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Article

Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide.

Nature medicine - 1 Jan 2022

Tran Hélène, Moazami Michael P, Yang Huiya, McKenna-Yasek Diane, Douthwright Catherine L, Pinto Courtney, Metterville Jake, Shin Minwook, Sanil Nitasha, Dooley Craig, Puri Ajit, Weiss Alexandra, Wightman Nicholas, Gray-Edwards Heather, Marosfoi Miklos, King Robert M, Kenderdine Thomas, Fabris Daniele, Bowser Robert, Watts Jonathan K, Brown Robert H

Abstract excerpt

Expansions of a G4C2 repeat in the C9ORF72 gene are the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two devastating adult-onset neurodegenerative disorders. Using C9-ALS/FTD patient-derived cells and C9ORF72 BAC transgenic mice, we generated and optimized antisense oligonucleotides (ASOs) that selectively blunt expression of G4C2 repeat-containing...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Identifiers

PMID: 34949835
DOI: 10.1038/s41591-021-01557-6
PMCID: PMC8861976

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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