Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.

doi:10.1073/pnas.1318835110

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Article

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration.

Proceedings of the National Academy of Sciences of the United States of America - 19 Nov 2013

Lagier-Tourenne Clotilde, Baughn Michael, Rigo Frank, Sun Shuying, Liu Patrick, Li Hai-Ri, Jiang Jie, Watt Andrew T, Chun Seung, Katz Melanie, Qiu Jinsong, Sun Ying, Ling Shuo-Chien, Zhu Qiang, Polymenidou Magdalini, Drenner Kevin, Artates Jonathan W, McAlonis-Downes Melissa, Markmiller Sebastian, Hutt Kasey R, Pizzo Donald P, Cady Janet, Harms Matthew B, Baloh Robert H, Vandenberg Scott R, Yeo Gene W, Fu Xiang-Dong, Bennett C Frank, Cleveland Don W, Ravits John

Abstract excerpt

Expanded hexanucleotide repeats in the chromosome 9 open reading frame 72 (C9orf72) gene are the most common genetic cause of ALS and frontotemporal degeneration (FTD). Here, we identify nuclear RNA foci containing the hexanucleotide expansion (GGGGCC) in patient cells, including white blood cells, fibroblasts, glia, and multiple neuronal cell types (spinal motor, cortical, hippocampal, and cerebellar neurons)....

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 24170860
DOI: 10.1073/pnas.1318835110
PMCID: PMC3839752

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SOD1

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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