AAV-based gene therapies for the muscular dystrophies

Crudele JM, Chamberlain JS. AAV-based gene therapies for the muscular dystrophies. Human molecular genetics 2019;28:R102-R107   Abstract Muscular dystrophy is a group of progressive genetic diseases affecting the musculature which are characterized by inflammatory infiltrates, necrosis and connective tissue and fat replacement of the affected muscles. Unfortunately, treatments do not exist for the vast majority […]