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Article

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.

Nature - 20 Apr 2017

Scoles Daniel R, Meera Pratap, Schneider Matthew D, Paul Sharan, Dansithong Warunee, Figueroa Karla P, Hung Gene, Rigo Frank, Bennett C Frank, Otis Thomas S, Pulst Stefan M

Abstract excerpt

There are no disease-modifying treatments for adult human neurodegenerative diseases. Here we test RNA-targeted therapies in two mouse models of spinocerebellar ataxia type 2 (SCA2), an autosomal dominant polyglutamine disease. Both models recreate the progressive adult-onset dysfunction and degeneration of a neuronal network that are seen in patients, including decreased firing frequency of cerebellar Purkinje...

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Identifiers

PMID: 28405024
DOI: 10.1038/nature22044
PMCID: PMC6625650

Genes

ATXN2

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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