</a></div><figure class="mw-default-size" typeof="mw:File/Thumb"><a href="/wiki/File:CRISPR-Cas9_mode_of_action.png" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/9/93/CRISPR-Cas9_mode_of_action.png/220px-CRISPR-Cas9_mode_of_action.png" decoding="async" width="220" height="60" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/9/93/CRISPR-Cas9_mode_of_action.png/330px-CRISPR-Cas9_mode_of_action.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/9/93/CRISPR-Cas9_mode_of_action.png/440px-CRISPR-Cas9_mode_of_action.png 2x" data-file-width="717" data-file-height="197" /></a><figcaption>A duplex of crRNA and <a href="/wiki/Trans-activating_crRNA" title="Trans-activating crRNA">tracrRNA</a> acts as guide RNA to introduce a specifically located gene modification based on the RNA 5' upstream of the crRNA. Cas9 binds the tracrRNA and needs a DNA binding sequence (5'NGG3'), which is called protospacer adjacent motif (PAM). After binding, Cas9 introduces a DNA double strand break, which is then followed by gene modification via homologous recombination (HDR) or non-homologous end joining (NHEJ).</figcaption></figure>The concept of gene therapy is to fix a genetic problem at its source. If, for instance, a mutation in a certain gene causes the production of a dysfunctional protein resulting (usually recessively) in an inherited disease, gene therapy could be used to deliver a copy of this gene that does not contain the deleterious mutation and thereby produces a functional protein. This strategy is referred to as gene replacement therapy and could be employed to treat inherited retinal diseases.<a href="#cite_note-Maguire_2008-17">[17]</a><a href="#cite_note-pmid24439297-66">[66]</a> While the concept of gene replacement therapy is mostly suitable for recessive diseases, novel strategies have been suggested that are capable of also treating conditions with a dominant pattern of inheritance. <ul><li>The introduction of <a href="/wiki/CRISPR_gene_editing" title="CRISPR gene editing">CRISPR gene editing</a> has opened new doors for its application and utilization in gene therapy, as instead of pure replacement of a gene, it enables correction of the particular genetic defect.<a href="#cite_note-Bak_600–611-40">[40]</a> Solutions to medical hurdles, such as the eradication of latent human immunodeficiency virus (<a href="/wiki/HIV" title="HIV">HIV</a>) reservoirs and correction of the mutation that causes sickle cell disease, may be available as a therapeutic option in the future.<a href="#cite_note-67">[67]</a><a href="#cite_note-68">[68]</a><a href="#cite_note-69">[69]</a></li> <li>Prosthetic gene therapy aims to enable cells of the body to take over functions they physiologically do not carry out. One example is the so-called vision restoration gene therapy, that aims to restore vision in patients with end-stage retinal diseases.<a href="#cite_note-70">[70]</a><a href="#cite_note-71">[71]</a> In end-stage retinal diseases, the photoreceptors, as the primary light sensitive cells of the retina are irreversibly lost. By the means of prosthetic gene therapy light sensitive proteins are delivered into the remaining cells of the retina, to render them light sensitive and thereby enable them to signal visual information towards the brain.</li></ul> In vivo, gene editing systems using <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a> have been used in studies with mice to treat cancer and have been effective at reducing tumors.<a href="#cite_note-:8-72">[72]</a>: 18  In vitro, the CRISPR system has been used to treat HPV cancer tumors. <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">Adeno-associated virus</a>, <a href="/wiki/Lentivirus" title="Lentivirus">Lentivirus</a> based vectors have been to introduce the genome for the CRISPR system.<a href="#cite_note-:8-72">[72]</a>: 6  <div class="mw-heading mw-heading2"><h2 id="Vectors">Vectors</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=8" title="Edit section: Vectors">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">Main article: <a href="/wiki/Vectors_in_gene_therapy" title="Vectors in gene therapy">Vectors in gene therapy</a></div> The delivery of DNA into cells can be accomplished by multiple <a href="/wiki/Vectors_in_Gene_Therapy" class="mw-redirect" title="Vectors in Gene Therapy">methods</a>. The two major classes are <a href="/wiki/Recombinant_virus" title="Recombinant virus">recombinant viruses</a> (sometimes called biological <a href="/wiki/Nanoparticle" title="Nanoparticle">nanoparticles</a> or viral vectors) and <a href="/wiki/Naked_DNA" class="mw-redirect" title="Naked DNA">naked DNA</a> or DNA complexes (non-viral methods).<a href="#cite_note-73">[73]</a> <div class="mw-heading mw-heading3"><h3 id="Viruses">Viruses</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=9" title="Edit section: Viruses">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">Main article: <a href="/wiki/Viral_vector" title="Viral vector">Viral vector</a></div> <figure class="mw-default-size" typeof="mw:File/Thumb"><a href="/wiki/File:Gene_therapy.jpg" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/3/3d/Gene_therapy.jpg/220px-Gene_therapy.jpg" decoding="async" width="220" height="165" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/3/3d/Gene_therapy.jpg/330px-Gene_therapy.jpg 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/3/3d/Gene_therapy.jpg/440px-Gene_therapy.jpg 2x" data-file-width="495" data-file-height="371" /></a><figcaption>Gene therapy using an <a href="/wiki/Adenovirus" class="mw-redirect" title="Adenovirus">adenovirus</a> vector. In some cases, the adenovirus will insert the new gene into a cell. If the treatment is successful, the new gene will make a functional <a href="/wiki/Protein" title="Protein">protein</a> to treat a disease.</figcaption></figure> In order to <a href="/wiki/Viral_replication" title="Viral replication">replicate</a>, <a href="/wiki/Virus" title="Virus">viruses</a> introduce their genetic material into the host cell, tricking the host's cellular machinery into using it as blueprints for viral proteins.<a href="#cite_note-:4-54">[54]</a>: 39  <a href="/wiki/Retrovirus" title="Retrovirus">Retroviruses</a> go a stage further by having their genetic material copied into the nuclear genome of the host cell. Scientists exploit this by substituting part of a virus's genetic material with therapeutic DNA or RNA.<a href="#cite_note-:4-54">[54]</a>: 40 <a href="#cite_note-:2-74">[74]</a> Like the genetic material (DNA or RNA) in viruses, therapeutic genetic material can be designed to simply serve as a temporary blueprint that degrades naturally, as in a non-integrative vectors, or to enter the host's nucleus becoming a permanent part of the host's nuclear DNA in infected cells.<a href="#cite_note-:4-54">[54]</a>: 50  A number of viruses have been used for human gene therapy, including viruses such as <a href="/wiki/Lentivirus" title="Lentivirus">lentivirus</a>, <a href="/wiki/Adenovirus" class="mw-redirect" title="Adenovirus">adenoviruses</a>, <a href="/wiki/Herpes_simplex_virus" title="Herpes simplex virus">herpes simplex</a>, <a href="/wiki/Vaccinia_virus" class="mw-redirect" title="Vaccinia virus">vaccinia</a>, and <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a>.<a href="#cite_note-JGenMed_Database-5">[5]</a> <a href="/wiki/Adenoviridae" title="Adenoviridae">Adenovirus viral vectors</a> (Ad) temporarily modify a cell's genetic expression with genetic material that is not integrated into the host cell's DNA.<a href="#cite_note-:3-75">[75]</a>: 5  As of 2017, such vectors were used in 20% of trials for gene therapy.<a href="#cite_note-:2-74">[74]</a>: 10  Adenovirus vectors are mostly used in cancer treatments and novel genetic vaccines such as the <a href="/wiki/Ebola_vaccine" title="Ebola vaccine">Ebola vaccine</a>, vaccines used in clinical trials for <a href="/wiki/HIV" title="HIV">HIV</a> and <a href="/wiki/SARS-CoV-2" title="SARS-CoV-2">SARS-CoV-2</a>, or <a href="/wiki/Cancer_vaccine" title="Cancer vaccine">cancer vaccines</a>.<a href="#cite_note-:3-75">[75]</a>: 5  <a href="/wiki/Lentiviral_vector_in_gene_therapy" title="Lentiviral vector in gene therapy">Lentiviral vectors</a> based on <a href="/wiki/Lentivirus" title="Lentivirus">lentivirus</a>, a <a href="/wiki/Retrovirus" title="Retrovirus">retrovirus</a>, can modify a cell's nuclear <a href="/wiki/Genome" title="Genome">genome</a> to permanently express a gene, although vectors can be modified to prevent integration.<a href="#cite_note-:4-54">[54]</a>: 40,50  Retroviruses were used in 18% of trials before 2018.<a href="#cite_note-:2-74">[74]</a>: 10  <a href="/wiki/Atidarsagene_autotemcel" title="Atidarsagene autotemcel">Libmeldy</a> is an ex vivo stem cell treatment for <a href="/wiki/Metachromatic_leukodystrophy" title="Metachromatic leukodystrophy">metachromatic leukodystrophy</a> which uses a lentiviral vector and was approved by the European medical agency in 2020.<a href="#cite_note-76">[76]</a> <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">Adeno-associated virus</a> (AAV) is a virus that is incapable of transmission between cells unless the cell is infected by another virus, a helper virus. Adenovirus and the herpes viruses act as helper viruses for AAV. AAV persists within the cell outside of the cell's nuclear genome for an extended period of time through the formation of <a href="/wiki/Concatemer" title="Concatemer">concatemers</a> mostly organized as <a href="/wiki/Episome" title="Episome">episomes</a>.<a href="#cite_note-77">[77]</a>: 4  Genetic material from AAV vectors is integrated into the host cell's nuclear genome at a low frequency and likely mediated by the DNA-modifying enzymes of the host cell.<a href="#cite_note-:7-78">[78]</a>: 2647  Animal models suggest that integration of AAV genetic material into the host cell's nuclear genome may cause <a href="/wiki/Hepatocellular_carcinoma" title="Hepatocellular carcinoma">hepatocellular carcinoma</a>, a form of <a href="/wiki/Liver_cancer" title="Liver cancer">liver cancer</a>.<a href="#cite_note-:7-78">[78]</a> Several AAV investigational agents have been explored in treatment of <a href="/wiki/Wet_age-related_macular_degeneration" class="mw-redirect" title="Wet age-related macular degeneration">wet age related macular degeneration</a> by both <a href="/wiki/Intravitreal_administration" title="Intravitreal administration">intravitreal</a> and subretinal approaches as a potential application of AAV gene therapy for human disease.<a href="#cite_note-79">[79]</a><a href="#cite_note-80">[80]</a> <div class="mw-heading mw-heading3"><h3 id="Non-viral">Non-viral</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=10" title="Edit section: Non-viral">edit</a>]</div> <style data-mw-deduplicate="TemplateStyles:r1251242444">.mw-parser-output .ambox{border:1px solid #a2a9b1;border-left:10px solid #36c;background-color:#fbfbfb;box-sizing:border-box}.mw-parser-output .ambox+link+.ambox,.mw-parser-output .ambox+link+style+.ambox,.mw-parser-output .ambox+link+link+.ambox,.mw-parser-output .ambox+.mw-empty-elt+link+.ambox,.mw-parser-output .ambox+.mw-empty-elt+link+style+.ambox,.mw-parser-output .ambox+.mw-empty-elt+link+link+.ambox{margin-top:-1px}html body.mediawiki .mw-parser-output .ambox.mbox-small-left{margin:4px 1em 4px 0;overflow:hidden;width:238px;border-collapse:collapse;font-size:88%;line-height:1.25em}.mw-parser-output .ambox-speedy{border-left:10px solid #b32424;background-color:#fee7e6}.mw-parser-output .ambox-delete{border-left:10px solid #b32424}.mw-parser-output .ambox-content{border-left:10px solid #f28500}.mw-parser-output .ambox-style{border-left:10px solid #fc3}.mw-parser-output .ambox-move{border-left:10px solid #9932cc}.mw-parser-output .ambox-protection{border-left:10px solid #a2a9b1}.mw-parser-output .ambox .mbox-text{border:none;padding:0.25em 0.5em;width:100%}.mw-parser-output .ambox .mbox-image{border:none;padding:2px 0 2px 0.5em;text-align:center}.mw-parser-output .ambox .mbox-imageright{border:none;padding:2px 0.5em 2px 0;text-align:center}.mw-parser-output .ambox .mbox-empty-cell{border:none;padding:0;width:1px}.mw-parser-output .ambox .mbox-image-div{width:52px}@media(min-width:720px){.mw-parser-output .ambox{margin:0 10%}}@media print{body.ns-0 .mw-parser-output .ambox{display:none!important}}</style><table class="box-More_citations_needed_section plainlinks metadata ambox ambox-content ambox-Refimprove" role="presentation"><tbody><tr><td class="mbox-image"><div class="mbox-image-div"><a href="/wiki/File:Question_book-new.svg" class="mw-file-description"><img alt="" src="//upload.wikimedia.org/wikipedia/en/thumb/9/99/Question_book-new.svg/50px-Question_book-new.svg.png" decoding="async" width="50" height="39" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/9/99/Question_book-new.svg/75px-Question_book-new.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/9/99/Question_book-new.svg/100px-Question_book-new.svg.png 2x" data-file-width="512" data-file-height="399" /></a></div></td><td class="mbox-text"><div class="mbox-text-span">This section needs additional citations for <a href="/wiki/Wikipedia:Verifiability" title="Wikipedia:Verifiability">verification</a>. Please help <a href="/wiki/Special:EditPage/Gene_therapy" title="Special:EditPage/Gene therapy">improve this article</a> by <a href="/wiki/Help:Referencing_for_beginners" title="Help:Referencing for beginners">adding citations to reliable sources</a> in this section. Unsourced material may be challenged and removed. (April 2021) (<a href="/wiki/Help:Maintenance_template_removal" title="Help:Maintenance template removal">Learn how and when to remove this message</a>)</div></td></tr></tbody></table> Non-viral vectors for gene therapy<a href="#cite_note-81">[81]</a> present certain advantages over viral methods, such as large scale production and low host <a href="/wiki/Immunogenicity" title="Immunogenicity">immunogenicity</a>. However, non-viral methods initially produced lower levels of <a href="/wiki/Transfection" title="Transfection">transfection</a> and <a href="/wiki/Gene_expression" title="Gene expression">gene expression</a>, and thus lower therapeutic efficacy. Newer technologies offer promise of solving these problems, with the advent of increased cell-specific targeting and subcellular trafficking control. <a href="/wiki/Vectors_in_Gene_Therapy#Non-viral_methods" class="mw-redirect" title="Vectors in Gene Therapy">Methods for non-viral gene therapy</a> include the injection of naked DNA, <a href="/wiki/Electroporation" title="Electroporation">electroporation</a>, the <a href="/wiki/Gene_gun" title="Gene gun">gene gun</a>, <a href="/wiki/Sonoporation" title="Sonoporation">sonoporation</a>, <a href="/wiki/Magnetofection" title="Magnetofection">magnetofection</a>, the use of <a href="/wiki/Oligonucleotide" title="Oligonucleotide">oligonucleotides</a>, lipoplexes, dendrimers, and inorganic nanoparticles. These therapeutics can be administered directly or through <a href="/wiki/Tissue_engineering" title="Tissue engineering">scaffold enrichment</a>.<a href="#cite_note-82">[82]</a><a href="#cite_note-83">[83]</a> More recent approaches, such as those performed by companies such as <a href="/w/index.php?title=Ligandal&action=edit&redlink=1" class="new" title="Ligandal (page does not exist)">Ligandal</a>, offer the possibility of creating cell-specific targeting technologies for a variety of gene therapy modalities, including RNA, DNA and gene editing tools such as CRISPR. Other companies, such as <a href="/wiki/Arbutus_Biopharma" title="Arbutus Biopharma">Arbutus Biopharma</a> and <a href="/wiki/Arcturus_Therapeutics" title="Arcturus Therapeutics">Arcturus Therapeutics</a>, offer non-viral, non-cell-targeted approaches that mainly exhibit liver trophism. In more recent years, startups such as <a href="/w/index.php?title=Sixfold_Bio&action=edit&redlink=1" class="new" title="Sixfold Bio (page does not exist)">Sixfold Bio</a>, <a href="/w/index.php?title=GenEdit&action=edit&redlink=1" class="new" title="GenEdit (page does not exist)">GenEdit</a>, and <a href="/w/index.php?title=Spotlight_Therapeutics&action=edit&redlink=1" class="new" title="Spotlight Therapeutics (page does not exist)">Spotlight Therapeutics</a> have begun to solve the non-viral gene delivery problem. Non-viral techniques offer the possibility of repeat dosing and greater tailorability of genetic payloads, which in the future will be more likely to take over viral-based delivery systems. Companies such as <a href="/wiki/Editas_Medicine" title="Editas Medicine">Editas Medicine</a>, <a href="/wiki/Intellia_Therapeutics" title="Intellia Therapeutics">Intellia Therapeutics</a>, <a href="/wiki/CRISPR_Therapeutics" title="CRISPR Therapeutics">CRISPR Therapeutics</a>, <a href="/w/index.php?title=Casebia&action=edit&redlink=1" class="new" title="Casebia (page does not exist)">Casebia</a>, <a href="/wiki/Cellectis" title="Cellectis">Cellectis</a>, <a href="/wiki/Precision_Biosciences" class="mw-redirect" title="Precision Biosciences">Precision Biosciences</a>, <a href="/wiki/Bluebird_bio" title="Bluebird bio">bluebird bio</a>, <a href="/wiki/Excision_BioTherapeutics" title="Excision BioTherapeutics">Excision BioTherapeutics</a>, and <a href="/w/index.php?title=Sangamo&action=edit&redlink=1" class="new" title="Sangamo (page does not exist)">Sangamo</a> have developed non-viral gene editing techniques, however frequently still use viruses for delivering gene insertion material following genomic cleavage by guided <a href="/wiki/Nuclease" title="Nuclease">nucleases</a>. These companies focus on gene editing, and still face major delivery hurdles. <a href="/wiki/BioNTech" title="BioNTech">BioNTech</a>, <a href="/wiki/Moderna_Therapeutics" class="mw-redirect" title="Moderna Therapeutics">Moderna Therapeutics</a> and <a href="/wiki/CureVac" title="CureVac">CureVac</a> focus on delivery of <a href="/wiki/MRNA" class="mw-redirect" title="MRNA">mRNA</a> payloads, which are necessarily non-viral delivery problems. <a href="/wiki/Alnylam" class="mw-redirect" title="Alnylam">Alnylam</a>, <a href="/w/index.php?title=Dicerna_Pharmaceuticals&action=edit&redlink=1" class="new" title="Dicerna Pharmaceuticals (page does not exist)">Dicerna Pharmaceuticals</a>, and <a href="/wiki/Ionis_Pharmaceuticals" title="Ionis Pharmaceuticals">Ionis Pharmaceuticals</a> focus on delivery of <a href="/wiki/SiRNA" class="mw-redirect" title="SiRNA">siRNA</a> (antisense oligonucleotides) for gene suppression, which also necessitate non-viral delivery systems. In academic contexts, a number of laboratories are working on delivery of <a href="/wiki/Polyethylene_glycol" title="Polyethylene glycol">PEGylated</a> particles, which form serum protein coronas and chiefly exhibit <a href="/wiki/LDL_receptor" title="LDL receptor">LDL receptor</a> mediated uptake in cells in vivo.<a href="#cite_note-84">[84]</a> <div class="mw-heading mw-heading2"><h2 id="Treatment">Treatment</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=11" title="Edit section: Treatment">edit</a>]</div> <div class="mw-heading mw-heading3"><h3 id="Cancer">Cancer</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=12" title="Edit section: Cancer">edit</a>]</div> <figure class="mw-default-size" typeof="mw:File/Thumb"><a href="/wiki/File:Direct_gene_therapy.png" class="mw-file-description"><img alt="Direct gene therapy" src="//upload.wikimedia.org/wikipedia/commons/thumb/e/ef/Direct_gene_therapy.png/220px-Direct_gene_therapy.png" decoding="async" width="220" height="94" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/e/ef/Direct_gene_therapy.png/330px-Direct_gene_therapy.