RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
SAHENK, ZARIFE
申请号：
CA3079416
公开号：
CA3079416A1
申请日:
2018.10.19
申请国别(地区):
CA
年份:
2019
代理人:
摘要：
The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a neurotrophin 3 (NT-3) polynucleotide. The disclosure provides rAAV and methods of using the rAAV for NT-3 gene therapy to improve muscle strength, stimulate muscle growth and to treat muscle wasting disorders, such as muscular dystrophy and Charcot-Marie- Tooth neuropathy.
