Research Institute at Nationwide Children's Hospital
发明人:
RODINO-KLAPAC, Louise,MENDELL, Jerry, R.
申请号：
EP17783207
公开号：
EP3442600A4
申请日:
2017.04.14
申请国别(地区):
EP
年份:
2019
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.
