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Article

RNAi prevents and reverses phenotypes induced by mutant human ataxin-1.

Annals of neurology - 1 Nov 2016

Keiser Megan S, Monteys Alejandro Mas, Corbau Romuald, Gonzalez-Alegre Pedro, Davidson Beverly L

Abstract excerpt

OBJECTIVE: Spinocerebellar ataxia type 1 is an autosomal dominant fatal neurodegenerative disease caused by a polyglutamine expansion in the coding region of ATXN1. We showed previously that partial suppression of mutant ataxin-1 (ATXN1) expression, using virally expressed RNAi triggers, could prevent disease symptoms in a transgenic mouse model and a knockin mouse model of the disease, using a single dose of...

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Identifiers

PMID: 27686464
DOI: 10.1002/ana.24789
PMCID: PMC5115960

Genes

ATXN1

Provenance

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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