Article
[Molecular-targeted therapies for spinal and bulbar muscular atrophy].
Rinsho shinkeigaku = Clinical neurology - 1 Nov 2009
Katsuno Masahisa, Banno Haruhiko, Suzuki Keisuke, Adachi Hiroaki, Tanaka Fumiaki, Sobue Gen
Abstract excerpt
Spinal and bulbar muscular atrophy (SBMA) is a polyglutamine-mediated lower motor neuron disease characterized by slowly progressive muscle weakness and atrophy. The cause of this disease is the expansion of a trinucleotide CAG repeat, which encodes the polyglutamine tract, within the first exon of the androgen receptor (AR) gene. SBMA exclusively occurs in adult males, whereas both heterozygous and homozygous...
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