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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/152.html" target="_blank" rel="noopener noreferrer" title="4thWorldBioScienceTechnology-jan2025"> <img src="https://www.genetherapynet.com/images/banners/4thWorldBioScienceTechnology-jan2025.png#joomlaImage://local-images/banners/4thWorldBioScienceTechnology-jan2025.png?width=567&height=72" alt="4thWorldBioScienceTechnology-jan2025" > </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. 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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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