Article
Silencing neuronal mutant androgen receptor in a mouse model of spinal and bulbar muscular atrophy.
Human molecular genetics - 1 Nov 2015
Sahashi Kentaro, Katsuno Masahisa, Hung Gene, Adachi Hiroaki, Kondo Naohide, Nakatsuji Hideaki, Tohnai Genki, Iida Madoka, Bennett C Frank, Sobue Gen
Abstract excerpt
Spinal and bulbar muscular atrophy (SBMA), an adult-onset neurodegenerative disease that affects males, results from a CAG triplet repeat/polyglutamine expansions in the androgen receptor (AR) gene. Patients develop progressive muscular weakness and atrophy, and no effective therapy is currently available. The tissue-specific pathogenesis, especially relative pathological contributions between degenerative motor...
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