Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy


This study investigates the effectiveness of adeno-associated viral (AAV) vector-mediated microdystrophin gene addition therapy in a fibrotic mouse model of Duchenne muscular dystrophy (DMD). The researchers administered AAV8-MD1 in juvenile D2.mdx mice and explored its effect in the diaphragm, a critical respiratory muscle in DMD pathology. They reveal that AAV8-MD1 significantly improves body-wide muscle function, protects hindlimb muscles from contraction-induced damage, and prevents fibrosis deposition in the diaphragm. These findings support the potential benefits of gene therapy in preventing muscle fibrosis in DMD.