<div dir="ltr"><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">A very exciting development...</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">"It’s not that gene therapy was designed to be a rule-breaker, but until now, scientists haven’t been able to effectively control its strength. Once unleashed into the body, the treatment replaces a defective gene with a healthy one, allowing the body to produce functional proteins. But sometimes the level of those proteins are too low—essentially nixing any therapeutic effects—and other times too high, poisoning the cells they’re supposed to treat.</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">Last week, scientists <a href="https://www.nature.com/articles/s41586-021-03770-2?utm_source=other&utm_medium=other&utm_content=null&utm_campaign=JRCN_1_DD01_CN_NatureRJ_article_paid_XMOL" style="box-sizing:border-box;background-color:transparent;color:rgb(237,102,41);text-decoration-line:none">finally gained control</a> of the technology with a genius strategy. A team from the Children’s Hospital of Philadelphia (CHOP) tapped into a natural process during gene expression—when genes make proteins—and hijacked it to make a “dimmer” that controls the strength of potentially any gene therapy.</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">Dubbed X<span style="box-sizing:border-box;font-size:15px;line-height:0;vertical-align:baseline">on</span>, the on switch is easy: just pop a simple drug, one that’s already in later-stage clinical trials. The drug then works with the team’s custom-designed switch to turn on the therapy throughout the body—the larger the dose, the stronger the gene expression. As the body metabolizes the drug, the dimmer gradually turns off the treatment. Need more protein? Take another pill.</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">In mice, the team fine-tuned the expression of proteins that help treat anemia in chronic kidney disease, upped the level of a protein that protects against a type of dementia, and controlled the strength of CRISPR for editing genes in the liver.</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px">“We’re taking the field of gene therapy to an entirely new level where fine-tuned dosing is required for safety, utility, and success,” <a href="https://www.chop.edu/news/children-s-hospital-philadelphia-researchers-develop-dimmer-switch-help-control-gene-therapy" style="box-sizing:border-box;background-color:transparent;color:rgb(237,102,41);text-decoration-line:none">said</a> senior author Dr. Beverly L. Davidson."</p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px"><a href="https://singularityhub.com/2021/08/03/new-universal-switch-lets-scientists-fine-tune-gene-therapy/">https://singularityhub.com/2021/08/03/new-universal-switch-lets-scientists-fine-tune-gene-therapy/</a><br></p><p style="box-sizing:border-box;font-family:proxima-nova,sans-serif;font-size:20px;line-height:26px;margin-top:0px;margin-bottom:26px"><br></p></div>