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 pathogenesis. Here, we tested this hypothesis using AR113Q knockin… (More)
DOI: 10.1016/j.celrep.2014.02.008


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