Halting muscular dystrophy with a 'backup' protein
A new use of CRISPR gene editing has halted and even reversed the progression of a model of Duchenne muscular dystrophy, according to work led by scientists at the Perelman School of Medicine. The technique boosted a protein, utrophin, as a stand-in to take on the job of a different protein, dystrophin, which helps maintain healthy muscles and is in short supply in those with muscular dystrophy.
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