Abstract
DMD is a rare genetic disease characterized by progressive muscle degeneration caused by mutations in the dystrophin protein, which aids in maintaining muscle cells. Recently, gene therapy for neuromuscular disease has made substantial progress with AAV vectors as they are not linked with any pathogenicity, have low immunogenicity, and can maintain transgene expression in non-dividing cells for years. In this review, the efficacy and safety of AAV vector mediated gene therapy for DMD is highlighted.
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