RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL
发明人:
申请号：
IL27397620
公开号：
IL273976D0
申请日:
2020.04.16
申请国别(地区):
IL
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.
