Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.

doi:10.1038/s41467-021-21112-8

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Article

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models.

Nature communications - 8 Feb 2021

Liu Yuanjing, Dodart Jean-Cosme, Tran Helene, Berkovitch Shaunna, Braun Maurine, Byrne Michael, Durbin Ann F, Hu Xiao Shelley, Iwamoto Naoki, Jang Hyun Gyung, Kandasamy Pachamuthu, Liu Fangjun, Longo Kenneth, Ruschel Jörg, Shelke Juili, Yang Hailin, Yin Yuan, Donner Amy, Zhong Zhong, Vargeese Chandra, Brown Robert H

Abstract excerpt

A large G4C2-repeat expansion in C9orf72 is the most common genetic cause of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). Neuronal degeneration associated with this expansion arises from a loss of C9orf72 protein, the accumulation of RNA foci, the expression of dipeptide repeat (DPR) proteins, or all these factors. We report the discovery of a new targeting sequence that is common to all...

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PMID: 33558503
DOI: 10.1038/s41467-021-21112-8
PMCID: PMC7870851

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

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