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Article

Characterization of a C9orf72 Knockout Danio rerio model for ALS and cross-species validation of potential therapeutics screened in Caenorhabditis elegans.

PloS one - 1 Jan 2026

Emond Alexandre, Laflamme Carl, Therrien Martine, Liao Meijiang, Maios Claudia, Labarre Audrey, Drapeau Pierre, Parker J Alex

Abstract excerpt

Intronic hexanucleotide repeat expansions in the C9orf72 gene represent the most common genetic cause of the neurodegenerative diseases amyotrophic lateral sclerosis (ALS) and frontotemporal dementia. This expansion decreases C9orf72 expression in affected patients, indicating that loss of C9orf72 function (LOF) acts as a pathogenic mechanism. Several models using Danio rerio (zebrafish) for C9orf72 depletion...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 41961863
DOI: 10.1371/journal.pone.0346613
PMCID: PMC13068224

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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