Enhanced CRISPR-Cas9 correction of Duchenne muscular dystrophy in mice by a self-complementary AAV delivery system.

The article focuses on the Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the dystrophin gene (DMD). It mentions that high doses of adeno-associated virus (AAV) are required for efficient in vivo genome editing, posing challenges for clinical application; and also mentions the dose of scAAV required for efficient genome editing were at least 20-fold lower than with single-stranded AAV (ssAAV).