Research Institute at Nationwide Children`s Hospital;Ludwig Institute for Cancer Research
发明人:
KASPAR, Brian,FOUST, Kevin,CLEVELAND, Don
申请号：
LT14761771
公开号：
LT3039146T
申请日:
2014.08.26
申请国别(地区):
LT
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.