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/e/ef/Direct_gene_therapy.png/440px-Direct_gene_therapy.png 2x" data-file-width="1174" data-file-height="501" /></a><figcaption>Suicide gene therapy graphic used to treat cancer</figcaption></figure> There have been attempts to treat <a href="/wiki/Cancer" title="Cancer">cancer</a> using gene therapy. As of 2017, 65% of gene therapy trials were for cancer treatment.<a href="#cite_note-:2-74">[74]</a>: 7  Adenovirus vectors are useful for some cancer gene therapies because adenovirus can transiently insert genetic material into a cell without permanently altering the cell's nuclear genome. These vectors can be used to cause <a href="/wiki/Antigen" title="Antigen">antigens</a> to be added to cancers causing an immune response, or hinder <a href="/wiki/Angiogenesis" title="Angiogenesis">angiogenesis</a> by expressing certain proteins.<a href="#cite_note-:5-85">[85]</a>: 5  An Adenovirus vector is used in the commercial products <a href="/wiki/Gendicine" title="Gendicine">Gendicine</a> and <a href="/wiki/Oncorine" class="mw-redirect" title="Oncorine">Oncorine</a>.<a href="#cite_note-:5-85">[85]</a>: 10  Another commercial product, <a href="/wiki/Rexin_G" title="Rexin G">Rexin G</a>, uses a retrovirus-based vector and selectively binds to receptors that are more expressed in tumors.<a href="#cite_note-:5-85">[85]</a>: 10  One approach, <a href="/wiki/Suicide_gene" title="Suicide gene">suicide gene therapy</a>, works by introducing genes encoding enzymes that will cause a cancer cell to die. Another approach is the use <a href="/wiki/Oncolytic_virus" title="Oncolytic virus">oncolytic viruses</a>, such as Oncorine,<a href="#cite_note-86">[86]</a>: 165  which are viruses that selectively reproduce in cancerous cells leaving other cells unaffected.<a href="#cite_note-87">[87]</a>: 6 <a href="#cite_note-88">[88]</a>: 280  <a href="/wiki/MRNA_vaccine" title="MRNA vaccine">mRNA</a> has been suggested as a non-viral vector for cancer gene therapy that would temporarily change a cancerous cell's function to create antigens or kill the cancerous cells and there have been several trials.<a href="#cite_note-89">[89]</a> <a href="/wiki/Afamitresgene_autoleucel" title="Afamitresgene autoleucel">Afamitresgene autoleucel</a>, sold under the brand name Tecelra, is an autologous T cell immunotherapy used for the treatment of <a href="/wiki/Synovial_sarcoma" title="Synovial sarcoma">synovial sarcoma</a>. It is a T cell receptor (TCR) gene therapy.<a href="#cite_note-FDA_20240802-90">[90]</a> It is the first FDA-approved engineered cell therapy for a solid tumor.<a href="#cite_note-91">[91]</a> It uses a self-inactivating lentiviral vector to express a T-cell receptor specific for MAGE-A4, a melanoma-associated antigen.[<a href="/wiki/Wikipedia:Identifying_reliable_sources_(medicine)" title="Wikipedia:Identifying reliable sources (medicine)">medical citation needed</a>] <div class="mw-heading mw-heading3"><h3 id="Genetic_diseases">Genetic diseases</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=13" title="Edit section: Genetic diseases">edit</a>]</div> Gene therapy approaches to replace a faulty gene with a healthy gene have been proposed and are being studied for treating some genetic diseases. As of 2017, 11.1% of gene therapy clinical trials targeted monogenic diseases.<a href="#cite_note-:2-74">[74]</a>: 9  Diseases such as <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a> that are caused by <a href="/wiki/Autosomal_recessive_disorder" class="mw-redirect" title="Autosomal recessive disorder">autosomal recessive disorders</a> for which a person's normal <a href="/wiki/Phenotype" title="Phenotype">phenotype</a> or cell function may be restored in cells that have the disease by a normal copy of the gene that is mutated, may be a good candidate for gene therapy treatment.<a href="#cite_note-pmid_28696921-92">[92]</a><a href="#cite_note-:0-93">[93]</a> The risks and benefits related to gene therapy for sickle cell disease are not known.<a href="#cite_note-:0-93">[93]</a> <a href="/wiki/Gene_therapy_of_the_human_retina" title="Gene therapy of the human retina">Gene therapy has been used in the eye</a>. The eye is especially suitable for <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a> vectors. <a href="/wiki/Voretigene_neparvovec" title="Voretigene neparvovec">Voretigene neparvovec</a> is an approved gene therapy to treat <a href="/wiki/Leber%27s_hereditary_optic_neuropathy" title="Leber's hereditary optic neuropathy">Leber's hereditary optic neuropathy</a>.<a href="#cite_note-:6-94">[94]</a>: 1354  <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a>, a treatment for <a href="/wiki/Pancreatitis" title="Pancreatitis">pancreatitis</a> caused by a genetic condition, and <a href="/wiki/Zolgensma" class="mw-redirect" title="Zolgensma">Zolgensma</a> for the treatment of <a href="/wiki/Spinal_muscular_atrophy" title="Spinal muscular atrophy">spinal muscular atrophy</a> both use an adeno-associated virus vector.<a href="#cite_note-:7-78">[78]</a>: 2647  <div class="mw-heading mw-heading3"><h3 id="Infectious_diseases">Infectious diseases</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=14" title="Edit section: Infectious diseases">edit</a>]</div> As of 2017, 7% of genetic therapy trials targeted infectious diseases. 69.2% of trials targeted <a href="/wiki/HIV" title="HIV">HIV</a>, 11% <a href="/wiki/Hepatitis" title="Hepatitis">hepatitis</a> B or C, and 7.1% <a href="/wiki/Malaria" title="Malaria">malaria</a>.<a href="#cite_note-:2-74">[74]</a> <div class="mw-heading mw-heading3"><h3 id="List_of_gene_therapies_for_treatment_of_disease">List of gene therapies for treatment of disease</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=15" title="Edit section: List of gene therapies for treatment of disease">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">See also: <a href="/wiki/List_of_gene_therapies" title="List of gene therapies">List of gene therapies</a></div> Some genetic therapies have been approved by the U.S. <a href="/wiki/Food_and_Drug_Administration" title="Food and Drug Administration">Food and Drug Administration</a> (FDA), the <a href="/wiki/European_Medicines_Agency" title="European Medicines Agency">European Medicines Agency</a> (EMA), and for use in Russia and China. <table class="sortable wikitable"> <caption>List of approved gene therapies for the treatment of disease </caption> <tbody><tr> <th>INN </th> <th>Brand name </th> <th>Type </th> <th>Manufacturer </th> <th>Target </th> <th>US <a href="/wiki/Food_and_Drug_Administration" title="Food and Drug Administration">Food and Drug Administration</a> (FDA) approved </th> <th><a href="/wiki/European_Medicines_Agency" title="European Medicines Agency">European Medicines Agency</a> (EMA) authorized </th></tr> <tr> <td><a href="/wiki/Afamitresgene_autoleucel" title="Afamitresgene autoleucel">afamitresgene autoleucel</a> </td> <td>Tecelra </td> <td>Ex vitro </td> <td><a href="/wiki/Adaptimmune" title="Adaptimmune">Adaptimmune</a> </td> <td><a href="/wiki/Synovial_sarcoma" title="Synovial sarcoma">synovial sarcoma</a> </td> <td>August 2024<a href="#cite_note-FDA_20240802-90">[90]</a> </td></tr> <tr> <td><a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a> </td> <td>Glybera </td> <td>In vivo </td> <td><a href="/wiki/Chiesi_Farmaceutici" title="Chiesi Farmaceutici">Chiesi Farmaceutici</a> </td> <td><a href="/wiki/Lipoprotein_lipase_deficiency" title="Lipoprotein lipase deficiency">lipoprotein lipase deficiency</a> </td> <td> </td> <td>Withdrawn </td></tr> <tr> <td><a href="/wiki/Atidarsagene_autotemcel" title="Atidarsagene autotemcel">atidarsagene autotemcel</a> </td> <td>Libmeldy, Lenmeldy (<a href="/wiki/Arylsulfatase_A" title="Arylsulfatase A">Arylsulfatase A</a> gene encoding autologous <a href="/wiki/CD34" title="CD34">CD34+</a> cells) </td> <td>Ex vitro </td> <td><a href="/w/index.php?title=Orchard_Therapeutics&action=edit&redlink=1" class="new" title="Orchard Therapeutics (page does not exist)">Orchard Therapeutics</a> </td> <td><a href="/wiki/Metachromatic_leukodystrophy" title="Metachromatic leukodystrophy">metachromatic leukodystrophy</a> </td> <td>March 2024<a href="#cite_note-95">[95]</a> </td> <td>December 2020<a href="#cite_note-96">[96]</a> </td></tr> <tr> <td><a href="/wiki/Strimvelis" title="Strimvelis">autologous CD34+</a> </td> <td>Strimvelis </td> <td> </td> <td> </td> <td>adenosine deaminase deficiency (ADA-SCID) </td> <td> </td> <td>May 2016 </td></tr> <tr> <td><a href="/wiki/Axicabtagene_ciloleucel" title="Axicabtagene ciloleucel">axicabtagene ciloleucel</a> </td> <td>Yescarta </td> <td>Ex vitro </td> <td><a href="/wiki/Kite_Pharma" title="Kite Pharma">Kite pharma</a> </td> <td><a href="/wiki/Large-cell_lymphoma" title="Large-cell lymphoma">large B-cell lymphoma</a> </td> <td>October 2017 </td> <td>August 2018 </td></tr> <tr> <td><a href="/wiki/Beremagene_geperpavec" title="Beremagene geperpavec">beremagene geperpavec</a> </td> <td>Vyjuvek </td> <td>In vivo </td> <td>Krystal Biotech </td> <td><a href="/wiki/Dystrophic_epidermolysis_bullosa" class="mw-redirect" title="Dystrophic epidermolysis bullosa">dystrophic epidermolysis bullosa</a> (DEB) </td> <td>May 2023<a href="#cite_note-:9-97">[97]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Betibeglogene_autotemcel" title="Betibeglogene autotemcel">betibeglogene autotemcel</a> </td> <td>Zynteglo </td> <td> </td> <td> </td> <td>beta thalassemia </td> <td>August 2022<a href="#cite_note-98">[98]</a> </td> <td>May 2019 </td></tr> <tr> <td><a href="/wiki/Brexucabtagene_autoleucel" title="Brexucabtagene autoleucel">brexucabtagene autoleucel</a> </td> <td>Tecartus </td> <td>Ex vitro </td> <td><a href="/wiki/Kite_Pharma" title="Kite Pharma">Kite Pharma</a> </td> <td><a href="/wiki/Mantle_cell_lymphoma" title="Mantle cell lymphoma">mantle cell lymphoma</a> and <a href="/wiki/Acute_lymphoblastic_leukemia" title="Acute lymphoblastic leukemia">acute lymphoblastic leukemia</a> </td> <td>July 2020<a href="#cite_note-FDA_PR-99">[99]</a><a href="#cite_note-FDA_20210101-100">[100]</a> </td> <td>December 2020<a href="#cite_note-101">[101]</a> </td></tr> <tr> <td><a href="/wiki/Cambiogenplasmid" title="Cambiogenplasmid">cambiogenplasmid</a> </td> <td>Neovasculgen </td> <td> </td> <td> </td> <td>vascular endothelial growth factor peripheral artery disease </td> <td> </td> <td> </td></tr> <tr> <td><a href="/wiki/Delandistrogene_moxeparvovec" title="Delandistrogene moxeparvovec">delandistrogene moxeparvovec</a> </td> <td>Elevidys </td> <td>In vivo </td> <td><a href="/wiki/Catalent" title="Catalent">Catalent</a> </td> <td><a href="/wiki/Duchenne_muscular_dystrophy" title="Duchenne muscular dystrophy">Duchenne muscular dystrophy</a> </td> <td>June 2023<a href="#cite_note-auto-102">[102]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Eladocagene_exuparvovec" title="Eladocagene exuparvovec">eladocagene exuparvovec</a> </td> <td>Kebilidi, Upstaza </td> <td>In vivo </td> <td><a href="/wiki/PTC_Therapeutics" title="PTC Therapeutics">PTC Therapeutics</a> </td> <td><a href="/wiki/Aromatic_L-amino_acid_decarboxylase_deficiency" title="Aromatic L-amino acid decarboxylase deficiency">aromatic L‑amino acid decarboxylase (AADC) deficiency</a> </td> <td>November 2024<a href="#cite_note-FDA_PR_20241114-103">[103]</a> </td> <td>July 2022<a href="#cite_note-Upstaza_EPAR-104">[104]</a> </td></tr> <tr> <td><a href="/wiki/Elivaldogene_autotemcel" title="Elivaldogene autotemcel">elivaldogene autotemcel</a> </td> <td>Skysona </td> <td> </td> <td> </td> <td>cerebral adrenoleukodystrophy </td> <td> </td> <td>July 2021 </td></tr> <tr> <td><a href="/wiki/Exagamglogene_autotemcel" title="Exagamglogene autotemcel">exagamglogene autotemcel</a> </td> <td>Casgevy </td> <td>Ex vivo </td> <td><a href="/wiki/Vertex_Pharmaceuticals" title="Vertex Pharmaceuticals">Vertex Pharmaceuticals</a> </td> <td><a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a> </td> <td>December 2023<a href="#cite_note-FDA_Casgevy-105">[105]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Gendicine" title="Gendicine">gendicine</a> </td> <td> </td> <td> </td> <td> </td> <td>head and neck squamous cell carcinoma </td> <td> </td> <td> </td></tr> <tr> <td><a href="/wiki/Idecabtagene_vicleucel" title="Idecabtagene vicleucel">idecabtagene vicleucel</a> </td> <td>Abecma </td> <td>Ex vivo </td> <td><a href="/wiki/Celgene" title="Celgene">Celgene</a> </td> <td><a href="/wiki/Multiple_myeloma" title="Multiple myeloma">multiple myeloma</a> </td> <td>March 2021<a href="#cite_note-106">[106]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Lisocabtagene_maraleucel" title="Lisocabtagene maraleucel">lisocabtagene maraleucel</a> </td> <td>Breyanzi </td> <td>Ex vivo </td> <td><a href="/wiki/Juno_Therapeutics" title="Juno Therapeutics">Juno Therapeutics</a> </td> <td><a href="/wiki/B-cell_lymphoma" title="B-cell lymphoma">B-cell lymphoma</a> </td> <td>February 2021<a href="#cite_note-107">[107]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Lovotibeglogene_autotemcel" title="Lovotibeglogene autotemcel">lovotibeglogene autotemcel</a> </td> <td>Lyfgenia </td> <td>Ex vivo </td> <td><a href="/wiki/Bluebird_Bio" class="mw-redirect" title="Bluebird Bio">Bluebird Bio</a> </td> <td><a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a> </td> <td>December 2023<a href="#cite_note-FDA_Lyfgenia-108">[108]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Nadofaragene_firadenovec" title="Nadofaragene firadenovec">nadofaragene firadenovec</a> </td> <td>Adstiladrin </td> <td> </td> <td><a href="/wiki/Ferring_Pharmaceuticals" title="Ferring Pharmaceuticals">Ferring Pharmaceuticals</a> </td> <td>high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) </td> <td>Yes<a href="#cite_note-109">[109]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Obecabtagene_autoleucel" title="Obecabtagene autoleucel">obecabtagene autoleucel</a> </td> <td>Aucatzyl </td> <td> </td> <td><a href="/w/index.php?title=Autolus_Therapeutics&action=edit&redlink=1" class="new" title="Autolus Therapeutics (page does not exist)">Autolus Therapeutics</a> </td> <td><a href="/wiki/Acute_lymphoblastic_leukemia" title="Acute lymphoblastic leukemia">acute lymphoblastic leukemia</a> </td> <td>November 2024<a href="#cite_note-FDA_Aucatzyl-110">[110]</a><a href="#cite_note-111">[111]</a> </td> <td> </td></tr> <tr> <td><a href="/wiki/Onasemnogene_abeparvovec" title="Onasemnogene abeparvovec">onasemnogene abeparvovec</a> </td> <td>Zolgensma </td> <td>In vivo </td> <td><a href="/wiki/Novartis_Gene_Therapies" title="Novartis Gene Therapies">Novartis Gene Therapies</a> </td> <td><a href="/wiki/Spinal_muscular_atrophy" title="Spinal muscular atrophy">spinal muscular atrophy</a> type I </td> <td>May 2019<a href="#cite_note-112">[112]</a> </td> <td>March 2020<a href="#cite_note-113">[113]</a> </td></tr> <tr> <td><a href="/wiki/Talimogene_laherparepvec" title="Talimogene laherparepvec">talimogene laherparepvec</a> </td> <td>Imlygic </td> <td>In vivo </td> <td><a href="/wiki/Amgen" title="Amgen">Amgen</a> </td> <td><a href="/wiki/Melanoma" title="Melanoma">melanoma</a> </td> <td>October 2015<a href="#cite_note-114">[114]</a> </td> <td>December 2015<a href="#cite_note-115">[115]</a> </td></tr> <tr> <td><a href="/wiki/Tisagenlecleucel" title="Tisagenlecleucel">tisagenlecleucel</a> </td> <td>Kymriah </td> <td> </td> <td> </td> <td>B cell lymphoblastic leukemia </td> <td> </td> <td>August 2018 </td></tr> <tr> <td><a href="/wiki/Valoctocogene_roxaparvovec" title="Valoctocogene roxaparvovec">valoctocogene roxaparvovec</a> </td> <td>Roctavian </td> <td> </td> <td><a href="/wiki/BioMarin_Pharmaceutical" title="BioMarin Pharmaceutical">BioMarin International Limited</a> </td> <td><a href="/wiki/Hemophilia_A" class="mw-redirect" title="Hemophilia A">hemophilia A</a> </td> <td> </td> <td>August 2022<a href="#cite_note-116">[116]</a><a href="#cite_note-Roctavian:_Pending_EC_decision-117">[117]</a><a href="#cite_note-Roctavian_auth-118">[118]</a> </td></tr> <tr> <td><a href="/wiki/Voretigene_neparvovec" title="Voretigene neparvovec">voretigene neparvovec</a> </td> <td>Luxturna </td> <td>In vivo </td> <td><a href="/wiki/Spark_Therapeutics" title="Spark Therapeutics">Spark Therapeutics</a> </td> <td>biallelic <a href="/wiki/RPE65" title="RPE65">RPE65</a> mutation associated <a href="/wiki/Leber_congenital_amaurosis" title="Leber congenital amaurosis">Leber congenital amaurosis</a> </td> <td>December 2017<a href="#cite_note-119">[119]</a> </td> <td>November 2018<a href="#cite_note-120">[120]</a> </td></tr></tbody></table> <div class="mw-heading mw-heading2"><h2 id="Adverse_effects,_contraindications_and_hurdles_for_use">Adverse effects, contraindications and hurdles for use</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=16" title="Edit section: Adverse effects, contraindications and hurdles for use">edit</a>]</div> Some of the unsolved problems include: <ul><li>Off-target effects – The possibility of unwanted, likely harmful, changes to the genome present a large barrier to the widespread implementation of this technology.<a href="#cite_note-121">[121]</a> Improvements to the specificity of gRNAs and Cas enzymes present viable solutions to this issue as well as the refinement of the delivery method of CRISPR.<a href="#cite_note-122">[122]</a> It is likely that different diseases will benefit from different delivery methods.</li> <li>Short-lived nature – Before gene therapy can become a permanent cure for a condition, the therapeutic DNA introduced into target cells must remain functional and the cells containing the therapeutic DNA must be stable. Problems with integrating therapeutic DNA into the nuclear <a href="/wiki/Genome" title="Genome">genome</a> and the rapidly dividing nature of many cells prevent it from achieving long-term benefits. Patients require multiple treatments.</li> <li>Immune response – Any time a foreign object is introduced into human tissues, the immune system is stimulated to attack the invader. Stimulating the immune system in a way that reduces gene therapy effectiveness is possible. The <a href="/wiki/Immune_system" title="Immune system">immune system</a>'s enhanced response to viruses that it has seen before reduces the effectiveness to repeated treatments.</li> <li>Problems with viral vectors – Viral vectors carry the risks of toxicity, inflammatory responses, and gene control and targeting issues.</li> <li>Multigene disorders – Some commonly occurring disorders, such as <a href="/wiki/Heart_disease" class="mw-redirect" title="Heart disease">heart disease</a>, <a href="/wiki/High_blood_pressure" class="mw-redirect" title="High blood pressure">high blood pressure</a>, <a href="/wiki/Alzheimer%27s_disease" title="Alzheimer's disease">Alzheimer's disease</a>, <a href="/wiki/Arthritis" title="Arthritis">arthritis</a>, and <a href="/wiki/Diabetes" title="Diabetes">diabetes</a>, are affected by variations in multiple genes, which complicate gene therapy.</li> <li>Some therapies may breach the <a href="/wiki/Weismann_barrier" title="Weismann barrier">Weismann barrier</a> (between soma and germ-line) protecting the testes, potentially modifying the germline, falling afoul of regulations in countries that prohibit the latter practice.