RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
JERRY R. MENDELL,LOUISE RODINO-KLAPAC
申请号：
BR112018071182
公开号：
BR112018071182A2
申请日:
2017.04.14
申请国别(地区):
BR
年份:
2019
代理人:
摘要：
The invention provides recombinant αv vectors comprising a miniaturized human micro-dystrophin gene and methods of using recombinant vectors to reduce or prevent fibrosis in subjects suffering from muscular dystrophy.a invenção fornece vetores de aav recombinantes que compreendem um gene de micro-distrofina humana miniaturizado e métodos de uso dos vetores recombinantes para reduzir ou prevenir a fibrose em sujeitos que sofrem de distrofia muscular.
