Adeno-associated virus vector delivery of muscle-specific microdystrophin for treating muscular dystrophy

摘要

The present invention provides a gene therapy vector such as an adeno-associated virus (AAV) vector expressing a miniaturized human microdystrophin gene, and a microdystrophin expressed in skeletal muscle including diaphragm and myocardium, in a subject suffering from muscular dystrophy, Methods of using these vectors to protect muscle fibers from damage, increase muscle strength, and reduce and / or prevent fibrosis are provided. In one embodiment, the invention provides a rAAV vector comprising a nucleotide sequence of a muscle-specific regulatory element and a nucleotide sequence encoding a microdystrophin protein. [Selection diagram] Figure 1