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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 class="uk-panel _menu menulist tm-child-list" id="module-67"> <h3> Most Popular Items </h3> <ul class="mostread"> <li><a href="/breaking-gene-therapy-news.html">Breaking Gene Therapy News</a></li> <li><a href="/diseases-treated-by-gene-therapy.html">Diseases Treated by Gene Therapy </a></li> <li><a href="/types-of-gene-therapy.html">Types of Gene Therapy</a></li> <li><a href="/viral-vector/adeno-associated-viruses.html">Adeno-Associated Viral Vectors</a></li> <li><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-66"> <h3> Who's Online </h3> <div class="mod-whosonline"> <p>We have 70 guests and one member online</p> </div> </div> </div> <div> <div class="uk-panel" id="module-29"> <h3> Statistics </h3> <ul class="mod-stats list-group"> <li class="list-group-item"> Users <span class="badge bg-secondary float-end rounded-pill">5529</span> </li> <li class="list-group-item"> Articles <span class="badge bg-secondary float-end rounded-pill">733</span> </li> <li class="list-group-item"> Articles View Hits <span class="badge bg-secondary float-end rounded-pill">17085660</span> </li> </ul> </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-row-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"> <a href="/user-login.html" class="uk-link-reset">Welcome to Gene Therapy Net</a> </h3> <div class="el-content uk-panel uk-margin-top"><strong>Gene Therapy Net</strong> is the web resource for <a href="/patient-information.html">patients</a> and professionals interested in <a href="/what-is-gene-therapy.html">gene therapy</a>. The objectives of Gene Therapy Net are to be the information resource for basic and clinical research in gene therapy, cell therapy, and genetic vaccines, and to serve as a network in the exchange of information and news related to above areas. In addition, Gene Therapy Net provides an overview for sponsors and researchers of the different international <a href="/regulatory-affairs.html">regulations and guidelines</a> associated with <a href="/clinicaltrialsgov.html">clinical gene therapy trials</a>. For resources pertaining to regenerative cell therapy and clinical treatment, check out <a href="https://r3stemcell.com" target="_blank">R3 Stem Cell</a>.<br /><center><br /> <div class="moduletable "> <div class="mod-banners bannergroup"> <div class="mod-banners__item banneritem"> <a href="/component/banners/click/141.html" target="_blank" rel="noopener noreferrer" title="CellGeneTherapySummit-jul2025"> <img src="https://www.genetherapynet.com/images/banners/CellGeneTherapySummit-jul2025.jpg#joomlaImage://local-images/banners/CellGeneTherapySummit-jul2025.jpg?width=2031&height=259" alt="CellGeneTherapySummit-jul2025" > </a> </div> </div> </div> </center></div> <div class="uk-margin-top"><a href="/user-login.html" class="el-link uk-button uk-button-default">Log in or register to read more</a></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"> <a href="/user-login.html" class="uk-link-reset">A New Gene Therapy Technique Aims to Deliver Treatment Before Birth</a> </h3> <div class="el-content uk-panel uk-margin-top"><em>Posted on: 30 October 2024, source: Labroots</em><br />Researchers have developed a novel delivery system for sending CRISPR reagents to brain cells, where they perform their gene editing function. In this approach, a nanoparticle exposes cells to the instructions for building CRISPR reagents and the correct gene sequence to repair a gene defect. The method also aims to repair genetic problems at very early stages of development. While more research will be needed, the investigators are hopeful that this technique will eventually be used to treat human patients who are identified during prenatal testing. <center><div class="fastsocialshare_container fastsocialshare-align-left"><div class="fastsocialshare-subcontainer"><script> var loadAsyncDeferredFacebook = function() { (function(d, s, id) { var js, fjs = d.getElementsByTagName(s)[0]; if (d.getElementById(id)) return; js = d.createElement(s); js.id = id; js.src = "//connect.facebook.net/en_GB/sdk.js#xfbml=1&version=v3.0&appId=201432789890280"; fjs.parentNode.insertBefore(js, fjs); }(document, 'script', 'facebook-jssdk')); } if (window.addEventListener) window.addEventListener("load", loadAsyncDeferredFacebook, false); else if (window.attachEvent) window.attachEvent("onload", loadAsyncDeferredFacebook); else window.onload = loadAsyncDeferredFacebook; </script><div class="fastsocialshare-share-fbl fastsocialshare-standard"> <div class="fb-like" data-href="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-layout="standard" data-width="100" data-action="like" data-show-faces="true" data-share="false"> </div></div><div class="fastsocialshare-share-fbsh fb-shareme-core"> <div class="fb-share-button fb-shareme-core" data-href="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-layout="button_count" data-size="small"> </div></div><div class="fastsocialshare-share-tw"> <a href="https://twitter.com/intent/tweet" data-dnt="true" class="twitter-share-button" data-text="A New Gene Therapy Technique Aims to Deliver Treatment Before Birth" data-count="horizontal" data-via="genetherapynet" data-url="https://www.genetherapynet.com/gene-therapy-news/799-a-new-gene-therapy-technique-aims-to-deliver-treatment-before-birth.html" data-lang="en"></a> </div> <script> var loadAsyncDeferredTwitter = function() { var d = document; 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The findings, from a study published in The New England Journal of Medicine (NEJM) yesterday (9 October), show that seven out of 67 patients under 18 years of age who took part in a Phase II and Phase III trial for Skysona developed haematologic cancers. 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The clinical trial, an open-label study (NCT06270316) focused on safety and early efficacy, is still recruiting about 12 adult male patients at a site in Fairfax, Virginia. Eligible men will have had a suboptimal response to enzyme replacement therapy (ERT). 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The protein Tau is essential to the function of cells in the brain and central nervous system, but when over-produced under certain conditions, it forms tangles that clog the cells’ internal structures. These tangles have also been found in Alzheimer’s disease patients. 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It’s an all-or-nothing proposition. But what if it wasn’t? What if gene therapy could be dialed up or down, on a daily basis, with a simple pill? The possibility has inspired MeiraGTx to develop a riboswitch technology that is designed to allow for the precise, dose-responsive control of gene expression by oral small molecules. The riboswitch technology is just part of MeiraGTx’s work in gene therapy. The company has technologies for the optimization of adeno-associated virus (AAV) vectors and for the design of promoter sequences. Also, the company has an internally developed manufacturing platform process and several production facilities. 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The family of the toddler taking part in a medical trial has called the change in their daughter "mind-blowing." Opal Sandy, now 18 months old, was born with total deafness due to a fault in the OTOF gene, which makes a protein called Otoferlin. Otoferlin enables communication between cells of the inner ear, or cochlea, and the brain. As part of a trial run by Cambridge University, Opal received an infusion of a working copy of the OTOF gene in her right ear. 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