Research Institute at Nationwide Children's Hospital;Research Institute at Nationwide Childrens Hospital
发明人:
Flanigan, Kevin,Vulin-Chaffiol, Adeline,Wein, Nicolas
申请号：
AU2014253730
公开号：
AU2014253730B2
申请日:
2014.04.18
申请国别(地区):
AU
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.
