Research Institute at Nationwide Children's Hospital;Ludwig Institute for Cancer Research
发明人:
Kaspar, Brian K.,Foust, Kevin,Cleveland, Don W.
申请号：
HRP20200862
公开号：
HRP20200862T1
申请日:
2020.05.29
申请国别(地区):
HR
年份:
2020
代理人:
摘要：
The present invention relates to RNA-based methods for inhibiting the expression of the superoxide dismutase 1 (SOD-1) gene. Recombinant adeno-associated viruses of the invention deliver DNAs encoding RNAs that knock down the expression of SOD-1. The methods have application in the treatment of amyotrophic lateral sclerosis.