<a href="#cite_note-123">[123]</a></li> <li>Insertional <a href="/wiki/Mutagenesis" title="Mutagenesis">mutagenesis</a> – If the DNA is integrated in a sensitive spot in the genome, for example in a <a href="/wiki/Tumor_suppressor_gene" title="Tumor suppressor gene">tumor suppressor gene</a>, the therapy could induce a <a href="/wiki/Tumor" class="mw-redirect" title="Tumor">tumor</a>. This has occurred in clinical trials for <a href="/wiki/X-linked_severe_combined_immunodeficiency" title="X-linked severe combined immunodeficiency">X-linked severe combined immunodeficiency</a> (X-SCID) patients, in which <a href="/wiki/Hematopoietic" class="mw-redirect" title="Hematopoietic">hematopoietic</a> stem cells were transduced with a corrective transgene using a <a href="/wiki/Retrovirus" title="Retrovirus">retrovirus</a>, and this led to the development of <a href="/wiki/T_cell_leukemia" class="mw-redirect" title="T cell leukemia">T cell leukemia</a> in 3 of 20 patients.<a href="#cite_note-124">[124]</a><a href="#cite_note-125">[125]</a> One possible solution is to add a functional tumor suppressor gene to the DNA to be integrated. This may be problematic since the longer the DNA is, the harder it is to integrate into cell genomes.<a href="#cite_note-126">[126]</a> <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a> technology allows researchers to make much more precise genome changes at exact locations.<a href="#cite_note-MIT-127">[127]</a></li> <li>Cost – <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">alipogene tiparvovec</a> (Glybera), for example, at a cost of $1.6 million per patient, was reported in 2013, to be the world's most expensive drug.<a href="#cite_note-128">[128]</a><a href="#cite_note-129">[129]</a></li></ul> <div class="mw-heading mw-heading3"><h3 id="Deaths">Deaths</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=17" title="Edit section: Deaths">edit</a>]</div> Three patients' deaths have been reported in gene therapy trials, putting the field under close scrutiny. The first was that of <a href="/wiki/Jesse_Gelsinger" title="Jesse Gelsinger">Jesse Gelsinger</a>, who died in 1999, because of immune rejection response.<a href="#cite_note-130">[130]</a><a href="#cite_note-131">[131]</a> One X-SCID patient died of leukemia in 2003.<a href="#cite_note-Gene_Therapy_Finds_Its_Niche-13">[13]</a> In 2007, a <a href="/wiki/Rheumatoid_arthritis" title="Rheumatoid arthritis">rheumatoid arthritis</a> patient died from an infection; the subsequent investigation concluded that the death was not related to gene therapy.<a href="#cite_note-132">[132]</a> <div class="mw-heading mw-heading2"><h2 id="Regulations">Regulations</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=18" title="Edit section: Regulations">edit</a>]</div> Regulations covering genetic modification are part of general guidelines about human-involved biomedical research.[<a href="/wiki/Wikipedia:Citation_needed" title="Wikipedia:Citation needed">citation needed</a>] There are no international treaties which are legally binding in this area, but there are recommendations for national laws from various bodies.[<a href="/wiki/Wikipedia:Citation_needed" title="Wikipedia:Citation needed">citation needed</a>] The <a href="/wiki/Declaration_of_Helsinki" title="Declaration of Helsinki">Helsinki Declaration</a> (Ethical Principles for Medical Research Involving Human Subjects) was amended by the <a href="/wiki/World_Medical_Association" title="World Medical Association">World Medical Association</a>'s General Assembly in 2008. This document provides principles physicians and researchers must consider when involving humans as research subjects. The Statement on Gene Therapy Research initiated by the <a href="/wiki/Human_Genome_Organization" class="mw-redirect" title="Human Genome Organization">Human Genome Organization</a> (HUGO) in 2001, provides a legal baseline for all countries. HUGO's document emphasizes human freedom and adherence to human rights, and offers recommendations for somatic gene therapy, including the importance of recognizing public concerns about such research.<a href="#cite_note-human_genome-133">[133]</a> <div class="mw-heading mw-heading3"><h3 id="United_States">United States</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=19" title="Edit section: United States">edit</a>]</div> No federal legislation lays out protocols or restrictions about human genetic engineering. This subject is governed by overlapping regulations from local and federal agencies, including the <a href="/wiki/Department_of_Health_and_Human_Services" class="mw-redirect" title="Department of Health and Human Services">Department of Health and Human Services</a>, the FDA and NIH's Recombinant DNA Advisory Committee. Researchers seeking federal funds for an investigational new drug application, (commonly the case for somatic human genetic engineering,) must obey international and federal guidelines for the protection of human subjects.<a href="#cite_note-regulatory-134">[134]</a> NIH serves as the main gene therapy regulator for federally funded research. Privately funded research is advised to follow these regulations. NIH provides funding for research that develops or enhances genetic engineering techniques and to evaluate the ethics and quality in current research. The NIH maintains a mandatory registry of human genetic engineering research protocols that includes all federally funded projects.<a href="#cite_note-135">[135]</a> An NIH advisory committee published a set of guidelines on gene manipulation.<a href="#cite_note-national-136">[136]</a> The guidelines discuss lab safety as well as human test subjects and various experimental types that involve genetic changes. Several sections specifically pertain to human genetic engineering, including Section III-C-1. This section describes required review processes and other aspects when seeking approval to begin clinical research involving genetic transfer into a human patient.<a href="#cite_note-us-137">[137]</a> The protocol for a gene therapy clinical trial must be approved by the NIH's Recombinant DNA Advisory Committee prior to any clinical trial beginning; this is different from any other kind of clinical trial.<a href="#cite_note-national-136">[136]</a> As with other kinds of drugs, the FDA regulates the quality and safety of gene therapy products and supervises how these products are used clinically. Therapeutic alteration of the human genome falls under the same regulatory requirements as any other medical treatment. Research involving human subjects, such as <a href="/wiki/Clinical_trials" class="mw-redirect" title="Clinical trials">clinical trials</a>, must be reviewed and approved by the FDA and an <a href="/wiki/Institutional_Review_Board" class="mw-redirect" title="Institutional Review Board">Institutional Review Board</a>.<a href="#cite_note-fda2-138">[138]</a><a href="#cite_note-health-139">[139]</a> <div class="mw-heading mw-heading2"><h2 id="Gene_doping">Gene doping</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=20" title="Edit section: Gene doping">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">Main article: <a href="/wiki/Gene_doping" title="Gene doping">Gene doping</a></div> Athletes may adopt gene therapy technologies to improve their performance.<a href="#cite_note-Gene_Doping-140">[140]</a> <a href="/wiki/Gene_doping" title="Gene doping">Gene doping</a> is not known to occur, but multiple gene therapies may have such effects. Kayser et al. argue that gene doping could <a href="/wiki/Level_playing_field" title="Level playing field">level the playing field</a> if all athletes receive equal access. Critics claim that any therapeutic intervention for non-therapeutic/enhancement purposes compromises the ethical foundations of medicine and sports.<a href="#cite_note-141">[141]</a> <div class="mw-heading mw-heading2"><h2 id="Genetic_enhancement">Genetic enhancement</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=21" title="Edit section: Genetic enhancement">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">Further information: <a href="/wiki/Human_genetic_enhancement" title="Human genetic enhancement">Human genetic enhancement</a></div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236090951"><div role="note" class="hatnote navigation-not-searchable">See also: <a href="/wiki/Human_germline_engineering" title="Human germline engineering">Human germline engineering</a> and <a href="/wiki/Assisted_reproductive_technology" title="Assisted reproductive technology">Assisted reproductive technology</a></div> Genetic engineering could be used to cure diseases, but also to change physical appearance, <a href="/wiki/Metabolism" title="Metabolism">metabolism</a>, and even <a href="/wiki/Human_enhancement" title="Human enhancement">improve</a> physical capabilities and mental faculties such as <a href="/wiki/Memory" title="Memory">memory</a> and <a href="/wiki/Intelligence" title="Intelligence">intelligence</a>. Ethical claims about germline engineering include beliefs that every <a href="/wiki/Fetus" title="Fetus">fetus</a> has a right to remain genetically unmodified, that parents hold the right to genetically modify their offspring, and that every child has the right to be born free of preventable diseases.<a href="#cite_note-Evolution-142">[142]</a><a href="#cite_note-Baylis,_Francoise_2004-143">[143]</a><a href="#cite_note-144">[144]</a> For parents, genetic engineering could be seen as another child enhancement technique to add to diet, exercise, education, training, cosmetics, and plastic surgery.<a href="#cite_note-Enhancement-145">[145]</a><a href="#cite_note-Roco_Bainbridge_2002-146">[146]</a> Another theorist claims that moral concerns limit but do not prohibit germline engineering.<a href="#cite_note-147">[147]</a> A 2020 issue of the journal Bioethics was devoted to moral issues surrounding germline genetic engineering in people.<a href="#cite_note-148">[148]</a> Possible regulatory schemes include a complete ban, provision to everyone, or professional self-regulation. The <a href="/wiki/American_Medical_Association" title="American Medical Association">American Medical Association</a>'s Council on Ethical and Judicial Affairs stated that "genetic interventions to enhance traits should be considered permissible only in severely restricted situations: (1) clear and meaningful benefits to the fetus or child; (2) no trade-off with other characteristics or traits; and (3) equal access to the genetic technology, irrespective of income or other socioeconomic characteristics."<a href="#cite_note-149">[149]</a> As early in the history of <a href="/wiki/Biotechnology" title="Biotechnology">biotechnology</a> as 1990, there have been scientists opposed to attempts to modify the human <a href="/wiki/Germline" title="Germline">germline</a> using these new tools,<a href="#cite_note-150">[150]</a> and such concerns have continued as technology progressed.<a href="#cite_note-151">[151]</a><a href="#cite_note-152">[152]</a> With the advent of new techniques like <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a>, in March 2015 a group of scientists urged a worldwide moratorium on clinical use of gene editing technologies to edit the <a href="/wiki/Human_genome" title="Human genome">human genome</a> in a way that can be inherited.<a href="#cite_note-NYT-20150319-153">[153]</a><a href="#cite_note-NYT-20150303-AP-154">[154]</a><a href="#cite_note-SCI-20150319-155">[155]</a><a href="#cite_note-NAT-20150312-156">[156]</a> In April 2015, researchers sparked controversy when they <a href="/wiki/CRISPR#Society_and_culture" title="CRISPR">reported</a> results of <a href="/wiki/Basic_research" title="Basic research">basic research</a> to edit the <a href="/wiki/DNA" title="DNA">DNA</a> of non-viable <a href="/wiki/Human_embryos" class="mw-redirect" title="Human embryos">human embryos</a> using CRISPR.<a href="#cite_note-PC-20150418-157">[157]</a><a href="#cite_note-NYT-20150423-158">[158]</a> A committee of the American <a href="/wiki/National_Academy_of_Sciences" title="National Academy of Sciences">National Academy of Sciences</a> and <a href="/wiki/National_Academy_of_Medicine" title="National Academy of Medicine">National Academy of Medicine</a> gave qualified support to human genome editing in 2017<a href="#cite_note-159">[159]</a><a href="#cite_note-160">[160]</a> once answers have been found to safety and efficiency problems "but only for serious conditions under stringent oversight."<a href="#cite_note-161">[161]</a> <div class="mw-heading mw-heading2"><h2 id="History">History</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=22" title="Edit section: History">edit</a>]</div> <link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1251242444"><table class="box-Summarize_section plainlinks metadata ambox ambox-style" role="presentation"><tbody><tr><td class="mbox-image"><div class="mbox-image-div"><img alt="" src="//upload.wikimedia.org/wikipedia/en/thumb/f/f2/Edit-clear.svg/40px-Edit-clear.svg.png" decoding="async" width="40" height="40" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/f/f2/Edit-clear.svg/60px-Edit-clear.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/f/f2/Edit-clear.svg/80px-Edit-clear.svg.png 2x" data-file-width="48" data-file-height="48" /></div></td><td class="mbox-text"><div class="mbox-text-span">This section may be <a href="/wiki/Help:Section#Section_size_policies" title="Help:Section">too long</a> and excessively detailed. Please consider summarizing the material. (November 2018)</div></td></tr></tbody></table> <div class="mw-heading mw-heading3"><h3 id="1970s_and_earlier">1970s and earlier</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=23" title="Edit section: 1970s and earlier">edit</a>]</div> In 1972, Friedmann and Roblin authored a paper in <a href="/wiki/Science_(journal)" title="Science (journal)">Science</a> titled "Gene therapy for human genetic disease?".<a href="#cite_note-Friedman_1972-162">[162]</a> Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those with genetic defects.<a href="#cite_note-163">[163]</a> <div class="mw-heading mw-heading3"><h3 id="1980s">1980s</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=24" title="Edit section: 1980s">edit</a>]</div> In 1984, a retrovirus vector system was designed that could efficiently insert foreign genes into mammalian chromosomes.<a href="#cite_note-164">[164]</a> <div class="mw-heading mw-heading3"><h3 id="1990s">1990s</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=25" title="Edit section: 1990s">edit</a>]</div> The first approved gene therapy clinical research in the US took place on 14 September 1990, at the <a href="/wiki/National_Institutes_of_Health" title="National Institutes of Health">National Institutes of Health</a> (NIH), under the direction of <a href="/wiki/William_French_Anderson" title="William French Anderson">William French Anderson</a>.<a href="#cite_note-165">[165]</a> Four-year-old Ashanti DeSilva received treatment for a genetic defect that left her with <a href="/wiki/Adenosine_deaminase_deficiency" title="Adenosine deaminase deficiency">adenosine deaminase deficiency</a> (ADA-SCID), a severe immune system deficiency. The defective gene of the patient's blood cells was replaced by the functional variant. Ashanti's immune system was partially restored by the therapy. Production of the missing enzyme was temporarily stimulated, but the new cells with functional genes were not generated. She led a normal life only with the regular injections performed every two months. The effects were successful, but temporary.<a href="#cite_note-166">[166]</a> Cancer gene therapy was introduced in 1992/93 (Trojan et al. 1993).<a href="#cite_note-167">[167]</a> The treatment of <a href="/wiki/Glioblastoma" title="Glioblastoma">glioblastoma</a> multiforme, the malignant brain tumor whose outcome is always fatal, was done using a vector expressing antisense IGF-I RNA (clinical trial approved by NIH protocol no.1602 24 November 1993,<a href="#cite_note-pmid22400112-168">[168]</a> and by the FDA in 1994). This therapy also represents the beginning of cancer immunogene therapy, a treatment which proves to be effective due to the anti-tumor mechanism of IGF-I antisense, which is related to strong immune and apoptotic phenomena. In 1992, <a href="/wiki/Claudio_Bordignon" title="Claudio Bordignon">Claudio Bordignon</a>, working at the <a href="/wiki/Vita-Salute_San_Raffaele_University" title="Vita-Salute San Raffaele University">Vita-Salute San Raffaele University</a>, performed the first gene therapy procedure using <a href="/wiki/Hematopoietic_stem_cell" title="Hematopoietic stem cell">hematopoietic stem cells</a> as vectors to deliver genes intended to correct <a href="/wiki/Hereditary_diseases" class="mw-redirect" title="Hereditary diseases">hereditary diseases</a>.<a href="#cite_note-169">[169]</a> In 2002, this work led to the publication of the first successful gene therapy treatment for ADA-SCID. The success of a multi-center trial for treating children with SCID (<a href="/wiki/Severe_combined_immune_deficiency" class="mw-redirect" title="Severe combined immune deficiency">severe combined immune deficiency</a> or "bubble boy" disease) from 2000 and 2002, was questioned when two of the ten children treated at the trial's Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the US, the United Kingdom, France, Italy, and Germany.<a href="#cite_note-170">[170]</a> In 1993, Andrew Gobea was born with SCID following prenatal <a href="/wiki/Genetic_screening" class="mw-redirect" title="Genetic screening">genetic screening</a>. Blood was removed from his mother's <a href="/wiki/Placenta" title="Placenta">placenta</a> and <a href="/wiki/Umbilical_cord" title="Umbilical cord">umbilical cord</a> immediately after birth, to acquire stem cells. The <a href="/wiki/Allele" title="Allele">allele</a> that codes for <a href="/wiki/Adenosine_deaminase" title="Adenosine deaminase">adenosine deaminase</a> (ADA) was obtained and inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses inserted the gene into the stem cell chromosomes. Stem cells containing the working ADA gene were injected into Andrew's blood. Injections of the ADA enzyme were also given weekly. For four years <a href="/wiki/T_cell" title="T cell">T cells</a> (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed.<a href="#cite_note-171">[171]</a> In 1996, <a href="/wiki/Luigi_Naldini" title="Luigi Naldini">Luigi Naldini</a> and <a href="/wiki/Didier_Trono" title="Didier Trono">Didier Trono</a> developed a new class of gene therapy vectors based on <a href="/wiki/HIV" title="HIV">HIV</a> capable of infecting non-dividing cells that have since then been widely used in clinical and research settings, pioneering <a href="/wiki/Lentiviral_vector_in_gene_therapy" title="Lentiviral vector in gene therapy">lentivirals vector in gene therapy</a>.<a href="#cite_note-172">[172]</a> <a href="/wiki/Jesse_Gelsinger" title="Jesse Gelsinger">Jesse Gelsinger</a>'s death in 1999 impeded gene therapy research in the US.<a href="#cite_note-173">[173]</a><a href="#cite_note-174">[174]</a> As a result, the FDA suspended several clinical trials pending the reevaluation of ethical and procedural practices.