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 …

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused
by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly …

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by
the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene …

Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene
(DMD), is characterized by fatal degeneration of striated muscles. Dilated cardiomyopathy is …

Duchenne muscular dystrophy (DMD) is an X-linked recessive, infancy-onset
neuromuscular disorder characterized by progressive muscle weakness and atrophy …

Recombinant adeno-associated virus vectors (rAAV) are being explored as gene delivery
vehicles for the treatment of various inherited and acquired disorders. rAAVs are attractive …

Micro-dystrophins are highly promising candidates for treating Duchenne muscular
dystrophy, a lethal muscle disease caused by dystrophin deficiency. Here, we report robust …

Adeno-associated virus–mediated (AAV-mediated) CRISPR editing is a revolutionary
approach for treating inherited diseases. Sustained, often life-long mutation correction is …

Gene therapy with adeno-associated viral (AAV) vectors has reached the clinical stage for
many inherited and acquired diseases. However, due to a cargo capacity limited to< 5 kb …

Highlights•Only intravascular delivery can truly change the course of systemic
diseases.•AAV has the capacity to escape from the blood and lead to bodywide gene …