Muscle‐specific, liver‐detargeted adeno‐associated virus gene therapy rescues Pompe phenotype in adult and neonate Gaa−/− mice
Pompe disease (PD) is a neuromuscular disorder caused by acid α‐glucosidase (GAA)
deficiency. Reduced GAA activity leads to pathological glycogen accumulation in cardiac …
deficiency. Reduced GAA activity leads to pathological glycogen accumulation in cardiac …