<a href="#cite_note-175">[175]</a> <div class="mw-heading mw-heading3"><h3 id="2000s">2000s</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=26" title="Edit section: 2000s">edit</a>]</div> The modified gene therapy strategy of antisense IGF-I RNA (NIH n˚ 1602)<a href="#cite_note-pmid22400112-168">[168]</a> using antisense / triple helix anti-IGF-I approach was registered in 2002, by Wiley gene therapy clinical trial - n˚ 635 and 636. The approach has shown promising results in the treatment of six different malignant tumors: <a href="/wiki/Glioblastoma" title="Glioblastoma">glioblastoma</a>, cancers of liver, colon, prostate, uterus, and ovary (Collaborative NATO Science Programme on Gene Therapy USA, France, Poland n˚ LST 980517 conducted by J. Trojan) (Trojan et al., 2012). This anti-gene antisense/triple helix therapy has proven to be efficient, due to the mechanism stopping simultaneously IGF-I expression on translation and transcription levels, strengthening anti-tumor immune and apoptotic phenomena. <div class="mw-heading mw-heading4"><h4 id="2002">2002</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=27" title="Edit section: 2002">edit</a>]</div> <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">Sickle cell disease</a> can be treated in mice.<a href="#cite_note-176">[176]</a> The mice – which have essentially the same defect that causes human cases – used a viral vector to induce production of <a href="/wiki/Fetal_hemoglobin" title="Fetal hemoglobin">fetal hemoglobin</a> (HbF), which normally ceases to be produced shortly after birth. In humans, the use of <a href="/wiki/Hydroxyurea" class="mw-redirect" title="Hydroxyurea">hydroxyurea</a> to stimulate the production of HbF temporarily alleviates sickle cell symptoms. The researchers demonstrated this treatment to be a more permanent means to increase therapeutic HbF production.<a href="#cite_note-St_Jude-177">[177]</a> A new gene therapy approach repaired errors in <a href="/wiki/Messenger_RNA" title="Messenger RNA">messenger RNA</a> derived from defective genes. This technique has the potential to treat <a href="/wiki/Thalassaemia" class="mw-redirect" title="Thalassaemia">thalassaemia</a>, <a href="/wiki/Cystic_fibrosis" title="Cystic fibrosis">cystic fibrosis</a> and some cancers.<a href="#cite_note-178">[178]</a> Researchers created <a href="/wiki/Liposomes" class="mw-redirect" title="Liposomes">liposomes</a> 25 nanometers across that can carry therapeutic DNA through pores in the <a href="/wiki/Nuclear_membrane" class="mw-redirect" title="Nuclear membrane">nuclear membrane</a>.<a href="#cite_note-179">[179]</a> <div class="mw-heading mw-heading4"><h4 id="2003">2003</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=28" title="Edit section: 2003">edit</a>]</div> In 2003, a research team inserted genes into the brain for the first time. They used <a href="/wiki/Liposomes" class="mw-redirect" title="Liposomes">liposomes</a> coated in a <a href="/wiki/Polymer" title="Polymer">polymer</a> called <a href="/wiki/Polyethylene_glycol" title="Polyethylene glycol">polyethylene glycol</a>, which unlike viral vectors, are small enough to cross the <a href="/wiki/Blood%E2%80%93brain_barrier" title="Blood–brain barrier">blood–brain barrier</a>.<a href="#cite_note-180">[180]</a> Short pieces of <a href="/wiki/Double-stranded_RNA" title="Double-stranded RNA">double-stranded RNA</a> (short, interfering RNAs or <a href="/wiki/SiRNA" class="mw-redirect" title="SiRNA">siRNAs</a>) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.<a href="#cite_note-181">[181]</a> <a href="/wiki/Gendicine" title="Gendicine">Gendicine</a> is a cancer gene therapy that delivers the <a href="/wiki/Tumor_suppressor" class="mw-redirect" title="Tumor suppressor">tumor suppressor</a> gene <a href="/wiki/P53" title="P53">p53</a> using an engineered <a href="/wiki/Adenovirus" class="mw-redirect" title="Adenovirus">adenovirus</a>. In 2003, it was approved in China for the treatment of <a href="/wiki/Head_and_neck_cancer" title="Head and neck cancer">head and neck squamous cell carcinoma</a>.<a href="#cite_note-Gend-31">[31]</a> <div class="mw-heading mw-heading4"><h4 id="2006">2006</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=29" title="Edit section: 2006">edit</a>]</div> In March, researchers announced the successful use of gene therapy to treat two adult patients for X-linked <a href="/wiki/Chronic_granulomatous_disease" title="Chronic granulomatous disease">chronic granulomatous disease</a>, a disease which affects <a href="/wiki/Myeloid" class="mw-redirect" title="Myeloid">myeloid</a> cells and damages the <a href="/wiki/Immune_system" title="Immune system">immune system</a>. The study is the first to show that gene therapy can treat the myeloid system.<a href="#cite_note-182">[182]</a> In May, a team reported a way to prevent the immune system from rejecting a newly delivered gene.<a href="#cite_note-183">[183]</a> Similar to <a href="/wiki/Organ_transplant" class="mw-redirect" title="Organ transplant">organ transplantation</a>, gene therapy has been plagued by this problem. The immune system normally recognizes the new gene as foreign and rejects the cells carrying it. The research utilized a newly uncovered network of genes regulated by molecules known as <a href="/wiki/MicroRNA" title="MicroRNA">microRNAs</a>. This natural function selectively obscured their therapeutic gene in immune system cells and protected it from discovery. Mice infected with the gene containing an immune-cell microRNA target sequence did not reject the gene. In August, scientists successfully treated metastatic <a href="/wiki/Melanoma" title="Melanoma">melanoma</a> in two patients using <a href="/wiki/Cytotoxic_T_cell" title="Cytotoxic T cell">killer T cells</a> genetically retargeted to attack the cancer cells.<a href="#cite_note-184">[184]</a> In November, researchers reported on the use of VRX496, a gene-based <a href="/wiki/Immunotherapy" title="Immunotherapy">immunotherapy</a> for the treatment of <a href="/wiki/HIV" title="HIV">HIV</a> that uses a <a href="/wiki/Lentivirus" title="Lentivirus">lentiviral</a> <a href="/wiki/Viral_vector" title="Viral vector">vector</a> to deliver an <a href="/wiki/Sense_(molecular_biology)" title="Sense (molecular biology)">antisense</a> gene against the <a href="/wiki/HIV_envelope" class="mw-redirect" title="HIV envelope">HIV envelope</a>. In a <a href="/wiki/Phase_I_clinical_trial" class="mw-redirect" title="Phase I clinical trial">phase I clinical trial</a>, five subjects with chronic HIV infection who had failed to respond to at least two <a href="/wiki/Antiretroviral" class="mw-redirect" title="Antiretroviral">antiretroviral</a> regimens were treated. A single intravenous infusion of <a href="/wiki/Autologous" class="mw-redirect" title="Autologous">autologous</a> <a href="/wiki/CD4" title="CD4">CD4</a> T cells genetically modified with VRX496 was well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. All five patients had stable or increased immune response to HIV <a href="/wiki/Antigen" title="Antigen">antigens</a> and other <a href="/wiki/Pathogen" title="Pathogen">pathogens</a>. This was the first evaluation of a lentiviral vector administered in a US human clinical trial.<a href="#cite_note-185">[185]</a><a href="#cite_note-186">[186]</a> <div class="mw-heading mw-heading4"><h4 id="2007">2007</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=30" title="Edit section: 2007">edit</a>]</div> In May 2007, researchers announced the first gene therapy trial for inherited <a href="/wiki/Retinal_disease" class="mw-redirect" title="Retinal disease">retinal disease</a>. The first operation was carried out on a 23-year-old British male, Robert Johnson, in early 2007.<a href="#cite_note-187">[187]</a> <div class="mw-heading mw-heading4"><h4 id="2008">2008</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=31" title="Edit section: 2008">edit</a>]</div> <a href="/wiki/Leber%27s_congenital_amaurosis" class="mw-redirect" title="Leber's congenital amaurosis">Leber's congenital amaurosis</a> is an inherited blinding disease caused by mutations in the <a href="/wiki/RPE65" title="RPE65">RPE65</a> gene. The results of a small clinical trial in children were published in April.<a href="#cite_note-Maguire_2008-17">[17]</a> Delivery of recombinant <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a> (AAV) carrying RPE65 yielded positive results. In May, two more groups reported positive results in independent clinical trials using gene therapy to treat the condition. In all three clinical trials, patients recovered functional vision without apparent side-effects.<a href="#cite_note-Maguire_2008-17">[17]</a><a href="#cite_note-Simonelli_2010-18">[18]</a><a href="#cite_note-Cideciyan_2009-19">[19]</a><a href="#cite_note-Bainbridge_2008-20">[20]</a> <div class="mw-heading mw-heading4"><h4 id="2009">2009</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=32" title="Edit section: 2009">edit</a>]</div> In September researchers were able to give <a href="/wiki/Trichromatic_vision" class="mw-redirect" title="Trichromatic vision">trichromatic vision</a> to <a href="/wiki/Squirrel_monkeys" class="mw-redirect" title="Squirrel monkeys">squirrel monkeys</a>.<a href="#cite_note-188">[188]</a> In November 2009, researchers halted a fatal <a href="/wiki/Genetic_disorder" title="Genetic disorder">genetic disorder</a> called <a href="/wiki/Adrenoleukodystrophy" title="Adrenoleukodystrophy">adrenoleukodystrophy</a> in two children using a <a href="/wiki/Lentivirus" title="Lentivirus">lentivirus</a> vector to deliver a functioning version of <a href="/wiki/ABCD1" title="ABCD1">ABCD1</a>, the gene that is mutated in the disorder.<a href="#cite_note-pmid19892975-189">[189]</a> <div class="mw-heading mw-heading3"><h3 id="2010s">2010s</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=33" title="Edit section: 2010s">edit</a>]</div> <div class="mw-heading mw-heading4"><h4 id="2010">2010</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=34" title="Edit section: 2010">edit</a>]</div> An April paper reported that gene therapy addressed <a href="/wiki/Achromatopsia" title="Achromatopsia">achromatopsia</a> (color blindness) in dogs by targeting <a href="/wiki/Cone_(vision)" class="mw-redirect" title="Cone (vision)">cone</a> photoreceptors. Cone function and day vision were restored for at least 33 months in two young specimens. The therapy was less efficient for older dogs.<a href="#cite_note-Komáromy-190">[190]</a> In September it was announced that an 18-year-old male patient in France with <a href="/wiki/Beta_thalassemia" title="Beta thalassemia">beta thalassemia</a> major had been successfully treated.<a href="#cite_note-191">[191]</a> Beta thalassemia major is an inherited <a href="/wiki/Blood_disease" class="mw-redirect" title="Blood disease">blood disease</a> in which <a href="/wiki/HBB" class="mw-redirect" title="HBB">beta haemoglobin</a> is missing and patients are dependent on regular lifelong <a href="/wiki/Blood_transfusions" class="mw-redirect" title="Blood transfusions">blood transfusions</a>.<a href="#cite_note-192">[192]</a> The technique used a lentiviral vector to transduce the human β-globin gene into purified blood and <a href="/wiki/Bone_marrow" title="Bone marrow">marrow</a> cells obtained from the patient in June 2007.<a href="#cite_note-medscape1-193">[193]</a> The patient's haemoglobin levels were stable at 9 to 10 g/dL. About a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions were not needed.<a href="#cite_note-medscape1-193">[193]</a><a href="#cite_note-Leboulch-194">[194]</a> Further clinical trials were planned.<a href="#cite_note-NCT01639690-195">[195]</a> <a href="/wiki/Bone_marrow_transplant" class="mw-redirect" title="Bone marrow transplant">Bone marrow transplants</a> are the only cure for thalassemia, but 75% of patients do not find a matching donor.<a href="#cite_note-Leboulch-194">[194]</a> Cancer immunogene therapy using modified antigene, antisense/triple helix approach was introduced in South America in 2010/11 in La Sabana University, Bogota (Ethical Committee 14 December 2010, no P-004-10). Considering the ethical aspect of gene diagnostic and gene therapy targeting IGF-I, the IGF-I expressing tumors i.e. lung and epidermis cancers were treated (Trojan et al. 2016).<a href="#cite_note-196">[196]</a><a href="#cite_note-197">[197]</a> <div class="mw-heading mw-heading4"><h4 id="2011">2011</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=35" title="Edit section: 2011">edit</a>]</div> In 2007 and 2008, a man (<a href="/wiki/Timothy_Ray_Brown" title="Timothy Ray Brown">Timothy Ray Brown</a>) was cured of HIV by repeated <a href="/wiki/Hematopoietic_stem_cell_transplantation" title="Hematopoietic stem cell transplantation">hematopoietic stem cell transplantation</a> (see also <a href="/wiki/Allogeneic_stem_cell_transplantation" class="mw-redirect" title="Allogeneic stem cell transplantation">allogeneic stem cell transplantation</a>, <a href="/wiki/Allogeneic_bone_marrow_transplantation" class="mw-redirect" title="Allogeneic bone marrow transplantation">allogeneic bone marrow transplantation</a>, <a href="/wiki/Allotransplantation" title="Allotransplantation">allotransplantation</a>) with double-delta-32 mutation which disables the <a href="/wiki/CCR5" title="CCR5">CCR5</a> receptor. This cure was accepted by the medical community in 2011.<a href="#cite_note-198">[198]</a> It required complete <a href="/wiki/Ablation_therapy" class="mw-redirect" title="Ablation therapy">ablation</a> of existing <a href="/wiki/Bone_marrow" title="Bone marrow">bone marrow</a>, which is very debilitating.<a href="#cite_note-199">[199]</a> In August two of three subjects of a pilot study were confirmed to have been cured from <a href="/wiki/Chronic_lymphocytic_leukemia" title="Chronic lymphocytic leukemia">chronic lymphocytic leukemia</a> (CLL). The therapy used genetically modified <a href="/wiki/T_cells" class="mw-redirect" title="T cells">T cells</a> to attack cells that expressed the <a href="/wiki/CD19" title="CD19">CD19</a> protein to fight the disease.<a href="#cite_note-Porter-26">[26]</a> In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free.<a href="#cite_note-200">[200]</a> <a href="/wiki/Human_HGF_plasmid_DNA_therapy" title="Human HGF plasmid DNA therapy">Human HGF plasmid DNA therapy</a> of <a href="/wiki/Cardiomyocytes" class="mw-redirect" title="Cardiomyocytes">cardiomyocytes</a> is being examined as a potential treatment for <a href="/wiki/Coronary_artery_disease" title="Coronary artery disease">coronary artery disease</a> as well as treatment for the damage that occurs to the heart after <a href="/wiki/Myocardial_infarction" title="Myocardial infarction">myocardial infarction</a>.<a href="#cite_note-YangZhang2008-201">[201]</a><a href="#cite_note-HahnPyun2011-202">[202]</a> In 2011, <a href="/wiki/Neovasculgen" class="mw-redirect" title="Neovasculgen">Neovasculgen</a> was registered in Russia as the first-in-class gene-therapy drug for treatment of <a href="/wiki/Peripheral_artery_disease" title="Peripheral artery disease">peripheral artery disease</a>, including <a href="/wiki/Critical_limb_ischemia" class="mw-redirect" title="Critical limb ischemia">critical limb ischemia</a>; it delivers the gene encoding for <a href="/wiki/VEGF" class="mw-redirect" title="VEGF">VEGF</a>.<a href="#cite_note-Neuvasculgen-32">[32]</a> Neovasculogen is a <a href="/wiki/Plasmid" title="Plasmid">plasmid</a> encoding the <a href="/wiki/Cytomegalovirus#Genetic_engineering" title="Cytomegalovirus">CMV promoter</a> and the 165 amino acid form of <a href="/wiki/VEGF" class="mw-redirect" title="VEGF">VEGF</a>.<a href="#cite_note-203">[203]</a><a href="#cite_note-204">[204]</a> <div class="mw-heading mw-heading4"><h4 id="2012">2012</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=36" title="Edit section: 2012">edit</a>]</div> The FDA approved Phase I clinical trials on <a href="/wiki/Thalassemia" title="Thalassemia">thalassemia</a> major patients in the US for 10 participants in July.<a href="#cite_note-205">[205]</a> The study was expected to continue until 2015.<a href="#cite_note-NCT01639690-195">[195]</a> In July 2012, the <a href="/wiki/European_Medicines_Agency" title="European Medicines Agency">European Medicines Agency</a> recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment used <a href="/wiki/Alipogene_tiparvovec" title="Alipogene tiparvovec">Alipogene tiparvovec</a> (Glybera) to compensate for <a href="/wiki/Lipoprotein_lipase_deficiency" title="Lipoprotein lipase deficiency">lipoprotein lipase deficiency</a>, which can cause severe <a href="/wiki/Pancreatitis" title="Pancreatitis">pancreatitis</a>.<a href="#cite_note-206">[206]</a> The recommendation was endorsed by the <a href="/wiki/European_Commission" title="European Commission">European Commission</a> in November 2012,<a href="#cite_note-Richards2012-16">[16]</a><a href="#cite_note-Gallagher-33">[33]</a><a href="#cite_note-207">[207]</a><a href="#cite_note-208">[208]</a> and commercial rollout began in late 2014.<a href="#cite_note-reuters-209">[209]</a> Alipogene tiparvovec was expected to cost around $1.6 million per treatment in 2012,<a href="#cite_note-210">[210]</a> revised to $1 million in 2015,<a href="#cite_note-211">[211]</a> making it the most expensive medicine in the world at the time.<a href="#cite_note-212">[212]</a> As of 2016<a class="external text" href="https://en.wikipedia.org/w/index.php?title=Gene_therapy&action=edit">[update]</a>, only the patients treated in clinical trials and a patient who paid the full price for treatment have received the drug.<a href="#cite_note-MIT-TR2016-213">[213]</a> In December 2012, it was reported that 10 of 13 patients with <a href="/wiki/Multiple_myeloma" title="Multiple myeloma">multiple myeloma</a> were in remission "or very close to it" three months after being injected with a treatment involving genetically engineered <a href="/wiki/T_cells" class="mw-redirect" title="T cells">T cells</a> to target proteins <a href="/wiki/NY-ESO-1" class="mw-redirect" title="NY-ESO-1">NY-ESO-1</a> and <a href="/w/index.php?title=LAGE-1&action=edit&redlink=1" class="new" title="LAGE-1 (page does not exist)">LAGE-1</a>, which exist only on cancerous myeloma cells.<a href="#cite_note-Adaptimmune-28">[28]</a> <div class="mw-heading mw-heading4"><h4 id="2013">2013</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=37" title="Edit section: 2013">edit</a>]</div> In March researchers reported that three of five adult subjects who had <a href="/wiki/Acute_lymphocytic_leukemia" class="mw-redirect" title="Acute lymphocytic leukemia">acute lymphocytic leukemia</a> (ALL) had been in remission for five months to two years after being treated with genetically modified <a href="/wiki/T_cells" class="mw-redirect" title="T cells">T cells</a> which attacked cells with <a href="/wiki/CD19" title="CD19">CD19</a> genes on their surface, i.e. all <a href="/wiki/B_cell" title="B cell">B cells</a>, cancerous or not. The researchers believed that the patients' immune systems would make normal T cells and B cells after a couple of months. They were also given bone marrow. One patient relapsed and died and one died of a blood clot unrelated to the disease.<a href="#cite_note-SloanKettering-27">[27]</a> Following encouraging Phase I trials, in April, researchers announced they were starting Phase II clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients<a href="#cite_note-214">[214]</a> at several hospitals to combat <a href="/wiki/Heart_disease" class="mw-redirect" title="Heart disease">heart disease</a>. The therapy was designed to increase the levels of <a href="/wiki/SERCA" title="SERCA">SERCA</a>2, a protein in heart muscles, improving muscle function.<a href="#cite_note-215">[215]</a> The U.S. <a href="/wiki/Food_and_Drug_Administration" title="Food and Drug Administration">Food and Drug Administration</a> (FDA) granted this a <a href="/wiki/Breakthrough_therapy" title="Breakthrough therapy">breakthrough therapy</a> designation to accelerate the trial and approval process.