Sangamo Therapeutics, Inc.
Modulators of Chromosome 9 Open Reading Frame 72 Gene Expression and Uses Thereof

Abstract:

The present disclosure provides compositions and methods for modulating transcription of mutant C9orf72 gene alleles in patients in need thereof, including patients having a C9orf72-related disease such as amyotrophic lateral sclerosis (ALS) or frontotemporal dementia (FTD).