Diseases Treated by Gene Therapy

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Three types of diseases for gene therapy can be distinguished:</p> <table> <tbody> <tr> <td style="background-image: url('/images/virus1.gif'); width: 20px; background-repeat: no-repeat; background-position: 50% 5%;">聽</td> <td>Monogenic disorders, single locus (gene) is defective and responsible for the disease, 100% heritable. Examples: <a href="/glossary-of-gene-therapy-terms.html#sicklecellanemia">Sickle cell anemia</a>, Severe Combined Immunodeficiency (<a href="/glossary-of-gene-therapy-terms.html#ada">ADA-SCID</a> / <a href="/glossary-of-gene-therapy-terms.html#x-scid">X-SCID</a>), <a href="/glossary-of-gene-therapy-terms.html#cysticfibrosis">Cystic fibrosis</a>, <a href="/glossary-of-gene-therapy-terms.html#hemophilia">Hemophilia</a>, <a href="/glossary-of-gene-therapy-terms.html#duchenne">Duchenne muscular dystrophy</a>, <a href="/glossary-of-gene-therapy-terms.html#huntington">Huntington鈥檚 disease</a>, <a href="/glossary-of-gene-therapy-terms.html#parkinson">Parkinson鈥檚</a>, Hypercholesterolemia, Alpha-1 antitrypsin, Chronic granulomatous disease, <a href="/glossary-of-gene-therapy-terms.html#fanconi">Fanconi Anemia</a> and <a href="/glossary-of-gene-therapy-terms.html#gaucher">Gaucher Disease.</a></td> </tr> <tr> <td style="background-image: url('/images/virus1.gif'); width: 20px; background-repeat: no-repeat; background-position: 50% 5%;">聽</td> <td>Polygenic disorders, multiple genes involved, disease may be dependent on environmental factors and lifestyle. Examples: Heart disease, Cancer, Diabetes, Schizophrenia and <a href="/glossary-of-gene-therapy-terms.html#alzheimer">Alzheimer鈥檚 disease.</a></td> </tr> <tr> <td style="background-image: url('/images/virus1.gif'); width: 20px; background-repeat: no-repeat; background-position: 50% 5%;">聽</td> <td>Infectious diseases, such as <a href="https://www.ncbi.nlm.nih.gov/sites/entrez?Db=pubmed&Cmd=ShowDetailView&TermToSearch=15922953&ordinalpos=1&itool=EntrezSystem2.PEntrez.Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum" target="bank">HIV</a>.</td> </tr> </tbody> </table> <p><br />Click here for an <a href="/clinicaltrialsgov.html">overview of new gene therapy trials</a> or <a href="/search-clinical-gene-therapy-trials.html">search trials by indication</a>.</p> <center><br /><div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <a href="/component/banners/click/188.html" target="_blank" rel="noopener noreferrer" title="BioprocessingSummitEurope-mrt2025"> <img src="https://www.genetherapynet.com/images/banners/BioprocessingSummitEurope-mrt2025.jpg" alt="BioprocessingSummitEurope-mrt2025"> </a> </div> </div> </div> </center> <p>Remarkable strides have been achieved through gene therapy in the field of medical treatment, leading to extraordinary advancements in combating a wide spectrum of genetic disorders and diseases. See also <a href="https://en.wikipedia.org/wiki/Gene_therapy#Major_developments_in_gene_therapy">Major developments in gene therapy</a>, <a href="https://doi.org/10.1146/annurev.biochem.74.050304.091637">Gene Therapy: Medicine of the 21st Century</a> and <a href="#video6">Individualized Drugs & Gene Therapy (video 6)</a>. A comprehensive 20 minutes video on gene therapy: <a href="https://www.youtube.com/watch?v=U3RygvuSrok" target="_blank" rel="noopener">'Gene Therapy a new tool to cure human diseases'</a>. Some of the most notable breakthroughs encompass:</p> <p><em>Severe Combined Immune Deficiency (ADA-SCID)</em> <br />Commonly referred to as "bubble boy disease," <a href="/glossary-of-gene-therapy-terms.html#ada">ADA-SCID</a> is a condition where children are born with compromised immune systems, rendering them highly susceptible to infections. Pioneering research in Italy marked a significant milestone by achieving a form of "cure" or long-term correction for patients with this genetic disorder. This innovative approach involved introducing a therapeutic gene known as ADA into bone marrow cells within a controlled laboratory setting. Subsequently, these genetically modified cells were reintroduced into the patients, effectively restoring their immune systems. This groundbreaking procedure has enabled treated patients to lead normal lives without requiring further interventions. (see also <a href="https://ghr.nlm.nih.gov/condition/adenosine-deaminase-deficiency">Description of ADA deficiency</a>, <a href="https://history.nih.gov/exhibits/genetics/sect4.htm">ADA: The First Gene Therapy Trial</a>, from the National Institutes of Health and <a href="https://www.scid.net">SCID.net</a>)</p> <p><em>Chronic Granulomatus Disorder (CGD)</em> <br />CGD is a genetic disorder impairing the immune system's ability to combat bacterial and fungal infections, often leading to life-threatening consequences. Building upon techniques akin to the ADA-SCID trial, researchers in Germany successfully treated CGD patients. The genetically corrected immune systems of these individuals have provided robust defense against microbial infections for a minimum of two years.