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Article

Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.

Annals of neurology - 1 Jul 2018

McLoughlin Hayley S, Moore Lauren R, Chopra Ravi, Komlo Robert, McKenzie Megan, Blumenstein Kate G, Zhao Hien, Kordasiewicz Holly B, Shakkottai Vikram G, Paulson Henry L

Abstract excerpt

OBJECTIVE: Spinocerebellar ataxia type 3 (SCA3), also known as Machado-Joseph disease, is the most common dominantly inherited ataxia. Despite advances in understanding this CAG repeat/polyglutamine expansion disease, there are still no therapies to alter its progressive fatal course. Here, we investigate whether an antisense oligonucleotide (ASO) targeting the SCA3 disease gene, ATXN3, can prevent molecular,...

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Source

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Identifiers

PMID: 29908063
DOI: 10.1002/ana.25264
PMCID: PMC6119475

Genes

ATXN3

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Oligonucleotide therapy mitigates disease in spinocerebellar ataxia type 3 mice.PMID 29908063Read ↗

Evaluation of Antisense Oligonucleotides Targeting ATXN3 in SCA3 Mouse ModelsSemantic proximity Read ↗

Antisense Oligonucleotide Therapy Targeted Against ATXN3 Improves Potassium Channel-Mediated Purkinje Neuron Dysfunction in Spinocerebellar Ataxia Type 3.Semantic proximity Read ↗

ASOs are an effective treatment for disease-associated oligodendrocyte signatures in premanifest and symptomatic SCA3 miceSemantic proximity Read ↗

AAV-mediated allele-specific silencing alleviates neuropathology in a novel non-human primate model of Spinocerebellar ataxia type 3Semantic proximity Read ↗

rAAV-Delivered Bicistronic Artificial microRNAs for Allele-Specific Silencing Improve Motor and Molecular Outcomes in Spinocerebellar Ataxia Type 3Semantic proximity Read ↗

Antisense oligonucleotide therapy rescues aggresome formation in a novel Spinocerebellar Ataxia type 3 human embryonic stem cell lineSemantic proximity Read ↗

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