Gene Therapy Viral Vectors Explained

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Click here for more information about advertising.</a><center><hr style="margin-top: 10px;"/> <div style="font-size: 10pt;">You can also make a donation by PayPal and support the Gene Therapy Net website.</div> <form action="https://www.paypal.com/cgi-bin/webscr" method="post" target="_top"> <input type="hidden" name="cmd" value="_s-xclick"> <input type="hidden" name="hosted_button_id" value="YYU4T4HSRK4P8"> <input type="image" src="https://www.paypalobjects.com/en_US/i/btn/btn_donateCC_LG_global.gif" border="0" name="submit" alt="PayPal – The safer, easier way to pay online!"> <img alt="" border="0" src="https://www.paypalobjects.com/en_US/i/scr/pixel.gif" width="1" height="1"> </form> </center></div> </div> </div></div></div> </div> <div class="uk-width-1-2@m uk-width-3-5@l uk-flex-first"> <div class="afgeronde-artikelen uk-margin"> <div class="uk-grid uk-child-width-1-1 uk-grid-small uk-grid-match" uk-grid> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-margin-top uk-margin-remove-bottom"> Viral Vectors </h3> <div class="el-content uk-panel uk-margin-top">All <a href="/glossary-of-gene-therapy-terms.html#virus">viruses</a> attack their hosts and introduce their genetic material into the host <a href="/glossary-of-gene-therapy-terms.html#cell">cell</a> as part of their replication cycle. This genetic material contains basic 'instructions' of how to produce more copies of these viruses, hijacking the body's normal production machinery to serve the needs of the virus (see figure 1). The host cell will carry out these instructions and produce additional copies of the virus, leading to more and more cells becoming infected. Some types of viruses actually physically insert their <a href="/glossary-of-gene-therapy-terms.html#gene">genes</a> into the host's <a href="/glossary-of-gene-therapy-terms.html#genome">genome</a>. This incorporates the genes of that virus among the genes of the host cell for the life span of that cell. <p>Viruses like this could be used as vehicles to carry 'good' genes into a human cell. First, a scientist would <a href="/glossary-of-gene-therapy-terms.html#genedeletion">remove the genes</a> in the virus that cause disease. Then they would replace those genes with genes encoding the desired effect (for instance, insulin production in the case of diabetics). This procedure must be done in such a way that the genes which allow the virus to insert its genome into its host's genome are left intact.</p> <p><a href="javascript:void(0)" onclick="window.open('/understandingviruses.html','linkname','height=450, width=760,scrollbars=no')"><img border="0" alt="Click to play video in new window" src="/images/viruslifecycle.jpg" width="480" height="236" /></a><br /><br /> <em>Figure 1. How do viruses work? Recommended: Learn more about viruses by viewing a very informative video called <a href="javascript:void(0)" onclick="window.open('understandingviruses.html','linkname','height=450, width=760,scrollbars=no')">"Understanding viruses"</a> (<em>17 parts, total time 43:35, video will open a new window</em>).</em></p> <p>Many <a href="/glossary-of-gene-therapy-terms.html#genetherapy">gene therapy</a> clinical trials rely on <a href="/viral-vector/retroviruses.html">retroviruses</a> or <a href="/viral-vector/adenoviruses.html">adenoviruses</a> to deliver the desired gene. Other viruses used as vectors include <a href="/viral-vector/adeno-associated-viruses.html">adeno-associated viruses</a>, <a href="/viral-vector/lentiviruses.html">lentiviruses</a>, <a href="/viral-vector/vaccinia-viruses.html">pox viruses</a>, <a href="/viral-vector/alphaviruses.html">alphaviruses</a>, and <a href="/viral-vector/herpesviruses.html">herpes viruses</a>. These viruses differ in how well they transfer genes to the cells they recognize and are able to infect, and whether they alter the cell’s <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a> permanently or temporarily (see figure 2).