Research Institute at Nationwide Children's Hospital
发明人:
RODINO-KLAPAC, Louise,MENDELL, Jerry, R.,HELLER, Kristin, N.
申请号：
EP17783235
公开号：
EP3442561A4
申请日:
2017.04.14
申请国别(地区):
EP
年份:
2019
代理人:
摘要：
The invention provides for recombinant AAV vectors comprising a polynucleotide sequence comprising the guide strand of miR-29c and methods of using the recombinant vectors to reduce or prevent fibrosis in subjects suffering from dystrophinopathy or muscular dystrophy. The invention also provides for combination therapies comprising expressing both miR-29 and micro-dystrophin to reduce and prevent fibrosis in patients suffering from dystrophinopathy or muscular dystrophy.
