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Systemic delivery of an AAV9 exonskipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
Background: Duchenne muscular dystrophy (DMD) is a severe genetic disorder caused by mutations in the DMD gene. These mutations disrupt the dystrophin protein, essential for muscle function. Current treatments, like exon-skipping oligonucleotides, have limitations due to their short lifespan and need for frequent administration. Objective: This study explored the use of a self-complementary adeno-associated virus (scAAV9) vector expressing U7 small nuclear RNA (snRNA) to target exon 2 of …
