Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy

Abreu NJ, Waldrop MA. Overview of Gene Therapy in Spinal Muscular Atrophy and Duchenne Muscular Dystrophy. Pediatr Pulm. 2020   Abstract Both 5q-linked spinal muscular atrophy (SMA) and Duchenne muscular dystrophy (DMD) are fatal monogenic neuromuscular disorders caused by loss-of-function mutations. SMA is an autosomal recessive disorder affecting motor neurons that is typically caused by […]