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：
RSP20200638
公开号：
RS60434B1
申请日:
2014.08.26
申请国别(地区):
RS
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.
