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Gene-silencing tool shows promise as a future therapy against prion diseases
MIT Whitehead and Broad Institute researchers designed an epigenetic editor that can silence the prion protein gene, which could potentially be developed into a therapy against fatal prion diseases. The same approach could be used against other genetic diseases.
The two now run a lab at the Broad Institute, where they are working to develop drugs that can prevent and treat these diseases, and their deadline for success is not based on grant cycles or academic expectations but on the ticking time bomb in Vallabh’s genetic code. The researchers’ goal was to create a tool based on CRISPRoff that was just as potent but also safe for use in humans, small enough to deliver to the brain, and designed to minimize the risk of silencing the wrong genes or causing side effects. Meanwhile, a complementary project from Broad Institute scientist and collaborator Benjamin Deverman’s lab, focused on brain-wide gene delivery and published in Science on May 17, has brought the CHARM technology one step closer to being ready for clinical trials.
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