Research Institute at Nationwide Children's Hospital
发明人:
申请号：
EP14729770.9
公开号：
EP2986632B1
申请日:
2014.04.18
申请国别(地区):
EP
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.
