CRISPR gene editing successfully stopped Duchenne muscular dystrophy in dogs and human trials are next
CRISPR gene editing has stopped the progression of Duchenne muscular dystrophy (DMD) in dogs, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. unprecedented improvement in the muscle fibers of dogs with DMD – the most common fatal genetic disease in children, caused by a mutation that inhibits the production of dystrophin, a protein critical for muscle function. Researchers used a single-cut gene-editing technique to restore dystrophin in muscle and heart tissue by up to 92 percent of normal levels. Scientists have estimated a 15 percent threshold is
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