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：
CA3021057
公开号：
CA3021057A1
申请日:
2017.04.14
申请国别(地区):
CA
年份:
2017
代理人:
摘要：
Described herein are recombinant AAV vectors comprising a polynucleotide sequence comprising ß-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 ß-sarcoglycan and miR-29c to a mammalian subject suffering from a muscular dystrophy.