<a href="#cite_note-216">[216]</a> In 2016, it was reported that no improvement was found from the CUPID 2 trial.<a href="#cite_note-CUPID22016-217">[217]</a> In July researchers reported promising results for six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 7–32 months. Three of the children had <a href="/wiki/Metachromatic_leukodystrophy" title="Metachromatic leukodystrophy">metachromatic leukodystrophy</a>, which causes children to lose cognitive and motor skills.<a href="#cite_note-MLD-218">[218]</a> The other children had <a href="/wiki/Wiskott%E2%80%93Aldrich_syndrome" title="Wiskott–Aldrich syndrome">Wiskott–Aldrich syndrome</a>, which leaves them to open to infection, autoimmune diseases, and cancer.<a href="#cite_note-pmid23845947-219">[219]</a> Follow up trials with gene therapy on another six children with Wiskott–Aldrich syndrome were also reported as promising.<a href="#cite_note-220">[220]</a><a href="#cite_note-221">[221]</a> In October researchers reported that two children born with <a href="/wiki/Adenosine_deaminase_deficiency" title="Adenosine deaminase deficiency">adenosine deaminase severe combined immunodeficiency disease</a> (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and that their immune systems were showing signs of full recovery. Another three children were making progress.<a href="#cite_note-NewScientist1013-24">[24]</a> In 2014, a further 18 children with ADA-SCID were cured by gene therapy.<a href="#cite_note-222">[222]</a> ADA-SCID children have no functioning immune system and are sometimes known as "bubble children".<a href="#cite_note-NewScientist1013-24">[24]</a> Also in October researchers reported that they had treated six people with haemophilia in early 2011 using an adeno-associated virus. Over two years later all six were producing <a href="/wiki/Clotting_factor" class="mw-redirect" title="Clotting factor">clotting factor</a>.<a href="#cite_note-NewScientist1013-24">[24]</a><a href="#cite_note-223">[223]</a> <div class="mw-heading mw-heading4"><h4 id="2014">2014</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=38" title="Edit section: 2014">edit</a>]</div> In January researchers reported that six <a href="/wiki/Choroideremia" title="Choroideremia">choroideremia</a> patients had been treated with adeno-associated virus with a copy of <a href="/wiki/Rab_escort_protein" class="mw-redirect" title="Rab escort protein">REP1</a>. Over a six-month to two-year period all had improved their sight.<a href="#cite_note-pmid24439297-66">[66]</a><a href="#cite_note-224">[224]</a> By 2016, 32 patients had been treated with positive results and researchers were hopeful the treatment would be long-lasting.<a href="#cite_note-BBC2016-21">[21]</a> Choroideremia is an inherited genetic eye disease with no approved treatment, leading to loss of sight. In March researchers reported that 12 HIV patients had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation (<a href="/wiki/CCR5" title="CCR5">CCR5</a> deficiency) known to protect against HIV with promising results.<a href="#cite_note-225">[225]</a><a href="#cite_note-226">[226]</a> Clinical trials of gene therapy for <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a> were started in 2014.<a href="#cite_note-227">[227]</a><a href="#cite_note-228">[228]</a> In February <a href="/wiki/LentiGlobin_BB305" class="mw-redirect" title="LentiGlobin BB305">LentiGlobin BB305</a>, a gene therapy treatment undergoing clinical trials for treatment of <a href="/wiki/Beta_thalassemia" title="Beta thalassemia">beta thalassemia</a> gained FDA "breakthrough" status after several patients were able to forgo the frequent blood transfusions usually required to treat the disease.<a href="#cite_note-229">[229]</a> In March researchers delivered a <a href="/wiki/Recombinant_gene" class="mw-redirect" title="Recombinant gene">recombinant gene</a> encoding a <a href="/wiki/Broadly_neutralizing_antibody" class="mw-redirect" title="Broadly neutralizing antibody">broadly neutralizing antibody</a> into monkeys infected with simian <a href="/wiki/HIV/AIDS" title="HIV/AIDS">HIV</a>; the monkeys' cells produced the <a href="/wiki/Antibody" title="Antibody">antibody</a>, which cleared them of HIV. The technique is named immunoprophylaxis by gene transfer (IGT). Animal tests for antibodies to ebola, malaria, influenza, and hepatitis were underway.<a href="#cite_note-230">[230]</a><a href="#cite_note-231">[231]</a> In March, scientists, including an inventor of <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a>, <a href="/wiki/Jennifer_Doudna" title="Jennifer Doudna">Jennifer Doudna</a>, urged a worldwide moratorium on germline gene therapy, writing "scientists should avoid even attempting, in lax jurisdictions, germline genome modification for clinical application in humans" until the full implications "are discussed among scientific and governmental organizations".<a href="#cite_note-NYT-20150319-153">[153]</a><a href="#cite_note-NYT-20150303-AP-154">[154]</a><a href="#cite_note-SCI-20150319-155">[155]</a><a href="#cite_note-NAT-20150312-156">[156]</a> In December, scientists of major world academies called for a moratorium on inheritable <a href="/wiki/Human_genome" title="Human genome">human genome</a> edits, including those related to <a href="/wiki/Gene_drive#CRISPR" title="Gene drive">CRISPR-Cas9</a> technologies<a href="#cite_note-NYT-20151203-nw-232">[232]</a> but that basic research including embryo gene editing should continue.<a href="#cite_note-BBC31Dec-233">[233]</a> <div class="mw-heading mw-heading4"><h4 id="2015">2015</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=39" title="Edit section: 2015">edit</a>]</div> Researchers successfully treated a boy with <a href="/wiki/Epidermolysis_bullosa" title="Epidermolysis bullosa">epidermolysis bullosa</a> using skin grafts grown from his own skin cells, genetically altered to repair the mutation that caused his disease.<a href="#cite_note-234">[234]</a> In November, researchers announced that they had treated a baby girl, Layla Richards, with an experimental treatment using donor T cells genetically engineered using <a href="/wiki/Transcription_activator-like_effector_nuclease" title="Transcription activator-like effector nuclease">TALEN</a> to attack cancer cells. One year after the treatment she was still free of her cancer (a highly aggressive form of <a href="/wiki/Acute_lymphoblastic_leukaemia" class="mw-redirect" title="Acute lymphoblastic leukaemia">acute lymphoblastic leukaemia</a> [ALL]).<a href="#cite_note-235">[235]</a> Children with highly aggressive ALL normally have a very poor prognosis and Layla's disease had been regarded as terminal before the treatment.<a href="#cite_note-Layla-236">[236]</a><a href="#cite_note-:1-237">[237]</a> <div class="mw-heading mw-heading4"><h4 id="2016">2016</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=40" title="Edit section: 2016">edit</a>]</div> In April the <a href="/wiki/Committee_for_Medicinal_Products_for_Human_Use" title="Committee for Medicinal Products for Human Use">Committee for Medicinal Products for Human Use</a> of the <a href="/wiki/European_Medicines_Agency" title="European Medicines Agency">European Medicines Agency</a> endorsed a gene therapy treatment called <a href="/wiki/Strimvelis" title="Strimvelis">Strimvelis</a><a href="#cite_note-StrimvelisEMA-238">[238]</a><a href="#cite_note-Hirscheler-239">[239]</a> and the European Commission approved it in June.<a href="#cite_note-240">[240]</a> This treats children born with <a href="/wiki/Adenosine_deaminase_deficiency" title="Adenosine deaminase deficiency">adenosine deaminase deficiency</a> and who have no functioning immune system. This was the second gene therapy treatment to be approved in Europe.<a href="#cite_note-Strimvelis-241">[241]</a> In October, Chinese scientists reported they had started a trial to genetically modify T cells from 10 adult patients with lung cancer and reinject the modified T cells back into their bodies to attack the cancer cells. The T cells had the <a href="/wiki/Programmed_cell_death_protein_1" title="Programmed cell death protein 1">PD-1 protein</a> (which stops or slows the immune response) removed using CRISPR-Cas9.<a href="#cite_note-242">[242]</a><a href="#cite_note-243">[243]</a> A 2016 <a href="/wiki/Cochrane_(organisation)" title="Cochrane (organisation)">Cochrane systematic review</a> looking at data from four trials on <a href="/wiki/Cystic_fibrosis_transmembrane_conductance_regulator" title="Cystic fibrosis transmembrane conductance regulator">topical cystic fibrosis transmembrane conductance regulator</a> (CFTR) gene therapy does not support its clinical use as a mist inhaled into the lungs to treat cystic fibrosis patients with lung infections. One of the four trials did find weak evidence that liposome-based CFTR gene transfer therapy may lead to a small respiratory improvement for people with CF. This weak evidence is not enough to make a clinical recommendation for routine CFTR gene therapy.<a href="#cite_note-244">[244]</a> <div class="mw-heading mw-heading4"><h4 id="2017">2017</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=41" title="Edit section: 2017">edit</a>]</div> In February <a href="/wiki/Kite_Pharma" title="Kite Pharma">Kite Pharma</a> announced results from a clinical trial of <a href="/wiki/CAR-T" class="mw-redirect" title="CAR-T">CAR-T</a> cells in around a hundred people with advanced <a href="/wiki/Non-Hodgkin_lymphoma" title="Non-Hodgkin lymphoma">non-Hodgkin lymphoma</a>.<a href="#cite_note-245">[245]</a> In March, French scientists reported on clinical research of gene therapy to treat <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a>.<a href="#cite_note-246">[246]</a> In August, the FDA approved <a href="/wiki/Tisagenlecleucel" title="Tisagenlecleucel">tisagenlecleucel</a> for <a href="/wiki/Acute_lymphoblastic_leukemia" title="Acute lymphoblastic leukemia">acute lymphoblastic leukemia</a>.<a href="#cite_note-247">[247]</a> Tisagenlecleucel is an <a href="/wiki/Adoptive_cell_transfer" title="Adoptive cell transfer">adoptive cell transfer</a> therapy for <a href="/wiki/B-cell" class="mw-redirect" title="B-cell">B-cell</a> acute lymphoblastic leukemia; <a href="/wiki/T_cells" class="mw-redirect" title="T cells">T cells</a> from a person with cancer are removed, <a href="/wiki/Genetically_engineered" class="mw-redirect" title="Genetically engineered">genetically engineered</a> to make a specific <a href="/wiki/T-cell_receptor" title="T-cell receptor">T-cell receptor</a> (a chimeric T cell receptor, or "CAR-T") that reacts to the cancer, and are administered back to the person. The T cells are engineered to target a protein called <a href="/wiki/CD19" title="CD19">CD19</a> that is common on B cells. This is the first form of gene therapy to be approved in the United States. In October, a similar therapy called <a href="/wiki/Axicabtagene_ciloleucel" title="Axicabtagene ciloleucel">axicabtagene ciloleucel</a> was approved for non-Hodgkin lymphoma.<a href="#cite_note-248">[248]</a> In October, <a href="/wiki/Biophysics" title="Biophysics">biophysicist</a> and <a href="/wiki/Do-it-yourself_biology" title="Do-it-yourself biology">biohacker</a> <a href="/wiki/Josiah_Zayner" class="mw-redirect" title="Josiah Zayner">Josiah Zayner</a> claimed to have performed the very first in-vivo human genome editing in the form of a self-administered therapy.<a href="#cite_note-249">[249]</a><a href="#cite_note-250">[250]</a> On 13 November, medical scientists working with <a href="/wiki/Sangamo_Therapeutics" title="Sangamo Therapeutics">Sangamo Therapeutics</a>, headquartered in <a href="/wiki/Richmond,_California" title="Richmond, California">Richmond, California</a>, announced the first ever in-body <a href="/wiki/Human_gene_editing_therapy" class="mw-redirect" title="Human gene editing therapy">human gene editing therapy</a>.<a href="#cite_note-251">[251]</a><a href="#cite_note-252">[252]</a> The treatment, designed to permanently insert a healthy version of the flawed gene that causes <a href="/wiki/Hunter_syndrome" title="Hunter syndrome">Hunter syndrome</a>, was given to 44-year-old Brian Madeux and is part of the world's first study to permanently edit <a href="/wiki/DNA" title="DNA">DNA</a> inside the human body.<a href="#cite_note-253">[253]</a> The success of the gene insertion was later confirmed.<a href="#cite_note-254">[254]</a><a href="#cite_note-AP-20190207-255">[255]</a> Clinical trials by Sangamo involving gene editing using <a href="/wiki/Zinc_finger_nuclease" class="mw-redirect" title="Zinc finger nuclease">zinc finger nuclease</a> (ZFN) are ongoing.<a href="#cite_note-CT-201902-256">[256]</a> In December the results of using an adeno-associated virus with blood clotting <a href="/wiki/Factor_VIII" title="Factor VIII">factor VIII</a> to treat nine <a href="/wiki/Haemophilia_A" title="Haemophilia A">haemophilia A</a> patients were published. Six of the seven patients on the high dose regime increased the level of the blood clotting VIII to normal levels. The low and medium dose regimes had no effect on the patient's blood clotting levels.<a href="#cite_note-257">[257]</a><a href="#cite_note-258">[258]</a> In December, the FDA approved <a href="/wiki/Voretigene_neparvovec" title="Voretigene neparvovec">voretigene neparvovec</a>, the first in vivo gene therapy, for the treatment of blindness due to <a href="/wiki/Leber%27s_congenital_amaurosis" class="mw-redirect" title="Leber's congenital amaurosis">Leber's congenital amaurosis</a>.<a href="#cite_note-259">[259]</a> The price of this treatment is US$850,000 for both eyes.<a href="#cite_note-260">[260]</a><a href="#cite_note-261">[261]</a> <div class="mw-heading mw-heading4"><h4 id="2019">2019</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=42" title="Edit section: 2019">edit</a>]</div> In May, the FDA approved <a href="/wiki/Onasemnogene_abeparvovec" title="Onasemnogene abeparvovec">onasemnogene abeparvovec</a> (Zolgensma) for treating <a href="/wiki/Spinal_muscular_atrophy" title="Spinal muscular atrophy">spinal muscular atrophy</a> in children under two years of age. The list price of Zolgensma was set at US$2.125 million per dose, making it the most expensive drug ever.<a href="#cite_note-262">[262]</a> In May, the EMA approved <a href="/wiki/Betibeglogene_autotemcel" title="Betibeglogene autotemcel">betibeglogene autotemcel</a> (Zynteglo) for treating <a href="/wiki/Beta_thalassemia" title="Beta thalassemia">beta thalassemia</a> for people twelve years of age and older.<a href="#cite_note-263">[263]</a><a href="#cite_note-Zynteglo_EPAR-264">[264]</a> In July, <a href="/wiki/Allergan" title="Allergan">Allergan</a> and <a href="/wiki/Editas_Medicine" title="Editas Medicine">Editas Medicine</a> announced phase I/II clinical trial of AGN-151587 for the treatment of <a href="/wiki/Leber_congenital_amaurosis" title="Leber congenital amaurosis">Leber congenital amaurosis</a> 10.<a href="#cite_note-265">[265]</a> This is one of the first studies of a <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a>-based <a href="/wiki/In_vivo" title="In vivo">in vivo</a> <a href="/wiki/Human_gene_editing_therapy" class="mw-redirect" title="Human gene editing therapy">human gene editing therapy</a>, where the editing takes place inside the human body.<a href="#cite_note-266">[266]</a> The first injection of the CRISPR-Cas System was confirmed in March 2020.<a href="#cite_note-267">[267]</a> <a href="/wiki/Exagamglogene_autotemcel" title="Exagamglogene autotemcel">Exagamglogene autotemcel</a>, a <a href="/wiki/CRISPR" title="CRISPR">CRISPR</a>-based <a href="/wiki/Human_gene_editing_therapy" class="mw-redirect" title="Human gene editing therapy">human gene editing therapy</a>, was used for <a href="/wiki/Sickle_cell" class="mw-redirect" title="Sickle cell">sickle cell</a> and <a href="/wiki/Thalassemia" title="Thalassemia">thalassemia</a> in clinical trials.<a href="#cite_note-268">[268]</a> <div class="mw-heading mw-heading3"><h3 id="2020s">2020s</h3>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=43" title="Edit section: 2020s">edit</a>]</div> <div class="mw-heading mw-heading4"><h4 id="2020">2020</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=44" title="Edit section: 2020">edit</a>]</div> In May, onasemnogene abeparvovec (Zolgensma) was approved by the European Union for the treatment of spinal muscular atrophy in people who either have clinical symptoms of SMA type 1 or who have no more than three copies of the <a href="/wiki/SMN2" title="SMN2">SMN2 gene</a>, irrespective of body weight or age.<a href="#cite_note-Zolgensma_EPAR-269">[269]</a> In August, <a href="/wiki/Astellas_Pharma" title="Astellas Pharma">Audentes Therapeutics</a> reported that three out of 17 children with <a href="/wiki/X-linked_myotubular_myopathy" title="X-linked myotubular myopathy">X-linked myotubular myopathy</a> participating the clinical trial of a AAV8-based gene therapy treatment AT132 have died. It was suggested that the treatment, whose dosage is based on body weight, exerts a disproportionately toxic effect on heavier patients, since the three patients who died were heavier than the others.<a href="#cite_note-270">[270]</a><a href="#cite_note-271">[271]</a> The trial has been put on clinical hold.<a href="#cite_note-272">[272]</a> On 15 October, the <a href="/wiki/Committee_for_Medicinal_Products_for_Human_Use" title="Committee for Medicinal Products for Human Use">Committee for Medicinal Products for Human Use</a> (CHMP) of the <a href="/wiki/European_Medicines_Agency" title="European Medicines Agency">European Medicines Agency</a> (EMA) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product <a href="/wiki/Libmeldy" class="mw-redirect" title="Libmeldy">Libmeldy</a> (autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene), a gene therapy for the treatment of children with the "late infantile" (LI) or "early juvenile" (EJ) forms of metachromatic leukodystrophy (MLD).<a href="#cite_note-Libmeldy:_Pending_EC_decision-273">[273]</a> The active substance of Libmeldy consists of the child's own stem cells which have been modified to contain working copies of the ARSA gene.<a href="#cite_note-Libmeldy:_Pending_EC_decision-273">[273]</a> When the modified cells are injected back into the patient as a one-time infusion, the cells are expected to start producing the ARSA enzyme that breaks down the build-up of sulfatides in the nerve cells and other cells of the patient's body.<a href="#cite_note-EMA_PR-274">[274]</a> Libmeldy was approved for medical use in the EU in December 2020.<a href="#cite_note-Libmeldy_EPAR-275">[275]</a> On 15 October, Lysogene, a French biotechnological company, reported the death of a patient in who has received LYS-SAF302, an experimental gene therapy treatment for <a href="/wiki/Sanfilippo_syndrome" title="Sanfilippo syndrome">mucopolysaccharidosis type IIIA</a> (Sanfilippo syndrome type A).