Research Institute at Nationwide Childrens Hospita
发明人:
FLANIGAN, Kevin,VULIN-CHAFFIOL, Adeline,WEIN, Nicolas
申请号：
HK16110120.4
公开号：
HK1221962A
申请日:
2016.08.24
申请国别(地区):
HK
年份:
2017
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.
