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Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy

Amoasii L, Hildyard JCW, Li H, et al. Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy. Science (New York, N.Y.) 2018;362:86-90   Abstract Mutations in the gene encoding dystrophin, a protein that maintains muscle integrity and function, cause Duchenne muscular dystrophy (DMD). The deltaE50-MD dog model of DMD harbors a […]