期刊
JOURNAL OF MOLECULAR MEDICINE-JMM
卷 89, 期 11, 页码 1065-1077出版社
SPRINGER
DOI: 10.1007/s00109-011-0781-z
关键词
RNAi; miRNA; Neurodegeneration; Myopathy; Muscular dystrophy
资金
- Parkinson's UK
- UK MRC
- Wellcome Trust
- Muscular Dystrophy Campaign and Action Duchenne
RNA interference (RNAi) represents a powerful post-transcriptional gene silencing network which fine-tunes gene expression in all eukaryotic cells. The endogenous triggers of RNAi, microRNAs (miRNAs), are proposed to regulate expression of up to a third of all protein-coding genes, and have been shown to have critical roles in developmental processes including in the central nervous system and skeletal muscle. Further, many have been reported to display differential expression in various disease states. Here we describe present understanding of the biogenesis and function of miRNAs, review current knowledge of miRNA abnormalities in both human neurological and skeletal muscle disease and discuss their potential as novel disease biomarkers. Finally, we highlight the many ways in which the miRNA pathway may be targeted for therapeutic benefit.
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