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

Neurology

Brain Disorders & Therapy

Author(s): Lieberman AP, Yu Z, Murray S, Peralta R, Low A,

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Abstract 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) transgenic mice that express the full-length polyQ-AR and display androgen-dependent weakness, muscle atrophy, and early death. We developed antisense oligonucleotides that suppressed AR gene expression in the periphery but not the CNS after subcutaneous administration. Suppression of polyQ-AR in the periphery rescued deficits in muscle weight, fiber size, and grip strength, reversed changes in muscle gene expression, and extended the lifespan of mutant males. We conclude that polyQ-AR expression in the periphery is an important contributor to pathology in SBMA mice and that peripheral administration of therapeutics should be explored for SBMA patients. Copyright © 2014 The Authors. Published by Elsevier Inc. All rights reserved.
This article was published in Cell Rep and referenced in Brain Disorders & Therapy

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