University of California

Ionis Pharmaceuticals, Inc.

Ludwig Institute for Cancer Research Limited

Ludwig Institute for Cancer

Clotilde Lagier-Tourenne

C Frank Bennett

Frank Rigo

Don W Cleveland

John M Ravits

Michael W Baughn

Susan M Freier

Disclosed herein are compositions and methods for reducing expression of C9ORF72 antisense transcript in an 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.

Methods for modulating expression of C9ORF72 antisense transcript

Disclosed herein are methods for reducing expression of C90RF72 mRNA and protein in an animal with C90RF72 specific inhibitors. Such methods are useful to treat, prevent, or ameliorate neurodegenerative diseases in an individual in need thereof. Such C90RF72 specific inhibitors include antisense compounds. Examples of neurodegenerative diseases that can be treated, prevented, and ameliorated with the administration C90RF72 specific inhibitors include amyotrophic lateral sclerosis (ALS), frontotemporal dementia (FTD), corticalbasal degeneration syndrome (CBD), atypical Parkinsonian syndrome, and olivopontocerellar degeneration (OPCD).

Methods for modulating C9ORF72 expression

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Winchester, MA, United States of America

Clotilde Lagier-Tourenne