Duchenne Muscular Dystrophy (DMD) is a rare disorder, but it is one of the most common genetic conditions, affecting roughly 1 in every 3,500 male births worldwide. Gene therapy for Duchenne Muscular Dystrophy (DMD) is poised to revolutionize the treatment landscape by addressing the underlying genetic cause of the disease rather than merely managing its symptoms. DMD is caused by mutations in the DMD gene, which encodes the dystrophin protein crucial for maintaining the integrity of muscle cells. The absence or dysfunction of dystrophin leads to progressive muscle degeneration and weakness, ultimately resulting in severe complications like cardiomyopathy and respirat...
