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：
CA2909807
公开号：
CA2909807A1
申请日:
2014.04.18
申请国别(地区):
CA
年份:
2014
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.
