Research Institute at Nationwide Children's Hospital
发明人:
SAHENK, Zarife
申请号：
AU2018351528
公开号：
AU2018351528A1
申请日:
2018.10.19
申请国别(地区):
AU
年份:
2020
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.
