Department of Physiology, Anatomy and Genetics, University of Oxford, South Parks Road, Oxford, OX1 3QX, UK.
J Mol Med (Berl). 2011 Nov;89(11):1065-77. doi: 10.1007/s00109-011-0781-z. Epub 2011 Jul 13.
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.
RNA 干扰 (RNAi) 代表了一种强大的转录后基因沉默网络，可精细调节所有真核细胞中的基因表达。RNAi 的内源性触发物 microRNAs (miRNAs) 被认为可以调节多达三分之一的蛋白质编码基因的表达，并已被证明在包括中枢神经系统和骨骼肌在内的发育过程中具有关键作用。此外，许多 miRNA 已被报道在各种疾病状态下显示出差异表达。在这里，我们描述了 miRNA 的生物发生和功能的现有认识，综述了 miRNA 在人类神经和肌肉疾病中的异常情况的现有知识，并讨论了它们作为新型疾病生物标志物的潜力。最后，我们强调了 miRNA 途径可以通过多种方式被靶向用于治疗获益。
