Gene Therapy Non-Viral Vectors Explained

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Previously, low levels of transfection and expression of the <a href="/glossary-of-gene-therapy-terms.html#gene">gene</a> held non-viral methods at a disadvantage; however, recent advances in <a href="/glossary-of-gene-therapy-terms.html#vector">vector</a> technology have yielded <a href="/glossary-of-gene-therapy-terms.html#molecule">molecules</a> and techniques with transfection efficiencies similar to those of <a href="/glossary-of-gene-therapy-terms.html#virus">viruses</a>. <br /><br /> </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"> Naked DNA </h3> <div class="el-content uk-panel uk-margin-top">This is the simplest method of non-viral transfection. <a href="/glossary-of-gene-therapy-terms.html#clinicaltrial">Clinical trials</a> carried out of intramuscular injection of a naked <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a> plasmid have occurred with some success; however, the expression has been very low in comparison to other methods of transfection. In addition to trials with plasmids, there have been trials with naked <a href="/glossary-of-gene-therapy-terms.html#pcr">PCR</a> product, which have had similar or greater success. This success, however, does not compare to that of the other methods, leading to research into more efficient methods for delivery of the naked DNA such as electroporation and the use of a "gene gun", which shoots DNA coated gold particles into the cell using high pressure gas. <br /><br /> <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> <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"> Oligonucleotides </h3> <div class="el-content uk-panel uk-margin-top">The use of synthetic oligonucleotides in <a href="/glossary-of-gene-therapy-terms.html#genetherapy">gene therapy</a> is to inactivate the <a href="/glossary-of-gene-therapy-terms.html#gene">genes</a> involved in the disease process. There are several methods by which this is achieved. One strategy uses antisense specific to the target gene to disrupt the <a href="/glossary-of-gene-therapy-terms.html#transcription">transcription</a> of the faulty <a href="/glossary-of-gene-therapy-terms.html#gene">gene</a>. Another uses small <a href="/glossary-of-gene-therapy-terms.html#molecule">molecules</a> of <a href="/glossary-of-gene-therapy-terms.html#rna">RNA</a> called siRNA to signal the <a href="/glossary-of-gene-therapy-terms.html#cell">cell</a> to cleave specific unique sequences in the mRNA transcript of the faulty gene, disrupting <a href="/glossary-of-gene-therapy-terms.html#translation">translation</a> of the faulty mRNA, and therefore <a href="/glossary-of-gene-therapy-terms.html#geneexpression">expression</a> of the gene. <p>A further strategy uses double stranded oligodeoxynucleotides as a decoy for the transcription factors that are required to activate the <a href="/glossary-of-gene-therapy-terms.html#transcription">transcription</a> of the target <a href="/glossary-of-gene-therapy-terms.html#gene">gene.</a> The transcription factors bind to the decoys instead of the <a href="/glossary-of-gene-therapy-terms.html#promoter">promoter</a> of the faulty gene, which reduces the transcription of the target gene, lowering <a href="/glossary-of-gene-therapy-terms.html#geneexpression">expression.</a>.</p> <center> <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><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"> Lipoplexes and polyplexes </h3> <div class="el-content uk-panel uk-margin-top">To improve the delivery of the new <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a> into the <a href="/glossary-of-gene-therapy-terms.html#cell">cell</a>, the DNA must be protected from damage and its entry into the cell must be facilitated. To this end new <a href="/glossary-of-gene-therapy-terms.html#molecule">molecules</a>, lipoplexes and polyplexes, have been created that have the ability to protect the DNA from undesirable degradation during the transfection process. <p>Plasmid DNA can be covered with lipids in an organized structure like a micelle or a liposome. When the organized structure is complexed with DNA it is called a lipoplex. There are three types of lipids, anionic (negatively charged), neutral, or cationic (positively charged). Initially, anionic and neutral lipids were used for the construction of lipoplexes for synthetic <a href="/glossary-of-gene-therapy-terms.html#vector">vectors.</a> However, in spite of the facts that there is little toxicity associated with them, that they are compatible with body fluids and that there was a possibility of adapting them to be tissue specific; they are complicated and time consuming to produce so attention was turned to the cationic versions.</p> <p>Cationic lipids, due to their positive charge, naturally complex with the negatively charged <a href="/glossary-of-gene-therapy-terms.html#dna">DNA.</a> Also as a result of their charge they interact with the cell membrane, endocytosis of the lipoplex occurs and the DNA is released into the <a href="/glossary-of-gene-therapy-terms.html#cytoplasm">cytoplasm.</a> The cationic lipids also protect against degradation of the DNA by the cell.</p> <p>The most common use of lipoplexes has been in gene transfer into cancer cells, where the supplied genes have activated <a href="/glossary-of-gene-therapy-terms.html#tumorsuppressor">tumor suppressor</a> control genes in the cell and decrease the activity of oncogenes. Recent studies have shown lipoplexes to be useful in transfecting respiratory epithelial cells, so they may be used for treatment of genetic respiratory diseases such as <a href="/glossary-of-gene-therapy-terms.html#cysticfibrosis">cystic fibrosis.</a></p> <p>Complexes of polymers with <a href="/glossary-of-gene-therapy-terms.html#dna">DNA</a> are called polyplexes. Most polyplexes consist of cationic polymers and their production is regulated by ionic interactions. One large difference between the methods of action of polyplexes and lipoplexes is that polyplexes cannot release their DNA load into the <a href="/glossary-of-gene-therapy-terms.html#cytoplasm">cytoplasm</a>, so to this end, co-transfection with endosome-lytic agents (to lyse the endosome that is made during endocytosis, the process by which the polyplex enters the <a href="/glossary-of-gene-therapy-terms.html#cell">cell</a>) such as inactivated <a href="/glossary-of-gene-therapy-terms.html#adenovirus">adenovirus</a> must occur. However this isn't always the case, polymers such as polyethylenimine have their own method of endosome disruption as does chitosan and trimethylchitosan.</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> <br /> <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-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-active 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" 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 class="uk-panel" id="module-132"> <h3> Non-Viral Vector Books </h3> <div class="uk-margin-remove-last-child custom" ><div align="center"> <iframe src="https://rcm-na.amazon-adsystem.com/e/cm?t=genthenet-20&o=1&p=8&l=as1&asins=4431251227&fc1=000000&IS2=1&lt1=_blank&lc1=000000&bc1=E3E5E6&bg1=E3E5E6&f=ifr" style="width:120px;height:240px;" scrolling="no" marginwidth="0" marginheight="0" frameborder="0"></iframe> <br /> <iframe src="https://rcm-na.amazon-adsystem.com/e/cm?t=genthenet-20&o=1&p=8&l=as1&asins=1588290190&fc1=000000&IS2=1&lt1=_blank&lc1=000000&bc1=E3E5E6&bg1=E3E5E6&f=ifr" style="width:120px;height:240px;" scrolling="no" marginwidth="0" marginheight="0" frameborder="0"></iframe><br /><br /> <a href="https://www.amazon.com/gp/search?ie=UTF8&tag=genthenet-20&linkCode=ur2&linkId=de3ca9efcc4559cceb70f153e6dba6fd&camp=1789&creative=9325&index=aps&keywords=gene%20therapy">More gene therapy books</a></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-grid uk-child-width-1-1"> <div> <div class="uk-panel" id="module-249"> <div class="uk-margin-remove-last-child custom" ><p>漏 2024 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 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