this post was submitted on 01 May 2025
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Futurology

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HuidaGene Therapeutics used CRISPR gene-editing (Cas13) to modify genes in the brain for the first time, successfully treating a 9-year-old with MECP2 duplication syndrome. (archive.ph)
submitted 2 weeks ago by Lugh to c/futurology
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[–] Lugh 4 points 2 weeks ago

This is a tentative result, it's only one patient, and large scale trials would be needed to confirm it. Still, if it is confirmed it's a significant breakthrough. HuidaGene is also working on treatments for Huntington's Disease, Duchenne muscular dystrophy (DMD among other diseases. It's also working on various Ophthalmology related conditions.