Neurodegenerative disease: C9orf72 RNA foci—a therapeutic target for ALS and FTD?

Hexanucleotide repeat expansions in the C9orf72 gene have emerged as an important cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. In a new study led by John Ravits and Don Cleveland at the University of California, San Diego, sense and antisense C9orf72 RNA foci were identified in cells from patients with this repeat expansion, and could represent targets for oligonucleotide-based therapy.

NATURE REVIEWS NEUROLOGY 2013