Article
Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2.
Nature medicine - 1 Jul 2012
Miyazaki Yu, Adachi Hiroaki, Katsuno Masahisa, Minamiyama Makoto, Jiang Yue-Mei, Huang Zhe, Doi Hideki, Matsumoto Shinjiro, Kondo Naohide, Iida Madoka, Tohnai Genki, Tanaka Fumiaki, Muramatsu Shin-ichi, Sobue Gen
Abstract excerpt
Spinal and bulbar muscular atrophy (SBMA) is an inherited neurodegenerative disorder caused by the expansion of the polyglutamine (polyQ) tract of the androgen receptor (AR-polyQ). Characteristics of SBMA include proximal muscular atrophy, weakness, contraction fasciculation and bulbar involvement. MicroRNAs (miRNAs) are a diverse class of highly conserved small RNA molecules that function as crucial regulators...
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