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Abstract
Muscular dystrophy is a disease characterized by severe muscle weakness due to the destruction and repair of skeletal muscle cells. It can manifest from birth, potentially leading to early death, or later in adulthood. Duchenne Muscular Dystrophy (DMD) and Becker muscular dystrophy are among the types of muscular dystrophy. DMD is the most common, accounting for 85% of dystrophinopathies, but it's also the most severe, affecting approximately 1 in 3500 people. It primarily affects children under 5 years old. The disease stems from mutations in the dystrophin gene, leading to the functional and structural degradation of the 427 kDA cytoskeletal protein encoded by dystrophin. Consequently, most DMD patients lack dystrophin protein due to these mutations. This study delves into gene therapy methods for treating DMD.