</p> <p><em>Hemophilia </em><br /><a href="/glossary-of-gene-therapy-terms.html#hemophilia">Hemophilia</a>, characterized by impaired blood clotting, poses the risk of severe internal and external bleeding. A clinical trial conducted in the United States aimed to address this issue by introducing therapeutic genes into patients' livers. This approach granted patients the ability to achieve normal blood clotting times. Nonetheless, the therapeutic effects were transient due to the immune system recognizing the genetically modified liver cells as foreign entities. Strategies involving immune suppression or alternative gene delivery methods are currently being explored to attain more sustainable results.</p> <p><em>Blindness (Leber's Congenital Amaurosis)</em><br />Leber's congenital amaurosis, an uncommon inherited eye disease resulting in profound vision loss or blindness from birth or infancy, has spurred pioneering gene therapy clinical trials. Research conducted at institutions like Moorfields Eye Hospital and University College London has yielded promising outcomes in restoring vision or slowing the disease's progression through gene therapy techniques. The first patient was operated upon in early 2007. <a href="/gene-therapy-news/103-gene-therapy-improves-vision-in-nearly-blind-patients.html">Researchers at Children's Hospital of Philadelphia and the University of Pennsylvania</a> have treated six young people via gene therapy. Eye Surgeon Dr. Al Maguire and gene therapy expert Dr. Jean Bennett developed the technique used by the Children's Hospital (see also <a href="#video5">video 5</a>).</p> <p><em>Cancer </em><br />Gene therapy has introduced groundbreaking approaches for cancer treatment, encompassing strategies such as s<a href="/glossary-of-gene-therapy-terms.html#suicidegene">suicide gene therapy</a>, oncolytic virotherapy, <a href="/glossary-of-gene-therapy-terms.html#antiangiogenesis">anti-angiogenesis</a>, and therapeutic gene vaccines. Cancer research constitutes a substantial portion of gene therapy trials, accounting for approximately two-thirds. Advanced-phase trials, including Phase III trials of Ad.p53 for head and neck cancer, Phase III gene vaccine trials for prostate and pancreatic cancer, and Phase I/II trials for various cancers affecting the brain, skin, liver, colon, breast, and kidney, are presently underway and conducted in <a href="/research-institutes.html">academic medical centers</a> and <a href="/companies.html">biotechnology companies</a>.</p> <p><em>Neurodegenerative Diseases</em> <br />Recent gene therapy advancements offer hope for addressing neurodegenerative disorders such as <a href="/glossary-of-gene-therapy-terms.html#parkinson">Parkinson's Disease</a> and <a href="/glossary-of-gene-therapy-terms.html#huntington">Huntington's Disease</a>. Positive results in animal models have spurred the initiation of Phase I clinical trials for these conditions, providing critical insights into gene therapy's potential in treating neurodegenerative disorders.</p> <p><em>Mesothelioma </em><br />Gene therapy research has opened avenues for effective treatments in <a href="https://mesothelioma.net" target="blanc">mesothelioma patients</a>. While specific gene therapy approaches are tailored to different cancers, preliminary studies have shown promise in treating mesothelioma. Clinical trials involving suicide genes have exhibited encouraging early results. Nevertheless, further research is needed to refine and develop effective gene therapy treatments for this condition.</p> <p><em>Cystic Fibrosis<br /></em>Cystic fibrosis, a hereditary disorder affecting the respiratory and digestive systems, leads to the production of thick and sticky mucus that obstructs airways and ducts. Gene therapy aims to deliver functional copies of the faulty CFTR gene into affected cells, enabling them to produce normal mucus and improving lung function.</p> <p><em>Beta-Thalassemia</em><br />Beta-thalassemia, a blood disorder characterized by reduced or absent hemoglobin production, causing anemia and complications, seeks to introduce functional beta-globin genes into bone marrow cells via gene therapy. This enables the production of healthy red blood cells.