</p> <p><img border="0" alt="Click to download image as jpg or powerpoint file" src="/images/virusclassification.jpg" width="480" height="374" /><br /><br /> <em>Figure 2. A comparison of different viral vectors in use for gene therapy: overview of their advantages and disadvantages. * Adeno-associated viruses are able to integrate with low frequency into chromosome 19. Lentiviruses also infect non-dividing cells. </em></p> <br /> <center><div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <a href="/component/banners/click/165.html" target="_blank" rel="noopener noreferrer" title="4thmRNA-BasedTherapSummitEurope-jan2025"> <img src="https://www.genetherapynet.com/images/banners/4thmRNA-BasedTherapSummitEurope-jan2025.png#joomlaImage://local-images/banners/4thmRNA-BasedTherapSummitEurope-jan2025.png?width=650&height=83" alt="4thmRNA-BasedTherapSummitEurope-jan2025" > </a> </div> </div> </div> </center> <div align="right"><a href="#top"><span style="font-size: xx-small;" size="1">^ Back to Top</span></a></div></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-margin-top uk-margin-remove-bottom"> Risk Factors </h3> <div class="el-content uk-panel uk-margin-top">The concept of gene therapy seems straightforward, but this is clearly an oversimplification, and numerous problems and risks exist that prevent gene therapy using viral vectors. <a href="/glossary-of-gene-therapy-terms.html#virus">Viruses</a> can usually infect more than one type of <a href="/glossary-of-gene-therapy-terms.html#cell">cell.</a> Thus, when viral <a href="/glossary-of-gene-therapy-terms.html#vector">vectors</a> are used to carry <a href="/glossary-of-gene-therapy-terms.html#gene">genes</a> into the body, they might infect healthy cells as well as cancer cells. <p>Another danger is that the new <a href="/glossary-of-gene-therapy-terms.html#gene">gene</a> might be inserted in the wrong location in the <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a>, possibly causing harmful <a href="/glossary-of-gene-therapy-terms.html#mutation">mutations</a> to the DNA or even cancer. This has occurred in <a href="/glossary-of-gene-therapy-terms.html#clinicaltrial">clinical trials</a> for X-linked severe combined immunodeficiency (X-SCID) patients, in which hematopoietic stem cells were transduced with a corrective transgene using a <a href="/glossary-of-gene-therapy-terms.html#retrovirus">retrovirus</a>, and this led to the development of T cell <a href="/glossary-of-gene-therapy-terms.html#leukemia">leukemia</a> in 4 of 20 patients. See reports for <a href="https://www.unifr.ch/nfp37/adverse01.html" target="_blank">first patient</a>, <a href="https://www.unifr.ch/nfp37/adverse02.html" target="_blank">second patient</a> and <a href="https://www.unifr.ch/nfp37/adverse03.html" target="_blank">third patient</a>.</p> <p>In addition, when viruses are used to deliver DNA to cells inside the patient’s body, there is a slight chance that this DNA could unintentionally be introduced into the patient’s <a href="/glossary-of-gene-therapy-terms.html#reproductivecells">reproductive cells.</a> If this happens, it could produce changes that may be passed on if a patient has children after treatment. </p> <p>Other concerns include the possibility that transferred genes could be <a href="/glossary-of-gene-therapy-terms.html#geneexpression">overexpressed,</a> producing so much of the missing <a href="/glossary-of-gene-therapy-terms.html#protein">protein</a> as to be harmful; that the viral <a href="/glossary-of-gene-therapy-terms.html#vector">vector</a> could cause an immune reaction; and that the virus could be transmitted from the patient to other individuals or into the environment.</p> <p>However, this basic mode of gene introduction currently shows much promise and doctors and scientists are working hard to fix any potential problems that could exist. They use <a href="/glossary-of-gene-therapy-terms.html#preclinicaltrial">animal testing</a> and other precautions to identify and avoid these risks before any <a href="/glossary-of-gene-therapy-terms.html#clinicaltrial">clinical trials</a> are conducted in humans.</p> <center> <br /> <div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <a href="/component/banners/click/165.html" target="_blank" rel="noopener noreferrer" title="4thmRNA-BasedTherapSummitEurope-jan2025"> <img src="https://www.genetherapynet.com/images/banners/4thmRNA-BasedTherapSummitEurope-jan2025.png#joomlaImage://local-images/banners/4thmRNA-BasedTherapSummitEurope-jan2025.png?width=650&height=83" alt="4thmRNA-BasedTherapSummitEurope-jan2025" > </a> </div> </div> </div> </center><br /> <div align="right"><a href="#top"><font size="1">^ Back to Top</font></a></div></div> </div></div> <div> <div class="el-item uk-panel uk-tile-muted uk-padding-small uk-margin-remove-first-child"> <h3 class="el-title uk-card-title uk-margin-top uk-margin-remove-bottom"> Pseudotyping of Viral Vectors </h3> <div class="el-content uk-panel uk-margin-top">Viral <a href="/glossary-of-gene-therapy-terms.html#vector">vectors</a> have