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

2025-03-26

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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Literature Corpus work
df4d1190-fdc3-56d8-8087-ef61fb2235f1
DOI
10.1101/2025.03.24.644908
Open publication

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Characterization of a C9orf72 Knockout <i>Danio rerio</i> model for ALS and cross-species validation of potential therapeutics screened in <i>Caenorhabditis elegans</i>DOI 10.1101/2025.03.24.644908
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