Basic Process of Gene Therapy

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It must be delivered to the cell using a carrier, or <a href="/glossary-of-gene-therapy-terms.html#vector">vector</a>. Vector systems can be divided into: <table> <tbody> <tr> <td style="background-image: url('/images/virus1.gif'); width: 20px; background-repeat: no-repeat; background-position: 50% 5%;"> </td> <td><a href="/viral-vectors.html">Viral Vectors</a></td> </tr> <tr> <td style="background-image: url('/images/virus1.gif'); width: 20px; background-repeat: no-repeat; background-position: 50% 5%;"> </td> <td><a href="/non-viral-vectors.html">Non-viral Vectors</a></td> </tr> </tbody> </table> <p> </p> <p>Currently, the most common type of vectors are <a href="/glossary-of-gene-therapy-terms.html#virus">viruses</a> that have been genetically altered to carry normal human <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a> (see also <a href="https://www.abedia.com/wiley/" target="_blank">Wiley database on vectors used in gene therapy trials</a>). Viruses have evolved a way of encapsulating and delivering their genes to human cells in a pathogenic manner. Scientists have tried to harness this ability by manipulating the viral genome to remove disease-causing genes and insert therapeutic ones (see also video 2).</p> <p>Target cells such as the patient's liver or lung cells are infected with the vector. The vector then unloads its genetic material containing the therapeutic human gene into the target cell. The generation of a functional protein product from the therapeutic gene restores the target cell to a normal state.</p> <table width="100%" border="0"> <tr> <td width="48%"> <div align="left"> <object width="250"> <param name="movie" value="https://www.youtube.com/v/rrT5BT_7HdI&hl=nl&fs=1&rel=0&border=1"> <param name="allowFullScreen" value="true"> <embed src="https://www.youtube.com/v/rrT5BT_7HdI&hl=nl&fs=1&" type="application/x-shockwave-flash" allowfullscreen="true" width="250"></embed> </object> </div></td> <td width="52%"> <div align="left"> <object width="250"> <param name="movie" value="https://www.youtube.com/v/x2jUMG2E-ic&hl=nl&fs=1&rel=0&border=1"> </param> <param name="allowFullScreen" value="true"> </param> <embed src="https://www.youtube.com/v/x2jUMG2E-ic&hl=nl&fs=1&" type="application/x-shockwave-flash" allowfullscreen="true" width="250"></embed> </object> </div></td> </tr> <tr> <td><div align="left"><em>Video 1: Lecture, What is Gene Therapy<br /> (<a href="https://www.youtube.com/watch?v=rrT5BT_7HdI" target="_blank">YouTube</a>, 10:01)</em></div></td> <td><div align="left"><em>Video 2: What is Recombinant DNA<br />(<a href="https://www.youtube.com/watch?v=x2jUMG2E-ic" target="_blank">YouTube</a>, 4:35)</em></div></td> </tr> </table> <center><br /> <div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <a href="/component/banners/click/143.html" target="_blank" rel="noopener noreferrer" title="6thCellTheraAnalyDevelSummit-dec2024"> <img src="https://www.genetherapynet.com/images/banners/6thCellTheraAnalyDevelSummit-dec2024.jpg#joomlaImage://local-images/banners/6thCellTheraAnalyDevelSummit-dec2024.jpg?width=650&height=83" alt="6thCellTheraAnalyDevelSummit-dec2024" > </a> </div> </div> </div> </center> <div align="right"><a href="#top"><font size="1">^ Back to Top</font></a> </div></div> <div class="uk-margin-top"><a href="/basic-process.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-32"> <h3> What is Gene Therapy </h3> <ul class="uk-nav uk-nav-default"> <li class="item-26 uk-active"><a href="/basic-process.html"> Basic Process</a></li> <li class="item-27"><a href="/types-of-gene-therapy.html"> Types of gene therapy</a></li> <li class="item-28"><a href="/gene-doping.html"> Gene Doping</a></li> <li class="item-29 uk-parent"><a href="/viral-vectors.html"> Viral Vectors</a> <ul class="uk-nav-sub"> <li class="item-30"><a href="/viral-vector/adenoviruses.html"> Adenoviruses</a></li> <li class="item-31"><a href="/viral-vector/adeno-associated-viruses.html"> Adeno-associated viruses</a></li> <li class="item-32"><a href="/viral-vector/alphaviruses.html"> Alphaviruses</a></li> <li class="item-33"><a href="/viral-vector/herpesviruses.html"> Herpesviruses</a></li> <li class="item-34"><a href="/viral-vector/lentiviruses.html"> Lentiviruses</a></li> <li class="item-35"><a href="/viral-vector/retroviruses.html"> Retroviruses</a></li> <li class="item-36"><a href="/viral-vector/vaccinia-viruses.html"> Vaccinia viruses</a></li> <li class="item-132"><a href="/viral-vector/risk-factors.html"> Risk Factors</a></li> <li class="item-133"><a href="/viral-vector/pseudotyping-of-viral-vectors.html"> Pseudotyping of Viral Vectors</a></li></ul></li> <li class="item-37 uk-parent"><a href="/non-viral-vectors.html"> Non-viral Vectors</a> <ul class="uk-nav-sub"> <li class="item-129"><a href="/non-viral-vectors/naked-dna.html"> Naked DNA</a></li> <li class="item-130"><a href="/non-viral-vectors/oligonucleotides.html"> Oligonucleotides</a></li> <li class="item-131"><a href="/non-viral-vectors/lipoplexes-and-polyplexes.html"> Lipoplexes and polyplexes</a></li></ul></li> <li class="item-709 uk-parent"><a href="/gene-editing-tools.html"> Gene Editing Tools</a> <ul class="uk-nav-sub"> <li class="item-710"><a href="/gene-editing-tools/zinc-finger-nuclease.html"> Zinc Finger Nuclease</a></li> <li class="item-711"><a href="/gene-editing-tools/talen.html"> TALEN</a></li> <li class="item-712"><a href="/gene-editing-tools/crispr-cas9.html"> CRISPR-Cas9 </a></li></ul></li> <li class="item-38"><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" /> 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