RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL;RESEARCH INSTITUTE AT NATIONWIDE CHILDRENS HOSPITAL
发明人:
RODINO-KLAPAC, Louise,MENDELL, Jerry
申请号：
SG11201809029R
公开号：
SG11201809029RA
申请日:
2017.04.14
申请国别(地区):
SG
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.