<a href="#cite_note-276">[276]</a> <div class="mw-heading mw-heading4"><h4 id="2021">2021</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=45" title="Edit section: 2021">edit</a>]</div> In May, a new method using an altered version of <a href="/wiki/HIV" title="HIV">HIV</a> as a <a href="/wiki/Viral_vector#Lentiviruses" title="Viral vector">lentivirus vector</a> was reported in the treatment of 50 children with <a href="/wiki/Adenosine_deaminase_deficiency" title="Adenosine deaminase deficiency">ADA-SCID</a> obtaining positive results in 48 of them,<a href="#cite_note-277">[277]</a><a href="#cite_note-278">[278]</a><a href="#cite_note-279">[279]</a> this method is expected to be safer than <a href="/wiki/Viral_vector#Retroviruses" title="Viral vector">retroviruses vectors</a> commonly used in previous studies of SCID where the development of <a href="/wiki/Leukemia" title="Leukemia">leukemia</a> was usually observed<a href="#cite_note-280">[280]</a> and had already been used in 2019, but in a smaller group with X-SCID.<a href="#cite_note-281">[281]</a><a href="#cite_note-282">[282]</a><a href="#cite_note-283">[283]</a><a href="#cite_note-:02-284">[284]</a> In June a clinical trial on six patients affected with <a href="/wiki/Familial_amyloid_polyneuropathy" title="Familial amyloid polyneuropathy">transthyretin amyloidosis</a> reported a reduction the concentration of missfolded <a href="/wiki/Transthyretin" title="Transthyretin">transthretin</a> (TTR) protein in serum through <a href="/wiki/CRISPR_gene_editing" title="CRISPR gene editing">CRISPR</a>-based inactivation of the TTR gene in liver cells observing mean reductions of 52% and 87% among the lower and higher dose groups.This was done in vivo without taking cells out of the patient to edit them and reinfuse them later.<a href="#cite_note-285">[285]</a><a href="#cite_note-286">[286]</a><a href="#cite_note-287">[287]</a> In July results of a small gene therapy <a href="/wiki/Phases_of_clinical_research#Phase_I" title="Phases of clinical research">phase I</a> study was published reporting observation of dopamine restoration on seven patients between 4 and 9 years old affected by <a href="/wiki/Aromatic_L-amino_acid_decarboxylase_deficiency" title="Aromatic L-amino acid decarboxylase deficiency">aromatic L-amino acid decarboxylase deficiency</a> (AADC deficiency).<a href="#cite_note-288">[288]</a><a href="#cite_note-289">[289]</a><a href="#cite_note-290">[290]</a> <div class="mw-heading mw-heading4"><h4 id="2022">2022</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=46" title="Edit section: 2022">edit</a>]</div> In February, the first ever gene therapy for <a href="/wiki/Tay%E2%80%93Sachs_disease" title="Tay–Sachs disease">Tay–Sachs disease</a> was announced, it uses an <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a> to deliver the correct instruction for the <a href="/wiki/HEXA" title="HEXA">HEXA</a> gene on brain cells which causes the disease. Only two children were part of a compassionate trial presenting improvements over the natural course of the disease and no vector-related <a href="/wiki/Adverse_event" title="Adverse event">adverse events</a>.<a href="#cite_note-291">[291]</a><a href="#cite_note-292">[292]</a><a href="#cite_note-293">[293]</a> In May, <a href="/wiki/Eladocagene_exuparvovec" title="Eladocagene exuparvovec">eladocagene exuparvovec</a> is recommended for approval by the European Commission.<a href="#cite_note-Upstaza:_Pending_EC_decision-294">[294]</a><a href="#cite_note-PTC_PR_20220520-295">[295]</a> In July results of a gene therapy candidate for <a href="/wiki/Haemophilia_B" title="Haemophilia B">haemophilia B</a> called FLT180 were announced, it works using an <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a> (AAV) to restore the clotting <a href="/wiki/Factor_IX" title="Factor IX">factor IX</a> (FIX) protein, normal levels of the protein were observed with low doses of the therapy but immunosuppression was necessitated to decrease the risk of vector-related immune responses.<a href="#cite_note-296">[296]</a><a href="#cite_note-297">[297]</a><a href="#cite_note-298">[298]</a> In December, a 13-year girl that had been diagnosed with <a href="/wiki/T-cell_acute_lymphoblastic_leukemia" title="T-cell acute lymphoblastic leukemia">T-cell acute lymphoblastic leukaemia</a> was successfully treated at <a href="/wiki/Great_Ormond_Street_Hospital" title="Great Ormond Street Hospital">Great Ormond Street Hospital</a> (GOSH) in the first documented use of therapeutic gene editing for this purpose, after undergoing six months of an experimental treatment, where all attempts of other treatments failed. The procedure included reprogramming a healthy T-cell to destroy the cancerous T-cells to first rid her of leukaemia, and then rebuilding her immune system using healthy immune cells.<a href="#cite_note-299">[299]</a> The GOSH team used <a href="/wiki/Genome_editing#Editing_by_nucleobase_modification_(BASE_editing)" title="Genome editing">BASE editing</a> and had <a href="#2015">previously</a> treated a case of <a href="/wiki/Acute_lymphoblastic_leukaemia" class="mw-redirect" title="Acute lymphoblastic leukaemia">acute lymphoblastic leukaemia</a> in 2015 using <a href="/wiki/TALENs" class="mw-redirect" title="TALENs">TALENs</a>.<a href="#cite_note-:1-237">[237]</a> <div class="mw-heading mw-heading4"><h4 id="2023">2023</h4>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=47" title="Edit section: 2023">edit</a>]</div> In May 2023, the FDA approved <a href="/wiki/Beremagene_geperpavec" title="Beremagene geperpavec">beremagene geperpavec</a> for the treatment of wounds in people with <a href="/wiki/Dystrophic_epidermolysis_bullosa" class="mw-redirect" title="Dystrophic epidermolysis bullosa">dystrophic epidermolysis bullosa</a> (DEB) which is applied as a topical gel that delivers a herpes-simplex virus type 1 (HSV-1) vector encoding the collagen type VII alpha 1 chain (<a href="/wiki/COL7A1" class="mw-redirect" title="COL7A1">COL7A1</a>) gene that is dysfunctional on those affected by DEB . One trial found 65% of the Vyjuvek-treated wounds completely closed while only 26% of the placebo-treated at 24 weeks.<a href="#cite_note-:9-97">[97]</a> It has been also reported its use as an eyedrop for a patient with DEB that had vision loss due to the widespread blistering with good results.<a href="#cite_note-300">[300]</a> In June 2023, the FDA gave an <a href="/wiki/Accelerated_approval_(FDA)" title="Accelerated approval (FDA)">accelerated approval</a> to <a href="/wiki/Delandistrogene_moxeparvovec" title="Delandistrogene moxeparvovec">Elevidys</a> for <a href="/wiki/Duchenne_muscular_dystrophy" title="Duchenne muscular dystrophy">Duchenne muscular dystrophy</a> (DMD) only for boys 4 to 5 years old as they are more likely to benefit from the therapy which consists of one-time intravenous infusion of a virus (AAV rh74 vector) that delivers a functioning "microdystrophin" gene (138 <a href="/wiki/Dalton_(unit)" title="Dalton (unit)">kDa</a>) into the muscle cells to act in place of the normal <a href="/wiki/Dystrophin" title="Dystrophin">dystrophin</a> (427 kDa) that is found mutated in this disease.<a href="#cite_note-auto-102">[102]</a> In July 2023, it was reported that it had been developed a new method to affect genetic expressions through direct current.<a href="#cite_note-301">[301]</a> In December 2023, two gene therapies were approved for <a href="/wiki/Sickle_cell_disease" title="Sickle cell disease">sickle cell disease</a>, <a href="/wiki/Exagamglogene_autotemcel" title="Exagamglogene autotemcel">exagamglogene autotemcel</a><a href="#cite_note-FDA_Casgevy-105">[105]</a> and <a href="/wiki/Lovotibeglogene_autotemcel" title="Lovotibeglogene autotemcel">lovotibeglogene autotemcel</a>.<a href="#cite_note-FDA_Lyfgenia-108">[108]</a> 2024 In November 2024, FDA granted accelerated approval for <a href="/wiki/Eladocagene_exuparvovec" title="Eladocagene exuparvovec">eladocagene exuparvovec</a>-tneq (Kebilidi, <a href="/wiki/PTC_Therapeutics" title="PTC Therapeutics">PTC Therapeutics</a>), a direct-to-brain gene therapy for <a href="/wiki/Aromatic_L-amino_acid_decarboxylase_deficiency" title="Aromatic L-amino acid decarboxylase deficiency">aromatic L-amino acid decarboxylase deficiency</a>.<a href="#cite_note-h6262-302">[302]</a> It uses a recombinant <a href="/wiki/Adeno-associated_virus" title="Adeno-associated virus">adeno-associated virus</a> serotype 2 (rAAV2) to deliver a functioning <a href="/wiki/DOPA_decarboxylase" class="mw-redirect" title="DOPA decarboxylase">DOPA decarboxylase</a> (DDC) gene directly into the <a href="/wiki/Putamen" title="Putamen">putamen</a>, increasing the AADC enzyme and restoring <a href="/wiki/Dopamine" title="Dopamine">dopamine</a> production. It is administered through a <a href="/wiki/Stereotactic_injection" title="Stereotactic injection">stereotactic</a> surgical procedure.<a href="#cite_note-FDA_PR_20241114-103">[103]</a> <div class="mw-heading mw-heading2"><h2 id="List_of_gene_therapies">List of gene therapies</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=48" title="Edit section: List of gene therapies">edit</a>]</div> <ul><li><a href="/wiki/Gene_therapy_for_color_blindness" title="Gene therapy for color blindness">Gene therapy for color blindness</a></li> <li><a href="/wiki/Gene_therapy_for_epilepsy" title="Gene therapy for epilepsy">Gene therapy for epilepsy</a></li> <li><a href="/wiki/Gene_therapy_for_osteoarthritis" title="Gene therapy for osteoarthritis">Gene therapy for osteoarthritis</a></li> <li><a href="/wiki/Gene_therapy_in_Parkinson%27s_disease" title="Gene therapy in Parkinson's disease">Gene therapy in Parkinson's disease</a></li> <li><a href="/wiki/Gene_therapy_of_the_human_retina" title="Gene therapy of the human retina">Gene therapy of the human retina</a></li> <li><a href="/wiki/List_of_gene_therapies" title="List of gene therapies">List of gene therapies</a></li></ul> <div class="mw-heading mw-heading2"><h2 id="References">References</h2>[<a href="/w/index.php?title=Gene_therapy&action=edit&section=49" title="Edit section: References">edit</a>]</div> <style data-mw-deduplicate="TemplateStyles:r1239543626">.mw-parser-output .reflist{margin-bottom:0.5em;list-style-type:decimal}@media screen{.mw-parser-output .reflist{font-size:90%}}.mw-parser-output .reflist .references{font-size:100%;margin-bottom:0;list-style-type:inherit}.mw-parser-output .reflist-columns-2{column-width:30em}.mw-parser-output .reflist-columns-3{column-width:25em}.mw-parser-output .reflist-columns{margin-top:0.3em}.mw-parser-output .reflist-columns ol{margin-top:0}.mw-parser-output .reflist-columns li{page-break-inside:avoid;break-inside:avoid-column}.mw-parser-output .reflist-upper-alpha{list-style-type:upper-alpha}.mw-parser-output .reflist-upper-roman{list-style-type:upper-roman}.mw-parser-output .reflist-lower-alpha{list-style-type:lower-alpha}.mw-parser-output .reflist-lower-greek{list-style-type:lower-greek}.mw-parser-output .reflist-lower-roman{list-style-type:lower-roman}</style><div class="reflist"> <div class="mw-references-wrap mw-references-columns"><ol class="references"> <li id="cite_note-1"><a href="#cite_ref-1">^</a> <style data-mw-deduplicate="TemplateStyles:r1238218222">.mw-parser-output cite.citation{font-style:inherit;word-wrap:break-word}.mw-parser-output .citation q{quotes:"\"""\"""'""'"}.mw-parser-output .citation:target{background-color:rgba(0,127,255,0.133)}.mw-parser-output .id-lock-free.id-lock-free a{background:url("//upload.wikimedia.org/wikipedia/commons/6/65/Lock-green.svg")right 0.1em center/9px no-repeat}.mw-parser-output .id-lock-limited.id-lock-limited a,.mw-parser-output .id-lock-registration.id-lock-registration a{background:url("//upload.wikimedia.org/wikipedia/commons/d/d6/Lock-gray-alt-2.svg")right 0.1em center/9px no-repeat}.mw-parser-output .id-lock-subscription.id-lock-subscription a{background:url("//upload.wikimedia.org/wikipedia/commons/a/aa/Lock-red-alt-2.svg")right 0.1em center/9px no-repeat}.mw-parser-output .cs1-ws-icon a{background:url("//upload.wikimedia.org/wikipedia/commons/4/4c/Wikisource-logo.svg")right 0.1em center/12px no-repeat}body:not(.skin-timeless):not(.skin-minerva) .mw-parser-output .id-lock-free a,body:not(.skin-timeless):not(.skin-minerva) .mw-parser-output .id-lock-limited a,body:not(.skin-timeless):not(.skin-minerva) .mw-parser-output .id-lock-registration a,body:not(.skin-timeless):not(.skin-minerva) .mw-parser-output .id-lock-subscription a,body:not(.skin-timeless):not(.skin-minerva) .mw-parser-output .cs1-ws-icon a{background-size:contain;padding:0 1em 0 0}.mw-parser-output .cs1-code{color:inherit;background:inherit;border:none;padding:inherit}.mw-parser-output .cs1-hidden-error{display:none;color:var(--color-error,#d33)}.mw-parser-output .cs1-visible-error{color:var(--color-error,#d33)}.mw-parser-output .cs1-maint{display:none;color:#085;margin-left:0.3em}.mw-parser-output .cs1-kern-left{padding-left:0.2em}.mw-parser-output .cs1-kern-right{padding-right:0.2em}.mw-parser-output .citation .mw-selflink{font-weight:inherit}@media screen{.mw-parser-output .cs1-format{font-size:95%}html.skin-theme-clientpref-night .mw-parser-output .cs1-maint{color:#18911f}}@media screen and (prefers-color-scheme:dark){html.skin-theme-clientpref-os .mw-parser-output .cs1-maint{color:#18911f}}</style><cite id="CITEREFKajiLeiden2001" class="citation journal cs1">Kaji EH, Leiden JM (February 2001). <a rel="nofollow" class="external text" href="https://doi.org/10.1001%2Fjama.285.5.545">"Gene and stem cell therapies"</a>. 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class="nowraplinks hlist mw-collapsible autocollapse navbox-inner" style="border-spacing:0;background:transparent;color:inherit"><tbody><tr><th scope="col" class="navbox-title" colspan="2"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1239400231"><div class="navbar plainlinks hlist navbar-mini"><ul><li class="nv-view"><a href="/wiki/Template:Genetic_engineering" title="Template:Genetic engineering"><abbr title="View this template">v</abbr></a></li><li class="nv-talk"><a href="/wiki/Template_talk:Genetic_engineering" title="Template talk:Genetic engineering"><abbr title="Discuss this template">t</abbr></a></li><li class="nv-edit"><a href="/wiki/Special:EditPage/Template:Genetic_engineering" title="Special:EditPage/Template:Genetic engineering"><abbr title="Edit this template">e</abbr></a></li></ul></div><div id="Genetic_engineering" style="font-size:114%;margin:0 4em"><a href="/wiki/Genetic_engineering" title="Genetic engineering">Genetic engineering</a></div></th></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_organism" title="Genetically modified organism">Genetically modified organisms</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_crops" title="Genetically modified crops">Crops</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_maize" title="Genetically modified maize">Maize/corn</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/MON_810" title="MON 810">MON 810</a></li> <li><a href="/wiki/MON_863" title="MON 863">MON 863</a></li> <li><a href="/wiki/Starlink_corn_recall#StarLink_corn" class="mw-redirect" title="Starlink corn recall">StarLink</a></li> <li><a href="/wiki/List_of_varieties_of_genetically_modified_maize" title="List of varieties of genetically modified maize">List of varieties of genetically modified maize/corn</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_potato" title="Genetically modified potato">Potato</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Amflora" title="Amflora">Amflora</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_rice" title="Genetically modified rice">Rice</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Golden_rice" title="Golden rice">Golden rice</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_soybean" title="Genetically modified soybean">Soybean</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Roundup_ready_soybean" class="mw-redirect" title="Roundup ready soybean">Roundup ready soybean</a></li> <li><a href="/wiki/Vistive_Gold" title="Vistive Gold">Vistive Gold</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_tomato" title="Genetically modified tomato">Tomato</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Flavr_Savr" title="Flavr Savr">Flavr Savr</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Cotton#Genetic_modification" title="Cotton">Cotton</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Bt_cotton" title="Bt cotton">Bt cotton</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_wheat" title="Genetically modified wheat">Wheat</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/HB4_Wheat" title="HB4 Wheat">HB4</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Other</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Arctic_Apples" title="Arctic Apples">Apple</a></li> <li><a href="/wiki/Arabidopsis_thaliana" title="Arabidopsis thaliana">Arabidopsis</a></li> <li><a href="/wiki/Genetically_modified_brinjal" title="Genetically modified brinjal">Brinjal</a></li> <li><a href="/wiki/Genetically_modified_canola" title="Genetically modified canola">Canola</a></li> <li><a href="/wiki/Papaya#Genetically_engineered_cultivars" title="Papaya">Papaya</a></li> <li><a href="/wiki/Blue_rose" title="Blue rose">Rose</a></li> <li><a href="/wiki/SmartStax" title="SmartStax">SmartStax</a></li> <li><a href="/wiki/Genetically_modified_sugar_beet" title="Genetically modified sugar beet">Sugar beet</a></li> <li><a href="/wiki/Tobacco#Genetic_modification" title="Tobacco">Tobacco</a></li> <li><a href="/wiki/Genetically_modified_tree" title="Genetically modified tree">Trees</a></li> <li><a href="/wiki/DMH-11_Mustard" title="DMH-11 Mustard">Mustard</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_animal" title="Genetically modified animal">Animals</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_mammal" title="Genetically modified mammal">Mammals</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Genetically_modified_mouse" title="Genetically modified mouse">Mouse</a> <ul><li><a href="/wiki/Knockout_mouse" title="Knockout mouse">Knockout mouse</a></li> <li><a href="/wiki/Oncomouse" title="Oncomouse">Oncomouse</a></li></ul></li> <li><a href="/wiki/Enviropig" class="mw-redirect" title="Enviropig">Enviropig</a></li> <li><a href="/wiki/Herman_the_Bull" class="mw-redirect" title="Herman the Bull">Herman the Bull</a></li> <li><a href="/wiki/Knockout_rat" title="Knockout rat">Knockout rat</a></li> <li><a href="/wiki/Alba_(rabbit)" title="Alba (rabbit)">Rabbit</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Other animals</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Genetically_modified_insect" title="Genetically modified insect">Insects</a></li> <li><a href="/wiki/Genetically_modified_fish" title="Genetically modified fish">Fish</a> <ul><li><a href="/wiki/GloFish" title="GloFish">GloFish</a></li> <li><a href="/wiki/Genetically_modified_salmon" class="mw-redirect" title="Genetically modified salmon">Salmon</a></li></ul></li> <li><a href="/wiki/Genetically_modified_bird" class="mw-redirect" title="Genetically modified bird">Birds</a></li> <li><a href="/wiki/Genetically_modified_organism#Frogs" title="Genetically modified organism">Frogs</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetically_modified_bacteria" title="Genetically modified bacteria">Bacteria</a> and <a href="/wiki/Genetically_modified_virus" title="Genetically modified virus">viruses</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Ice-minus_bacteria" title="Ice-minus bacteria">Ice-minus bacteria</a></li> <li><a href="/wiki/Hepatitis_B_vaccine" title="Hepatitis B vaccine">Hepatitis B vaccine</a></li> <li><a href="/wiki/Oncolytic_virus" title="Oncolytic virus">Oncolytic virus</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Genetic_engineering_techniques" title="Genetic engineering techniques">Processes</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Transformation_(genetics)" class="mw-redirect" title="Transformation (genetics)">Inserting DNA</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Agrobacterium" title="Agrobacterium">Agrobacteria</a></li> <li><a href="/wiki/Gene_gun" title="Gene gun">Biolistics</a></li> <li><a href="/wiki/Electroporation" title="Electroporation">Electroporation</a></li> <li><a href="/wiki/Genetic_transduction" class="mw-redirect" title="Genetic transduction">Genetic transduction</a></li> <li><a href="/wiki/Lipofection" class="mw-redirect" title="Lipofection">Lipofection</a></li> <li><a href="/wiki/Microinjection" title="Microinjection">Microinjection</a></li> <li><a href="/wiki/Somatic_cell_nuclear_transfer" title="Somatic cell nuclear transfer">Somatic cell nuclear transfer</a></li> <li><a href="/wiki/Transfection" title="Transfection">Transfection</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Types</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Recombinant_DNA" title="Recombinant DNA">Recombinant DNA</a></li> <li><a href="/wiki/Transgenesis" class="mw-redirect" title="Transgenesis">Transgenesis</a></li> <li><a href="/wiki/Cisgenesis" title="Cisgenesis">Cisgenesis</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Uses</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%">In agriculture</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Genetically_modified_food" title="Genetically modified food">Genetically modified food</a> <ul><li><a href="/wiki/Genetically_modified_food_controversies" title="Genetically modified food controversies">Controversies</a></li></ul></li> <li><a href="/wiki/Pharming_(genetics)" title="Pharming (genetics)">Pharming</a></li> <li>Companies <ul><li><a href="/wiki/BASF_Plant_Science" title="BASF Plant Science">BASF</a></li> <li><a href="/wiki/Bayer#Bayer_CropScience" title="Bayer">Bayer</a></li> <li><a href="/wiki/Dow_AgroSciences" title="Dow AgroSciences">Dow AgroSciences</a></li> <li><a href="/wiki/DuPont_Pioneer" class="mw-redirect" title="DuPont Pioneer">DuPont Pioneer</a></li> <li><a href="/wiki/Monsanto" title="Monsanto">Monsanto</a></li> <li><a href="/wiki/Syngenta" title="Syngenta">Syngenta</a></li></ul></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">In <a href="/wiki/Human_genetic_engineering" class="mw-redirect" title="Human genetic engineering">humans</a> and diagnostics</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a class="mw-selflink selflink">Gene therapy</a></li> <li><a href="/wiki/Genetic_enhancement" class="mw-redirect" title="Genetic enhancement">Genetic enhancement</a></li> <li><a href="/wiki/Genetic_testing" title="Genetic testing">Genetic testing</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">In research</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Gene_knockout" title="Gene knockout">Gene knockout</a></li> <li><a href="/wiki/Gene_knockdown" title="Gene knockdown">Gene knockdown</a></li> <li><a href="/wiki/Gene_targeting" title="Gene targeting">Gene targeting</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Related articles</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Transgene" title="Transgene">Transgene</a></li> <li><a href="/wiki/Detection_of_genetically_modified_organisms" title="Detection of genetically modified organisms">Detection of genetically modified organisms</a></li> <li><a href="/wiki/Genetic_pollution" title="Genetic pollution">Genetic pollution</a></li> <li><a href="/wiki/Genetics_in_fiction" title="Genetics in fiction">Genetics in fiction</a></li> <li><a href="/wiki/Human_enhancement" title="Human enhancement">Human enhancement</a></li> <li><a href="/wiki/Reverse_transfection" title="Reverse transfection">Reverse transfection</a></li> <li><a href="/wiki/Transhumanism" title="Transhumanism">Transhumanism</a></li> <li><a href="/wiki/Genetic_use_restriction_technology" title="Genetic use restriction technology">Genetic use restriction technology</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Regulation_of_genetic_engineering" title="Regulation of genetic engineering">Regulation</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Cartagena_Protocol_on_Biosafety" title="Cartagena Protocol on Biosafety">Cartagena Protocol on Biosafety</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Geography</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Genetic_engineering_in_Europe" class="mw-redirect" title="Genetic engineering in Europe">Europe</a></li> <li><a href="/wiki/Genetic_engineering_in_Africa" class="mw-redirect" title="Genetic engineering in Africa">Africa</a></li> <li><a href="/wiki/Genetic_engineering_in_Asia" class="mw-redirect" title="Genetic engineering in Asia">Asia</a></li> <li><a href="/wiki/Genetic_engineering_in_North_America" class="mw-redirect" title="Genetic engineering in North America">North America</a> (<a href="/wiki/Genetic_engineering_in_the_United_States" class="mw-redirect" title="Genetic engineering in the United States">US</a>)</li> <li><a href="/wiki/Genetic_engineering_in_South_America" class="mw-redirect" title="Genetic engineering in South America">South America</a></li> <li><a href="/wiki/Genetic_engineering_in_Oceania" class="mw-redirect" title="Genetic engineering in Oceania">Oceania</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Similar fields</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Eugenics" title="Eugenics">Eugenics</a></li> <li><a href="/wiki/Cloning" title="Cloning">Cloning</a></li> <li><a href="/wiki/Stem_cell" title="Stem cell">Stem cell research</a></li> <li><a href="/wiki/Synthetic_biology" title="Synthetic biology">Synthetic biology</a></li></ul> </div></td></tr><tr><td class="navbox-abovebelow" colspan="2"><div> <ul><li><a href="/wiki/Biology" title="Biology">Biology</a></li> <li><a href="/wiki/Genetics" title="Genetics">Genetics</a></li> <li><a href="/wiki/Biotechnology" title="Biotechnology">Biotechnology</a></li> <li><a href="/wiki/Bioethics" title="Bioethics">Bioethics</a></li></ul> </div></td></tr></tbody></table></div> <div class="navbox-styles"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236075235"></div><div role="navigation" class="navbox" aria-labelledby="Emerging_technologies" style="padding:3px"><table class="nowraplinks hlist mw-collapsible autocollapse navbox-inner" style="border-spacing:0;background:transparent;color:inherit"><tbody><tr><th scope="col" class="navbox-title" colspan="2" style="text-align: center;"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1239400231"><div class="navbar plainlinks hlist navbar-mini"><ul><li class="nv-view"><a href="/wiki/Template:Emerging_technologies" title="Template:Emerging technologies"><abbr title="View this template">v</abbr></a></li><li class="nv-talk"><a href="/wiki/Template_talk:Emerging_technologies" title="Template talk:Emerging technologies"><abbr title="Discuss this template">t</abbr></a></li><li class="nv-edit"><a href="/wiki/Special:EditPage/Template:Emerging_technologies" title="Special:EditPage/Template:Emerging technologies"><abbr title="Edit this template">e</abbr></a></li></ul></div><div id="Emerging_technologies" style="font-size:114%;margin:0 4em"><a href="/wiki/Emerging_technologies" title="Emerging technologies">Emerging technologies</a></div></th></tr><tr><th scope="row" class="navbox-group" style="text-align: center;;width:1%">Fields</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%;text-align: center;"><a href="/wiki/Biomedical_technology" title="Biomedical technology">Biomedical</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/3D_bioprinting" title="3D bioprinting">3D bioprinting</a> <ul><li><a href="/wiki/Microgravity_bioprinting" title="Microgravity bioprinting">Microgravity bioprinting</a></li></ul></li> <li><a href="/wiki/Aldehyde-stabilized_cryopreservation" title="Aldehyde-stabilized cryopreservation">Aldehyde-stabilized cryopreservation</a></li> <li><a href="/wiki/Ampakine" title="Ampakine">Ampakine</a></li> <li><a href="/wiki/Artificial_organ" title="Artificial organ">Artificial organs</a> <ul><li><a href="/wiki/Organ_printing" title="Organ printing">Organ printing</a></li> <li><a href="/wiki/Artificial_womb" title="Artificial womb">Womb</a></li></ul></li> <li><a href="/wiki/Biofabrication" title="Biofabrication">Biofabrication</a></li> <li><a href="/wiki/Brain_transplant" title="Brain transplant">Brain transplant</a></li> <li><a href="/wiki/De-extinction" title="De-extinction">De-extinction</a></li> <li><a href="/wiki/Generative_biology" class="mw-redirect" title="Generative biology">Generative biology</a></li> <li><a href="/wiki/Genetic_engineering" title="Genetic engineering">Genetic engineering</a> <ul><li><a class="mw-selflink selflink">Gene therapy</a></li></ul></li> <li><a href="/wiki/Head_transplant" title="Head transplant">Head transplant</a></li> <li><a href="/wiki/Isolated_brain" title="Isolated brain">Isolated brain</a></li> <li><a href="/wiki/Life_extension" title="Life extension">Life extension</a> <ul><li><a href="/wiki/Strategies_for_engineered_negligible_senescence" title="Strategies for engineered negligible senescence">Strategies for engineered negligible senescence</a></li></ul></li> <li><a href="/wiki/Nanomedicine" title="Nanomedicine">Nanomedicine</a></li> <li><a href="/wiki/Nanosensor" title="Nanosensor">Nanosensors</a></li> <li><a href="/wiki/Organ_culture" title="Organ culture">Organ culture</a> <ul><li><a href="/wiki/Engineered_uterus" title="Engineered uterus">engineered uterus</a></li></ul></li> <li><a href="/wiki/Personalized_medicine" title="Personalized medicine">Personalized medicine</a></li> <li><a href="/wiki/Regenerative_medicine" title="Regenerative medicine">Regenerative medicine</a> <ul><li><a href="/wiki/Stem-cell_therapy" title="Stem-cell therapy">Stem-cell therapy</a></li> <li><a href="/wiki/Tissue_engineering" title="Tissue engineering">Tissue engineering</a></li></ul></li> <li><a href="/wiki/Robot-assisted_surgery" title="Robot-assisted surgery">Robot-assisted surgery</a></li> <li><a href="/wiki/Relational_biology" class="mw-redirect" title="Relational biology">Relational biology</a></li> <li><a href="/wiki/Synthetic_biology" title="Synthetic biology">Synthetic biology</a> <ul><li><a href="/wiki/Synthetic_genomics" title="Synthetic genomics">Synthetic genomics</a></li></ul></li> <li><a href="/wiki/Medical_tricorder" title="Medical tricorder">Tricorder</a></li> <li><a href="/wiki/Virotherapy" title="Virotherapy">Virotherapy</a> <ul><li><a href="/wiki/Oncolytic_virus" title="Oncolytic virus">Oncolytic virus</a></li></ul></li> <li><a href="/wiki/Whole_genome_sequencing" title="Whole genome sequencing">Whole genome sequencing</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="text-align: center;;width:1%">Topics</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Automation" title="Automation">Automation</a></li> <li><a href="/wiki/Collingridge_dilemma" title="Collingridge dilemma">Collingridge dilemma</a></li> <li><a href="/wiki/Differential_technological_development" title="Differential technological development">Differential technological development</a></li> <li><a href="/wiki/Disruptive_innovation" title="Disruptive innovation">Disruptive innovation</a></li> <li><a href="/wiki/Ephemeralization" title="Ephemeralization">Ephemeralization</a></li> <li><a href="/wiki/Ethics_of_technology" title="Ethics of technology">Ethics</a> <ul><li><a href="/wiki/Bioethics" title="Bioethics">Bioethics</a></li> <li><a href="/wiki/Cyberethics" title="Cyberethics">Cyberethics</a></li> <li><a href="/wiki/Neuroethics" title="Neuroethics">Neuroethics</a></li> <li><a href="/wiki/Robot_ethics" title="Robot ethics">Robot ethics</a></li></ul></li> <li><a href="/wiki/Exploratory_engineering" title="Exploratory engineering">Exploratory engineering</a></li> <li><a href="/wiki/Proactionary_principle" title="Proactionary principle">Proactionary principle</a></li> <li><a href="/wiki/Technological_change" title="Technological change">Technological change</a> <ul><li><a href="/wiki/Technological_unemployment" title="Technological unemployment">Technological unemployment</a></li></ul></li> <li><a href="/wiki/Technological_convergence" title="Technological convergence">Technological convergence</a></li> <li><a href="/wiki/Technological_evolution" title="Technological evolution">Technological evolution</a></li> <li><a href="/wiki/Technological_paradigm" title="Technological paradigm">Technological paradigm</a></li> <li><a href="/wiki/Technology_forecasting" title="Technology forecasting">Technology forecasting</a> <ul><li><a href="/wiki/Accelerating_change" title="Accelerating change">Accelerating change</a></li> <li><a href="/wiki/Future-oriented_technology_analysis" title="Future-oriented technology analysis">Future-oriented technology analysis</a></li> <li><a href="/wiki/Horizon_scanning" title="Horizon scanning">Horizon scanning</a></li> <li><a href="/wiki/Moore%27s_law" title="Moore's law">Moore's law</a></li> <li><a href="/wiki/Technological_singularity" title="Technological singularity">Technological singularity</a></li> <li><a href="/wiki/Technology_scouting" title="Technology scouting">Technology scouting</a></li></ul></li> <li><a href="/wiki/Technology_in_science_fiction" title="Technology in science fiction">Technology in science fiction</a></li> <li><a href="/wiki/Technology_readiness_level" title="Technology readiness level">Technology readiness level</a></li> <li><a href="/wiki/Technology_roadmap" title="Technology roadmap">Technology roadmap</a></li> <li><a href="/wiki/Transhumanism" title="Transhumanism">Transhumanism</a></li></ul> </div></td></tr><tr><td class="navbox-abovebelow" colspan="2" style="text-align: center;"><div> <ul><li><img alt="" src="//upload.wikimedia.org/wikipedia/en/thumb/d/db/Symbol_list_class.svg/16px-Symbol_list_class.svg.png" decoding="async" width="16" height="16" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/d/db/Symbol_list_class.svg/23px-Symbol_list_class.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/d/db/Symbol_list_class.svg/31px-Symbol_list_class.svg.png 2x" data-file-width="180" data-file-height="185" /> <a href="/wiki/List_of_emerging_technologies" title="List of emerging technologies">List</a></li></ul> </div></td></tr></tbody></table></div> <div class="navbox-styles"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236075235"></div><div role="navigation" class="navbox" aria-labelledby="Biotechnology" style="padding:3px"><table class="nowraplinks hlist mw-collapsible autocollapse navbox-inner" style="border-spacing:0;background:transparent;color:inherit"><tbody><tr><th scope="col" class="navbox-title" colspan="3"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1239400231"><div class="navbar plainlinks hlist navbar-mini"><ul><li class="nv-view"><a href="/wiki/Template:Biotechnology" title="Template:Biotechnology"><abbr title="View this template">v</abbr></a></li><li class="nv-talk"><a href="/wiki/Template_talk:Biotechnology" title="Template talk:Biotechnology"><abbr title="Discuss this template">t</abbr></a></li><li class="nv-edit"><a href="/wiki/Special:EditPage/Template:Biotechnology" title="Special:EditPage/Template:Biotechnology"><abbr title="Edit this template">e</abbr></a></li></ul></div><div id="Biotechnology" style="font-size:114%;margin:0 4em"><a href="/wiki/Biotechnology" title="Biotechnology">Biotechnology</a></div></th></tr><tr><th scope="row" class="navbox-group" style="width:1%">History</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/History_of_biotechnology" title="History of biotechnology">History of biotechnology</a></li> <li><a href="/wiki/Timeline_of_biotechnology" title="Timeline of biotechnology">Timeline of biotechnology</a></li> <li><a href="/wiki/Competitions_and_prizes_in_biotechnology" title="Competitions and prizes in biotechnology">Competitions and prizes in biotechnology</a></li></ul> </div></td><td class="noviewer navbox-image" rowspan="8" style="width:1px;padding:0 0 0 2px"><div><a href="/wiki/File:DNA_replication_split.svg" class="mw-file-description"><img src="//upload.wikimedia.org/wikipedia/commons/thumb/7/70/DNA_replication_split.svg/50px-DNA_replication_split.svg.png" decoding="async" width="50" height="101" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/commons/thumb/7/70/DNA_replication_split.svg/75px-DNA_replication_split.svg.png 1.5x, //upload.wikimedia.org/wikipedia/commons/thumb/7/70/DNA_replication_split.svg/100px-DNA_replication_split.svg.png 2x" data-file-width="315" data-file-height="635" /></a></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Branches</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Colors_of_biotechnology" class="mw-redirect" title="Colors of biotechnology">Colors of biotechnology</a></li> <li><a href="/wiki/Industrial_biotechnology" class="mw-redirect" title="Industrial biotechnology">Industrial biotechnology</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Biological concepts</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Allele" title="Allele">Allele</a></li> <li><a href="/wiki/Cell_(biology)" title="Cell (biology)">Cell</a></li> <li><a href="/wiki/DNA" title="DNA">DNA</a>/<a href="/wiki/RNA" title="RNA">RNA</a></li> <li><a href="/wiki/Fermentation" title="Fermentation">Fermentation</a></li> <li><a href="/wiki/Gene" title="Gene">Gene</a></li> <li><a href="/wiki/Plasmid" title="Plasmid">Plasmid</a></li> <li><a href="/wiki/Protein" title="Protein">Protein</a></li> <li><a href="/wiki/Selective_breeding" title="Selective breeding">Selective breeding</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">General concepts</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Biotechnology_industrial_park" class="mw-redirect" title="Biotechnology industrial park">Biotechnology industrial park</a></li> <li><a href="/wiki/Biotechnology_products" class="mw-redirect" title="Biotechnology products">Biotechnology products</a></li> <li><a href="/wiki/Biotechnology_law" class="mw-redirect" title="Biotechnology law">Biotechnology law</a></li> <li><a href="/wiki/Green_Revolution" title="Green Revolution">Green Revolution</a></li> <li><a href="/wiki/Human_Genome_Project" title="Human Genome Project">Human Genome Project</a></li> <li><a href="/wiki/Pharmaceutical_company" class="mw-redirect" title="Pharmaceutical company">Pharmaceutical company</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Basic techniques and tools</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%">Biology field</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Bioreactor" title="Bioreactor">Bioreactor</a></li> <li><a href="/wiki/Cell_culture" title="Cell culture">Cell culture</a></li> <li><a href="/wiki/Cultured_meat" title="Cultured meat">Cultured meat</a></li> <li><a href="/wiki/Flow_cytometry" title="Flow cytometry">Flow cytometry</a></li> <li><a href="/wiki/Hybridoma_technology" title="Hybridoma technology">Hybridoma technology</a></li> <li><a href="/wiki/High-performance_liquid_chromatography" title="High-performance liquid chromatography">HPLC</a></li> <li><a href="/wiki/Nuclear_magnetic_resonance" title="Nuclear magnetic resonance">NMR</a></li> <li><a href="/wiki/Spectroscopy" title="Spectroscopy">Spectroscopy</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Chemical field</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Centrifugation" title="Centrifugation">Centrifugation</a></li> <li><a href="/wiki/Continuous_stirred-tank_reactor" title="Continuous stirred-tank reactor">CSTR</a></li> <li><a href="/wiki/Crystallization" title="Crystallization">Crystallization</a></li> <li><a href="/wiki/Chromatography" title="Chromatography">Chromatography</a></li> <li><a