RESEARCH INSTITUTE ATA NATIONWIDE CHILDREN'S HOSPITAL;LUDWIG INSTITUTE FOR CANCER RESEARCH
发明人:
Brian K. Kaspar,Kevin Foust,Don W. Cleveland
申请号：
US14914861
公开号：
US20160272976A1
申请日:
2014.08.26
申请国别(地区):
US
年份:
2016
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.
