Research Institute at Nationwide Children's Hospital;Research Institute at Nationwide Childrens Hospital
发明人:
Rodino-Kplapac, Louise,Mendell, Jerry R.,Heller, Kristin N.
申请号：
AU2017250790
公开号：
AU2017250790A1
申请日:
2017.04.14
申请国别(地区):
AU
年份:
2018
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.