href="/wiki/Kidney_dialysis" title="Kidney dialysis">Kidney dialysis</a></li> <li><a href="/wiki/Electrophoresis" title="Electrophoresis">Electrophoresis</a></li> <li><a href="/wiki/Extraction_(chemistry)" title="Extraction (chemistry)">Extraction</a></li> <li><a href="/wiki/Fed-batch_culture" title="Fed-batch culture">Fed Batch</a></li> <li><a href="/wiki/Filtration" title="Filtration">Filtration</a></li> <li><a href="/wiki/Plug_flow_reactor_model" title="Plug flow reactor model">PFR</a></li> <li><a href="/wiki/Sedimentation" title="Sedimentation">Sedimentation</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Applications</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Animal_cell_culture" class="mw-redirect" title="Animal cell culture">Animal cell culture</a></li> <li><a href="/wiki/Biofabrication" title="Biofabrication">Biofabrication</a></li> <li><a href="/wiki/Bioinformatics" title="Bioinformatics">Bioinformatics</a></li> <li><a href="/wiki/Biosynthesis" title="Biosynthesis">Biosynthesis</a></li> <li><a href="/wiki/Bionic_architecture" title="Bionic architecture">Bionic architecture</a></li> <li><a href="/wiki/Cell_immunity" class="mw-redirect" title="Cell immunity">Cell immunity</a></li> <li><a href="/wiki/Cloning" title="Cloning">Cloning</a> <ul><li><a href="/wiki/Reproductive_cloning" class="mw-redirect" title="Reproductive cloning">Reproductive cloning</a></li> <li><a href="/wiki/Therapeutic_cloning" class="mw-redirect" title="Therapeutic cloning">Therapeutic cloning</a></li></ul></li> <li><a href="/wiki/Embryology" title="Embryology">Embryology</a></li> <li><a href="/wiki/Environmental_biotechnology" title="Environmental biotechnology">Environmental biotechnology</a></li> <li><a href="/wiki/Genetic_engineering" title="Genetic engineering">Genetic engineering</a> <ul><li><a href="/wiki/Genetically_modified_organism" title="Genetically modified organism">Genetically modified organism</a></li> <li><a href="/wiki/Molecular_genetics" title="Molecular genetics">Molecular genetics</a></li></ul></li> <li><a class="mw-selflink selflink">Gene therapy</a></li> <li><a href="/wiki/Microbial_biodegradation" title="Microbial biodegradation">Microbial biodegradation</a></li> <li><a href="/wiki/Omics" title="Omics">Omics</a></li> <li><a href="/wiki/Pharmacogenomics" title="Pharmacogenomics">Pharmacogenomics</a></li> <li><a href="/wiki/Stem_cells" class="mw-redirect" title="Stem cells">Stem cells</a></li> <li><a href="/wiki/Telomere" title="Telomere">Telomere</a></li> <li><a href="/wiki/Tissue_culture" title="Tissue culture">Tissue culture</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Interdisciplinary fields</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Biobased_economy" class="mw-redirect" title="Biobased economy">Bioeconomy</a></li> <li><a href="/wiki/Bioelectronics" title="Bioelectronics">Bioelectronics</a></li> <li><a href="/wiki/Biological_engineering" title="Biological engineering">Bioengineering</a></li> <li><a href="/wiki/Biology" title="Biology">Biology</a></li> <li><a href="/wiki/Biopharmaceutical" title="Biopharmaceutical">Biopharmacology</a></li> <li><a href="/wiki/Biomedical_engineering" title="Biomedical engineering">Biomedical engineering</a></li> <li><a href="/wiki/Biomedicine" title="Biomedicine">Biomedicine</a></li> <li><a href="/wiki/Biomimetics" title="Biomimetics">Biomimetics</a></li> <li><a href="/wiki/Biochemistry" title="Biochemistry">Biochemicals</a></li> <li><a href="/wiki/Biorobotics" title="Biorobotics">Biorobotics</a></li> <li><a href="/wiki/Chemical_engineering" title="Chemical engineering">Chemical engineering</a></li> <li><a href="/wiki/Microbiology" title="Microbiology">Microbiology</a></li> <li><a href="/wiki/Mining" title="Mining">Mining</a></li> <li><a href="/wiki/Molecular_biology" title="Molecular biology">Molecular biology</a></li> <li><a href="/wiki/Nanobiotechnology" title="Nanobiotechnology">Nanobiotechnology</a></li> <li><a href="/wiki/Virology" title="Virology">Virology</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Lists</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Index_of_biotechnology_articles" title="Index of biotechnology articles">Index of biotechnology articles</a></li> <li><a href="/wiki/List_of_biotechnology_articles" class="mw-redirect" title="List of biotechnology articles">List of biotechnology articles</a></li> <li><a href="/wiki/List_of_largest_biomedical_companies_by_market_capitalization" title="List of largest biomedical companies by market capitalization">List of largest biomedical companies by market capitalization</a></li></ul> </div></td></tr><tr><td class="navbox-abovebelow" colspan="3"><div> <ul><li><img alt="" src="//upload.wikimedia.org/wikipedia/en/thumb/9/96/Symbol_category_class.svg/16px-Symbol_category_class.svg.png" decoding="async" width="16" height="16" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/9/96/Symbol_category_class.svg/23px-Symbol_category_class.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/9/96/Symbol_category_class.svg/31px-Symbol_category_class.svg.png 2x" data-file-width="180" data-file-height="185" /> <a href="/wiki/Category:Biotechnology" title="Category:Biotechnology">Category</a></li> <li><img alt="" src="//upload.wikimedia.org/wikipedia/en/thumb/4/4a/Commons-logo.svg/12px-Commons-logo.svg.png" decoding="async" width="12" height="16" class="mw-file-element" srcset="//upload.wikimedia.org/wikipedia/en/thumb/4/4a/Commons-logo.svg/18px-Commons-logo.svg.png 1.5x, //upload.wikimedia.org/wikipedia/en/thumb/4/4a/Commons-logo.svg/24px-Commons-logo.svg.png 2x" data-file-width="1024" data-file-height="1376" /> <a href="https://commons.wikimedia.org/wiki/Category:Biotechnology" class="extiw" title="commons:Category:Biotechnology">Commons</a></li></ul> </div></td></tr></tbody></table></div> <div class="navbox-styles"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1236075235"></div><div role="navigation" class="navbox" aria-labelledby="Longevity" style="padding:3px"><table class="nowraplinks hlist mw-collapsible autocollapse navbox-inner" style="border-spacing:0;background:transparent;color:inherit"><tbody><tr><th scope="col" class="navbox-title" colspan="2"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1129693374"><link rel="mw-deduplicated-inline-style" href="mw-data:TemplateStyles:r1239400231"><div class="navbar plainlinks hlist navbar-mini"><ul><li class="nv-view"><a href="/wiki/Template:Longevity" title="Template:Longevity"><abbr title="View this template">v</abbr></a></li><li class="nv-talk"><a href="/wiki/Template_talk:Longevity" title="Template talk:Longevity"><abbr title="Discuss this template">t</abbr></a></li><li class="nv-edit"><a href="/wiki/Special:EditPage/Template:Longevity" title="Special:EditPage/Template:Longevity"><abbr title="Edit this template">e</abbr></a></li></ul></div><div id="Longevity" style="font-size:114%;margin:0 4em"><a href="/wiki/Longevity" title="Longevity">Longevity</a></div></th></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Ageing" title="Ageing">Ageing</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Old_age" title="Old age">Old age</a></li> <li><a href="/wiki/Longevity_myths" title="Longevity myths">Longevity myths</a></li> <li><a href="/wiki/Senescence" title="Senescence">Senescence</a> <ul><li><a href="/wiki/Aging-associated_diseases" title="Aging-associated diseases">aging-associated diseases</a></li> <li><a href="/wiki/Degenerative_disease" title="Degenerative disease">degenerative diseases</a></li> <li><a href="/wiki/Negligible_senescence" title="Negligible senescence">negligible senescence</a></li></ul></li> <li><a href="/wiki/Gerontology" title="Gerontology">Gerontology</a> <ul><li><a href="/wiki/Biogerontology" title="Biogerontology">biogerontology</a></li> <li><a href="/wiki/Cognitive_epidemiology" title="Cognitive epidemiology">cognitive epidemiology</a></li></ul></li> <li><a href="/wiki/Centenarian" title="Centenarian">Centenarian</a> <ul><li><a href="/wiki/Supercentenarian" title="Supercentenarian">supercentenarian</a></li> <li><a href="/wiki/Research_into_centenarians" title="Research into centenarians">research into centenarians</a></li></ul></li> <li><a href="/wiki/Life_expectancy" title="Life expectancy">Life expectancy</a> <ul><li><a href="/wiki/LGBT_life_expectancy" title="LGBT life expectancy">LGBT life expectancy</a></li></ul></li> <li><a href="/wiki/Maximum_life_span" title="Maximum life span">Maximum life span</a></li> <li><a href="/wiki/Biomarkers_of_aging" title="Biomarkers of aging">Biomarkers of aging</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Life_extension" title="Life extension">Life extension</a></th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Outline_of_life_extension" title="Outline of life extension">Topic outline</a> <ul><li><a href="/wiki/Index_of_topics_related_to_life_extension" title="Index of topics related to life extension">index</a></li></ul></li> <li><a href="/wiki/Anti-aging_movement" title="Anti-aging movement">Anti-aging movement</a></li> <li><a href="/wiki/Biodemography_of_human_longevity" title="Biodemography of human longevity">Biodemography of human longevity</a></li> <li><a href="/wiki/Life_extension" title="Life extension">Indefinite lifespan</a></li> <li><a href="/wiki/Longevity_escape_velocity" title="Longevity escape velocity">Longevity escape velocity</a></li> <li>Methods <ul><li><a href="/wiki/Calorie_restriction" title="Calorie restriction">calorie restriction</a></li> <li><a href="/wiki/Diet_and_longevity" title="Diet and longevity">Diet and longevity</a></li> <li><a class="mw-selflink selflink">gene therapy</a></li> <li><a href="/wiki/Nanomedicine" title="Nanomedicine">nanomedicine</a></li> <li><a href="/wiki/Organ_printing" title="Organ printing">organ printing</a></li> <li><a href="/wiki/Rejuvenation" title="Rejuvenation">rejuvenation</a></li> <li><a href="/wiki/Stem-cell_therapy" title="Stem-cell therapy">stem-cell therapy</a></li> <li><a href="/wiki/Strategies_for_engineered_negligible_senescence" title="Strategies for engineered negligible senescence">SENS</a></li></ul></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Lists of <a href="/wiki/Life_expectancy" title="Life expectancy">life expectancy</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"></div><table class="nowraplinks navbox-subgroup" style="border-spacing:0"><tbody><tr><th scope="row" class="navbox-group" style="width:1%">by country</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/List_of_countries_by_life_expectancy" title="List of countries by life expectancy">World</a></li> <li><a href="/wiki/List_of_African_countries_by_life_expectancy" title="List of African countries by life expectancy">Africa</a></li> <li><a href="/wiki/List_of_countries_in_the_Americas_by_life_expectancy" title="List of countries in the Americas by life expectancy">Americas</a> <ul><li><a href="/wiki/List_of_North_American_countries_by_life_expectancy" title="List of North American countries by life expectancy">North America</a></li> <li><a href="/wiki/List_of_South_American_countries_by_life_expectancy" title="List of South American countries by life expectancy">South America</a></li></ul></li> <li><a href="/wiki/List_of_Asian_countries_by_life_expectancy" title="List of Asian countries by life expectancy">Asia</a></li> <li><a href="/wiki/List_of_European_countries_by_life_expectancy" title="List of European countries by life expectancy">Europe</a></li> <li><a href="/wiki/List_of_Oceanian_countries_by_life_expectancy" title="List of Oceanian countries by life expectancy">Oceania</a></li> <li><a href="/wiki/List_of_world_regions_by_life_expectancy" title="List of world regions by life expectancy">World macroregions</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">regions of countries</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/List_of_Argentine_provinces_and_territories_by_life_expectancy" title="List of Argentine provinces and territories by life expectancy">Argentina</a></li> <li><a href="/wiki/List_of_Australian_states_by_life_expectancy" title="List of Australian states by life expectancy">Australia</a></li> <li><a href="/wiki/List_of_Belgian_provinces_by_life_expectancy" title="List of Belgian provinces by life expectancy">Belgium</a></li> <li><a href="/wiki/List_of_Brazilian_states_by_life_expectancy" title="List of Brazilian states by life expectancy">Brazil</a></li> <li><a href="/wiki/List_of_Canadian_provinces_and_territories_by_life_expectancy" title="List of Canadian provinces and territories by life expectancy">Canada</a></li> <li><a href="/wiki/List_of_Chinese_administrative_divisions_by_life_expectancy" title="List of Chinese administrative divisions by life expectancy">China</a> <ul><li><a href="/wiki/List_of_cities_in_China_by_life_expectancy" title="List of cities in China by life expectancy">Cities</a></li></ul></li> <li><a href="/wiki/List_of_French_departments_by_life_expectancy" title="List of French departments by life expectancy">France</a></li> <li><a href="/wiki/List_of_Finnish_regions_by_life_expectancy" title="List of Finnish regions by life expectancy">Finland</a></li> <li><a href="/wiki/List_of_German_states_by_life_expectancy" title="List of German states by life expectancy">Germany</a></li> <li><a href="/wiki/List_of_Greek_regions_by_life_expectancy" title="List of Greek regions by life expectancy">Greece</a></li> <li><a href="/wiki/List_of_Indian_states_by_life_expectancy_at_birth" title="List of Indian states by life expectancy at birth">India</a></li> <li><a href="/wiki/List_of_regions_of_Israel_and_Palestine_by_life_expectancy" title="List of regions of Israel and Palestine by life expectancy">Israel</a></li> <li><a href="/wiki/List_of_Italian_provinces_by_life_expectancy" title="List of Italian provinces by life expectancy">Italy</a></li> <li><a href="/wiki/List_of_Japanese_prefectures_by_life_expectancy" title="List of Japanese prefectures by life expectancy">Japan</a></li> <li><a href="/wiki/List_of_regions_of_Kazakhstan_by_life_expectancy" title="List of regions of Kazakhstan by life expectancy">Kazakhstan</a></li> <li><a href="/wiki/List_of_Mexican_states_by_life_expectancy" title="List of Mexican states by life expectancy">Mexico</a></li> <li><a href="/wiki/List_of_regions_of_Israel_and_Palestine_by_life_expectancy" title="List of regions of Israel and Palestine by life expectancy">Palestine</a></li> <li><a href="/wiki/List_of_federal_subjects_of_Russia_by_life_expectancy" title="List of federal subjects of Russia by life expectancy">Russia</a></li> <li><a href="/wiki/List_of_South_African_provinces_by_life_expectancy" title="List of South African provinces by life expectancy">South Africa</a></li> <li><a href="/wiki/List_of_Spanish_provinces_by_life_expectancy" title="List of Spanish provinces by life expectancy">Spain</a></li> <li><a href="/wiki/List_of_Swedish_counties_by_life_expectancy" title="List of Swedish counties by life expectancy">Sweden</a></li> <li><a href="/wiki/List_of_Tajikistani_regions_by_life_expectancy" title="List of Tajikistani regions by life expectancy">Tajikistan</a></li> <li><a href="/wiki/List_of_Turkish_provinces_by_life_expectancy" title="List of Turkish provinces by life expectancy">Turkey</a></li> <li><a href="/wiki/List_of_British_regions_by_life_expectancy" title="List of British regions by life expectancy">UK</a></li> <li><a href="/wiki/List_of_U.S._states_and_territories_by_life_expectancy" title="List of U.S. states and territories by life expectancy">USA</a> <ul><li><a href="/wiki/List_of_U.S._counties_with_shortest_life_expectancy" title="List of U.S. counties with shortest life expectancy">shortest</a></li> <li><a href="/wiki/List_of_U.S._counties_with_longest_life_expectancy" title="List of U.S. counties with longest life expectancy">longest</a></li></ul></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">regions by continents</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/List_of_African_regions_by_life_expectancy" title="List of African regions by life expectancy">Africa</a></li> <li><a href="/wiki/List_of_Asian_regions_by_life_expectancy" title="List of Asian regions by life expectancy">Asia</a></li> <li><a href="/wiki/List_of_European_regions_by_life_expectancy" title="List of European regions by life expectancy">Europe</a></li> <li><a href="/wiki/List_of_Oceanian_regions_by_life_expectancy" title="List of Oceanian regions by life expectancy">Oceania</a></li> <li><a href="/wiki/List_of_South_American_regions_by_life_expectancy" title="List of South American regions by life expectancy">South America</a></li></ul> </div></td></tr></tbody></table><div></div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Records</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Longevity_claims" title="Longevity claims">Longevity claims</a></li> <li><a href="/wiki/Oldest_people" title="Oldest people">Oldest people</a> <ul><li><a href="/wiki/List_of_the_verified_oldest_people" title="List of the verified oldest people">verified</a></li> <li><a href="/wiki/List_of_the_oldest_people_by_country" title="List of the oldest people by country">by country</a></li> <li><a href="/wiki/List_of_oldest_living_people" title="List of oldest living people">living</a></li></ul></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%"><a href="/wiki/Immortality" title="Immortality">Immortality</a></th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Biological_immortality" title="Biological immortality">Biological immortality</a> <ul><li><a href="/wiki/Regeneration_(biology)" title="Regeneration (biology)">Regeneration</a></li></ul></li> <li><a href="/wiki/Digital_immortality" title="Digital immortality">Digital immortality</a></li> <li><a href="/wiki/Eternal_youth" title="Eternal youth">Eternal youth</a></li> <li><a href="/wiki/Immortality_in_fiction" title="Immortality in fiction">Immortality in fiction</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Longevity genes</th><td class="navbox-list-with-group navbox-list navbox-even" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/FOXO3" title="FOXO3">FOXO3</a></li> <li><a href="/wiki/Apolipoprotein_E" title="Apolipoprotein E">APOE</a></li> <li><a href="/wiki/Cholesteryl_ester_transfer_protein" title="Cholesteryl ester transfer protein">CETP</a></li> <li><a href="/wiki/Angiotensin-converting_enzyme" title="Angiotensin-converting enzyme">ACE</a></li> <li><a href="/wiki/Interleukin_6" title="Interleukin 6">IL6</a></li> <li><a href="/wiki/Klotho_(biology)" title="Klotho (biology)">Klotho</a></li> <li><a href="/wiki/Sirtuin_1" title="Sirtuin 1">SIRT1</a></li> <li><a href="/wiki/Tumor_necrosis_factor" title="Tumor necrosis factor">TNF</a></li></ul> </div></td></tr><tr><th scope="row" class="navbox-group" style="width:1%">Related</th><td class="navbox-list-with-group navbox-list navbox-odd" style="width:100%;padding:0"><div style="padding:0 0.25em"> <ul><li><a href="/wiki/Blue_zone" title="Blue zone">Blue zone</a></li> <li><a href="/wiki/Longevity_insurance" title="Longevity insurance">Longevity insurance</a></li> <li><a href="/wiki/Longevity_risk" title="Longevity risk">Longevity risk</a></li> <li><a href="/wiki/Transhumanist_politics" title="Transhumanist politics">Transhumanist politics</a></li> <li><a href="/wiki/List_of_longest-living_organisms" title="List of longest-living organisms">Longest-living organisms</a></li></ul> </div></td></tr></tbody></table></div> <style data-mw-deduplicate="TemplateStyles:r1130092004">.mw-parser-output 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