Research Institute at Nationwide Children's Hospital
发明人:
FLANIGAN, Kevin,VULIN-CHAFFIOL, Adeline,WEIN, Nicolas
申请号：
AU2020217441
公开号：
AU2020217441A1
申请日:
2020.08.14
申请国别(地区):
AU
年份:
2020
代理人:
摘要：
#$%^&*AU2020217441A120200903.pdf#####Abstract 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.
