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As U.S. efforts stall, China pushes forward with CRISPR remedies for muscular dystrophy

As U.S. efforts stall, China pushes forward with CRISPR remedies for muscular dystrophy
October 10, 2024



Two of the primary efforts to regard Duchenne muscular dystrophy with CRISPR gene enhancing are getting off the bottom in China, at the same time as initiatives within the U.S. have apparently stalled.

Trials for the deadly muscle-wasting illness have been began within the remaining couple of months by way of two other Chinese language firms. One, GenAssist, has already dosed one younger boy. Huidagene, the opposite biotech, expects to dose the primary of 3 boys quickly and file information by way of Q1 subsequent 12 months.

The efforts are notable, as scientists and advocates have lengthy was hoping CRISPR may free up a extra tough remedy for Duchenne than the generation utilized in Elevidys, Sarepta’s much-debated gene treatment. The will for the sort of remedy handiest grew remaining October, when trial effects showed that Elevidys, whilst doubtlessly helpful, is a ways from a treatment. 

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