Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF2

Spinal and bulbar muscular atrophy (SBMA) is a neurodegenerative disease caused by aggregation of the androgen receptor (AR) protein. Here Gen Sobue and colleagues show that upregulation of miR-196a can reduce expression of AR mRNA and ameliorate disease symptoms in mouse models of SBMA.