</p> <p><em>Sickle Cell Disease</em><br />Sickle cell disease, resulting from a mutation affecting hemoglobin, causes misshapen red blood cells prone to clumping, leading to pain and organ damage. Gene therapy aims to modify hematopoietic stem cells to produce corrected hemoglobin, preventing the formation of sickled cells.</p> <p>These groundbreaking advancements underscore the immense potential of gene therapy as a transformative tool in addressing a broad array of genetic disorders and diseases. As ongoing research continues to push the boundaries of scientific understanding, the future of gene therapy holds unparalleled promise in revolutionizing approaches to medical treatment.</p> <hr /> <p><a name="video5"></a><a name="video6"></a></p> <table border="0" width="100%"> <tbody> <tr> <td width="48%"> <div align="left"><object width="250" height="150"> <param name="movie" value="https://www.youtube.com/v/NmftOETlyyg&hl=nl&fs=1&rel=0&border=1" /> <param name="allowFullScreen" value="true" /> <embed src="https://www.youtube.com/v/NmftOETlyyg&hl=nl&fs=1&" type="application/x-shockwave-flash" allowfullscreen="allowfullscreen" width="250" /> </object></div> </td> <td width="52%"> <div align="left"><object width="250" height="150"> <param name="movie" value="https://www.youtube.com/v/pMePYwKw_o4&hl=nl&fs=1&rel=0&border=1" /> <param name="allowFullScreen" value="true" /> <embed src="https://www.youtube.com/v/pMePYwKw_o4&hl=nl&fs=1&" type="application/x-shockwave-flash" allowfullscreen="allowfullscreen" width="250" /> </object></div> </td> </tr> <tr> <td> <div align="left"><em>Video 5: Gene Therapy for blindness (<a href="https://www.youtube.com/watch?v=NmftOETlyyg" target="_blank" rel="noopener">YouTube</a>, 0:58)</em></div> </td> <td> <div align="left"><em>Video 6: Individualized Drugs & Gene Therapy (<a href="https://www.youtube.com/watch?v=pMePYwKw_o4" target="_blank" rel="noopener">YouTube</a>, 8:58)</em></div> </td> </tr> </tbody> </table> <div align="right"><a href="#top"><span style="font-size: xx-small;">^ Back to Top</span></a></div></div> <div class="uk-margin-top"><a href="/diseases-treated-by-gene-therapy.html" class="el-link uk-button uk-button-default">Read more</a></div> </div></div> </div> </div> </div> <div class="uk-width-1-2 uk-width-1-4@m uk-width-1-5@l rechts"> <div> <div class="uk-grid uk-child-width-1-1 uk-grid-row-small" uk-grid> <div> <div class="uk-panel _menu menulist" id="module-86"> <h3> Patient Information </h3> <ul class="uk-nav uk-nav-default"> <li class="item-134"><a href="/historic-overview-of-gene-therapy.html">Historic Overview of Gene Therapy</a></li> <li class="item-135"><a href="/ethical-and-social-issues-in-gene-therapy.html">Ethical and Social Issues in Gene Therapy</a></li> <li class="item-136 uk-active"><a href="/diseases-treated-by-gene-therapy.html">Diseases Treated by Gene Therapy</a></li> <li class="item-150"><a href="/clinical-gene-therapy-trial-information.html">Clinical Trials Information</a></li> <li class="item-947"><a href="/gene-therapy-products-on-the-market.html">Gene Therapy Products on the Market</a></li> <li class="item-141"><a href="/patient-societies.html">Patient Societies</a></li> <li class="item-152"><a href="/gene-doping.html">Gene Doping</a></li> <li class="item-149"><a href="/glossary-of-gene-therapy-terms.html">Glossary of Gene Therapy Terms</a></li></ul> </div> </div> <div> <div class="uk-panel" id="module-40"> <h3> Resources </h3> <div class="uk-margin-remove-last-child custom" ><ul class="resources"> <li><a href="https://www.acgtfoundation.org" target="_blank">Alliance for Cancer Gene Therapy</a></li> <li><a href="https://www.cancer.org" target="_blank">American Cancer Society</a></li> <li><a href="https://www.bio-medicine.org" target="_blank">Bio-Medicine</a></li> <li><a href="https://www.bioscience-explained.org" target="_blank">Bioscience Explained</a></li> <li><a href="https://kidshealth.org/parent/system/medical/gene_therapy.html" target="_blank">Gene Therapy and Children, KidsHealth</a></li><li><a href="https://learn.genetics.utah.edu/content/genetherapy/" target="_blank">Genetic Science Learning Center</a></li> <li><a href="https://www.ornl.gov/sci/techresources/Human_Genome/home.shtml" target="_blank">Human Genome Project Information</a></li> <li><a href="https://ictvonline.org/" target="_blank">ICTVdB, The Universal Virus Database</a></li> <li><a