Personalized gene and cell therapy for Duchenne Muscular Dystrophy

Barthelemy F, Wein N. Personalized gene and cell therapy for Duchenne Muscular Dystrophy. Neuromuscular disorders : NMD 2018;28:803-824   Abstract Dystrophinopathies are diseases caused by mutations in the Duchenne Muscular Dystrophy gene (DMD) encoding the dystrophin protein. Depending on the type of mutation, patients develop either the severe DMD or the milder Becker Muscular Dystrophy. […]