Research Institute at Nationwide Children's Hospital;Research Institute at Nationwide Childrens Hospital
发明人:
Rodino-Klapac, Louise,Mendell, Jerry R.
申请号：
AU2017250756
公开号：
AU2017250756A1
申请日:
2017.04.14
申请国别(地区):
AU
年份:
2018
代理人:
摘要：
Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising β-sarcoglycan and methods of using the recombinant vectors to reduce or prevent fibrosis in a mammalian subject suffering from a muscular dystrophy. Also described herein are combination therapies comprising administering AAV vector(s) expressing β-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
