Gene Therapy for Duchenne Muscular Dystrophy - PMC

Duchenne muscular dystrophy (DMD), affecting 1 in 5000 males, is caused by mutations in the DMD gene, leading to muscle degeneration due to dystrophin absence. Gene therapy using AAV vectors to restore dystrophin shows promise, with ongoing clinical trials. Challenges include optimizing dosage, immune response, and manufacturing for effective treatment.