RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
Kevin Flanigan,Adeline Vulin-Chaffiol,Nicolas Wein
申请号：
US16032744
公开号：
US20190276822A1
申请日:
2018.07.11
申请国别(地区):
US
年份:
2019
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.
