RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
Kevin Flanigan,Adeline Vulin-Chaffiol,Nicolas Wein
申请号：
US14785769
公开号：
US09862945B2
申请日:
2014.04.18
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention relates to recombinant adeno-associated virus (rAAV) delivery of polynucleotides for treating Duchenne Muscular Dystrophy resulting from the duplication of DMD exon 2. The invention provides rAAV products and methods of using the rAAV in the treatment of Duchenne Muscular Dystrophy.
