Ionis Pharmaceuticals, Inc.
Compositions for modulating expression of C9ORF72 antisense transcript

Abstract:

Disclosed herein are compositions and methods for modulating expression of C9ORF72 antisense transcript in a cell or animal with C9ORF72 antisense transcript specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C9ORF72 antisense transcript specific inhibitors include antisense compounds.