natural host cell populations that they infect most efficiently. <a href="/glossary-of-gene-therapy-terms.html#retrovirus">Retroviruses</a> have limited natural host cell ranges, and although <a href="/glossary-of-gene-therapy-terms.html#adenovirus">adenovirus</a> and <a href="/glossary-of-gene-therapy-terms.html#aav">adeno-associated virus</a> are able to infect a relatively broader range of cells efficiently, some cell types are refractory to infection by these viruses as well. Attachment to and entry into a susceptible cell is mediated by the <a href="/glossary-of-gene-therapy-terms.html#protein">protein</a> envelope on the surface of a <a href="/glossary-of-gene-therapy-terms.html#virus">virus</a>. <p>Retroviruses and adeno-associated viruses have a single protein coating their membrane, while adenoviruses are coated with both an envelope protein and fibers that extend away from the surface of the virus. The envelope proteins on each of these viruses bind to cell-surface <a href="/glossary-of-gene-therapy-terms.html#molecule">molecules</a> such as heparin sulfate, which localizes them upon the surface of the potential host, as well as with the specific protein receptor that either induces entry-promoting structural changes in the viral protein, or localizes the virus in endosomes wherein acidification of the lumen (anatomy) induces this refolding of the viral coat. In either case, entry into potential host cells requires a favorable interaction between a protein on the surface of the virus and a protein on the surface of the cell.</p> <p>For the purposes of <a href="/glossary-of-gene-therapy-terms.html#genetherapy">gene therapy</a>, one might either want to limit or expand the range of <a href="/glossary-of-gene-therapy-terms.html#cell">cells</a> susceptible to transduction by a gene therapy <a href="/glossary-of-gene-therapy-terms.html#vector">vector</a>. To this end, many vectors have been developed in which the endogenous viral envelope proteins have been replaced by either envelope proteins from other viruses, or by chimeric proteins. Such chimera would consist of those parts of the viral protein necessary for incorporation into the virion as well as sequences meant to interact with specific host cell proteins. Viruses in which the envelope proteins have been replaced as described are referred to as pseudotyped viruses.</p> <p>For example, the most popular retroviral vector for use in gene therapy trials has been the <a href="/glossary-of-gene-therapy-terms.html#lentivirus">lentivirus</a> Simian immunodeficiency virus coated with the envelope <a href="/glossary-of-gene-therapy-terms.html#protein">proteins</a>, G-protein, from Vesicular Stomatitus virus. This vector is referred to as VSV G-pseudotyped lentivirus, and infects an almost universal set of cells. This <a href="/glossary-of-gene-therapy-terms.html#tropism">tropism</a> is characteristic of the VSV G-protein with which this vector is coated.</p> <p>Many attempts have been made to limit the <a href="/glossary-of-gene-therapy-terms.html#tropism">tropism</a> of viral vectors to one or a few host cell populations. This advance would allow for the systemic administration of a relatively small amount of <a href="/glossary-of-gene-therapy-terms.html#vector">vector.</a> The potential for off-target cell modification would be limited, as well as many concerns from the medical community. Most attempts to limit tropism have used chimeric envelope proteins bearing antibody fragments. These vectors show great promise for the development of "magic bullet" gene therapies.</p> <center> <style> .aangepast { width: 320px; } @media(min-width: 500px) { .aangepast { width: 468px; } ) @media(min-width: 800px) { .aangepast { width: 468px; } } </style> <script async src="https://pagead2.googlesyndication.com/pagead/js/adsbygoogle.js"></script> <ins class="adsbygoogle aangepast" style="display:block; text-align:center;" data-ad-layout="in-article" data-ad-format="fluid" data-ad-client="ca-pub-1253043591735915" data-ad-slot="5059787231"></ins> <script> (adsbygoogle = window.adsbygoogle || []).push({}); </script> </center> <div align="right"><a href="#top"><font size="1">^ Back to Top</font></a></div></div> </div></div> </div> </div> </div> <div class="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"><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-active 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" /> <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" 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