RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
Louise Rodino-Klapac,Jerry R. Mendell
申请号：
US16494614
公开号：
US20200199621A1
申请日:
2018.03.16
申请国别(地区):
US
年份:
2020
代理人:
摘要：
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.
