| abstract |
The invention provides gene therapy vectors, such as adeno-associated viral vectors (AAVs), expressing a functional fragment of the miniaturized human micro-dystrophin gene as well as methods of using these vectors to express the fragment of micro-dystrophin in skeletal muscles, including the diaphragm and cardiac muscle, and to protect muscle fibers from injury, to increase muscle strength and to reduce and / or prevent fibrosis in subjects with muscular dystrophy. |