The miRNA pathway in neurological and skeletal muscle disease: implications for pathogenesis and therapy

Abstract

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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Acknowledgments

C.R.S. is supported by funding from Parkinson’s UK; M.J.A.W. is supported by funding from the UK MRC, The Wellcome Trust, Parkinson’s UK, the Muscular Dystrophy Campaign and Action Duchenne. The authors would like to thank Thomas Roberts for the critical reading of this manuscript.

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Correspondence to Matthew J. A. Wood.

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Sibley, C.R., Wood, M.J.A. The miRNA pathway in neurological and skeletal muscle disease: implications for pathogenesis and therapy. J Mol Med 89, 1065 (2011). https://doi.org/10.1007/s00109-011-0781-z

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Keywords

  • RNAi
  • miRNA
  • Neurodegeneration
  • Myopathy
  • Muscular dystrophy