Spinocerebellar ataxia: miRNAs expose biological pathways underlying pervasive Purkinje cell degeneration

[摘要] Recent work has demonstrated the importance of miRNAs in the pathogenesis of various brain disorders including the neurodegenerative disorder spinocerebellar ataxia (SCA). This review focuses on the role of miRNAs in the shared pathogenesis of the different SCA types. We examine the novel findings of a recent cell-type-specific RNA sequencing study in mouse brain and discuss how the identification of Purkinje-cell-enriched miRNAs highlights biological pathways that expose the mechanisms behind pervasive Purkinje cell degeneration in SCA. These key pathways are likely to contain targets for therapeutic development and represent potential candidate genes for genetically unsolved SCAs. (C) 2017 Elsevier Inc. All rights reserved.

[发布日期] 2017-12-01

[关键词] miRNA;Spinocerebellar ataxia;Purkinje cells;Neurodegeneration;Cerebellum