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Article

Allele-specific RNA silencing of mutant ataxin-3 mediates neuroprotection in a rat model of Machado-Joseph disease.

PloS one - 8 Oct 2008

Alves Sandro, Nascimento-Ferreira Isabel, Auregan Gwennaëlle, Hassig Raymonde, Dufour Noëlle, Brouillet Emmanuel, Pedroso de Lima Maria C, Hantraye Philippe, Pereira de Almeida Luís, Déglon Nicole

Abstract excerpt

Recent studies have demonstrated that RNAi is a promising approach for treating autosomal dominant disorders. However, discrimination between wild-type and mutant transcripts is essential, to preserve wild-type expression and function. A single nucleotide polymorphism (SNP) is present in more than 70% of patients with Machado-Joseph disease (MJD). We investigated whether this SNP could be used to inactivate...

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PMID: 18841197
DOI: 10.1371/journal.pone.0003341
PMCID: PMC2553199

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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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