Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead

Heslop E, Turner C, Irvin A, Muntoni F, Straub V, Guglieri M. Gene therapy in Duchenne muscular dystrophy: Identifying and preparing for the challenges ahead. Neuromuscular Disord 2020   Introduction There is considerable interest, excitement and anticipation for the development of adeno associated virus (AAV) gene therapy as a treatment for Duchenne muscular dystrophy (DMD). […]