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1.发明申请
公开(公告)号：WO2018170408A1
公开(公告)日：2018-09-20
申请号：PCT/US2018/022881
申请日：2018-03-16
Inventor： RODINO-KLAPAC, Louise , MENDELL, Jerry, R.
IPC: C12N15/86 , C12N15/113 , C12N15/09
Abstract: The invention provides gene therapy vectors, such as adeno-associated virus (AAV) vectors, expressing a miniaturized human micro-dystrophin gene and method of using these vectors to express micro-dystrophin in skeletal muscle s including diaphragm and cardiac muscle and to protect muscle fibers from injury, increase muscle strength and reduce and/or prevent fibrosis in subjects suffering from muscular dystrophy.
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