ASSOCIATION INSTITUT DE MYOLOGIE;INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE;UNIVERSITE PIERRE ET MARIE CURIE (PARIS 6)
发明人:
Martine Barkats,Maria-Grazia Biferi,Thomas Voit
申请号：
US15327011
公开号：
US10590420B2
申请日:
2015.07.31
申请国别(地区):
US
年份:
2020
代理人:
摘要：
The invention relates to a method for the treatment of amyotrophic lateral sclerosis (ALS). Specifically, the invention implements the use of an antisense sequence adapted to affect alternative splicing in a human SOD1 pre-mRNA, thereby leading to the destruction of the skipped m RNA by the cell machinery.
