RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL;LUDWIG INSTITUTE FOR CANCER RESEARCH, LTD;RESEARCH INSTITUTE AT NATIONWIDE CHILDRENS HOSPITAL
发明人:
KASPAR, BRIAN K.,FOUST, KEVIN,CLEVELAND, DON W.
申请号：
CA2922500
公开号：
CA2922500A1
申请日:
2014.08.26
申请国别(地区):
CA
年份:
2015
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.
