Peripheral Androgen Receptor Gene Suppression Rescues Disease in Mouse Models of Spinal and Bulbar Muscular Atrophy

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 pathogen...

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Bibliographic Details
Main Authors: Andrew P. Lieberman, Zhigang Yu, Sue Murray, Raechel Peralta, Audrey Low, Shuling Guo, Xing Xian Yu, Constanza J. Cortes, C. Frank Bennett, Brett P. Monia, Albert R. La Spada, Gene Hung
Format: Article
Language:English
Published: Elsevier 2014-05-01
Series:Cell Reports
Online Access:http://www.sciencedirect.com/science/article/pii/S2211124714000874