Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy

Duan D. Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy. Molecular therapy : the journal of the American Society of Gene Therapy 2018;26:2337-2356   Abstract Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one would cure the disease. However, this […]