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Article

Peripheral androgen receptor gene suppression rescues disease in mouse models of spinal and bulbar muscular atrophy.

Cell reports - 8 May 2014

Lieberman Andrew P, Yu Zhigang, Murray Sue, Peralta Raechel, Low Audrey, Guo Shuling, Yu Xing Xian, Cortes Constanza J, Bennett C Frank, Monia Brett P, La Spada Albert R, Hung Gene

Abstract excerpt

Spinal and bulbar muscular atrophy (SBMA) is caused by the polyglutamine androgen receptor (polyQ-AR), a protein expressed by both lower motor neurons and skeletal muscle. Although viewed as a motor neuronopathy, data from patients and mouse models suggest that muscle contributes to disease pathogenesis. Here, we tested this hypothesis using AR113Q knockin and human bacterial artificial chromosome/clone (BAC)...

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Methodology Data sources Editorial policy Privacy Delete account & data About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 24746732
DOI: 10.1016/j.celrep.2014.02.008
PMCID: PMC4356525

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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