Adeno-associated virus vector delivery of microdystrophin fragments to treat muscular dystrophy

摘要

The present invention provides a gene therapy vector such as an adeno-associated virus (AAV) vector expressing a functional fragment of the miniaturized human microdystrophin gene, and using these vectors, the microdystrophin fragment in skeletal muscle including diaphragm and myocardium. Expression provides a method of protecting muscle fibers from damage, enhancing muscle strength, and reducing and / or preventing fibrosis in patients with muscular dystrophy.