Ionis Pharmaceuticals, Inc.
METHODS FOR REDUCING C9ORF72 EXPRESSION

Abstract:

Provided are methods for reducing the amount or activity of C9ORF72 RNA, and in certain instances of reducing the amount of C9ORF72 protein, in an animal. Such methods are useful to prevent or ameliorate at least one symptom of a neurodegenerative disease. Such symptoms include anxiety, reduced spatial learning, and memory loss. Such neurodegenerative diseases include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).