CRISPR halts Duchenne muscular dystrophy progression in dogs

CRISPR halts Duchenne muscular dystrophy progression in dogs

Scientists for the first time have used CRISPR gene editing to halt the progression of Duchenne muscular dystrophy (DMD) in a large mammal, according to a study by UT Southwestern that provides a strong indication that a lifesaving treatment may be in the pipeline. The research published in Science documents 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.

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