PRODUCTS AND METHODS FOR TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
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.
申请公布号
EP3039146(A1)
申请公布日期
2016.07.06
申请号
EP20140761771
申请日期
2014.08.26
申请人
THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN'S HOSPITAL;LUDWIG INSTITUTE FOR CANCER RESEARCH