href="https://www.iupui.edu/~wellsctr/MMIA/htm/animations.htm" target="_blank">Molecular Medicine in Action</a></li> <li><a href="https://www.cancer.gov" target="_blank">National Cancer Institute</a></li> <li><a href="https://www.genome.gov/Education/" target="_blank">Understanding the Human Genome Project - Online education kit</a></li> <li><a href="https://www.virology.net/garryfavwebgenether.html" target="_blank">Viral Vectors and Gene Therapy, All the Virology on the www</a></li> <li><a href="https://en.wikipedia.org/wiki/Wikipedia" target="_blank">Wikipedia</a></li> <hr /> <li> <a href="https://images.google.com/images?hl=en&q=%22gene+therapy'&um=1&ie=UTF-8&sa=N&tab=wi" target="_blank">Images of gene therapy</a></li> <li><a href="https://www.google.com/search?hl=nl&q=%22gene+therapy%22+.ppt&meta=" target="_blank">PowerPoint presentations on gene therapy</a></li> <li><a href="https://nl.youtube.com/results?search_query=+%22gene+therapy%22&as=1&and_queries=&exact_query=gene+therapy&or_queries=&negative_queries=&geo_name=&geo_latlong=&search_duration=&search_hl=&search_category_type=specific&search_category=28&search_sort=&uploaded=" target="_blank">YouTube videos about gene therapy</a></li> </ul> <div align="center"> <form action="https://www.wikipedia.org/search-redirect.php" method="get" target="_blank"> <table> <tr><td nowrap="nowrap" valign="top" align="center" height="32"><img src="/images/wikipedia.gif" alt="Search Wikipedia" width="75" height="84" border="0" /> <input type="hidden" name="language" value="en"> </td></tr> <tr><td valign="top" align="center"> <input type="text" name="search" size="18" value="Wikipedia search..." style="background:#ffffff; color:#666666" onfocus="if(this.value=='Wikipedia search...'){this.value='';}" onblur="if(this.value==''){this.value='Wikipedia search...';}"> </td></tr> <tr><td valign="top" align="center"> <input type="submit" name="go" value="Search"> </td></tr></table> </form> </div></div> </div> </div></div></div> </div></div> </div> </div> </main> <footer> <style class="uk-margin-remove-adjacent">style="margin-top: -80px;"style="position: relative;"</style> <div id="footer#1" class="uk-section-muted uk-section uk-padding-remove-bottom"> <div class="uk-container"> <div class="uk-grid tm-grid-expand uk-grid-margin" uk-grid> <div class="uk-width-1-2@s uk-width-2-5@m"> <div> <div class="uk-panel" id="module-249"> <div class="uk-margin-remove-last-child custom" ><p>漏 2025 Gene Therapy Net.<br /><a href="/contact-gene-therapy-net.html" style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400'" onmouseout="this.style.color='#000000'">Contact Us</a> | <a href="/about-us.html" style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400'" onmouseout="this.style.color='#000000'">Privacy Policy</a> | <a href="/about-us.html" style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400'" onmouseout="this.style.color='#000000'">About Us</a></p></div> </div> </div> </div> <div class="uk-width-expand@s uk-width-expand@m"> <div> <div class="uk-panel" id="module-251"> <div class="uk-margin-remove-last-child custom" ><div style="text-align: right;"><p><a style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400';window.status='Bookmark Gene Therapy Net'; return true;" onmouseout="this.style.color='#000000';window.status=' '; return true;">Bookmark</a> | <a href="https://www.genetherapynet.com/" style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400';window.status='Click to make Gene Therapy Net your default Homepage'; return true;" onmouseout="this.style.color='#000000';window.status=' '; return true;" onclick="this.style.behavior='url(#default#homepage)';this.setHomePage('https://www.genetherapynet.com');">Homepage</a> | <a href="/link-to-gene-therapy-net.html" style="color: #000000; text-decoration: none;" onmouseover="this.style.color='#A1C400';window.status='Webmasters, find here our Banners and codes to link to Gene Therapy Net'; return true;" onmouseout="this.style.color='#000000';window.status=' '; return true;">Link</a></p></div></div> </div> </div> </div> <div class="uk-width-auto@s uk-width-auto@m"> <div class="uk-margin uk-text-right"><a href="#" uk-totop uk-scroll></a></div> </div></div><div id="footer#0" class="uk-grid tm-grid-expand uk-grid-row-collapse uk-child-width-1-1"> <div class="uk-width-1-1"> <div class="uk-panel uk-margin-medium uk-text-right@s uk-text-left"><a href="https://www.alseenrodelap.nl" style="color: #bbb">Webdesign by Alseenrodelap.nl</a><br/><br/></div> </div></div> </div> </div> </footer> </div> </div> </body> </html>

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