Rácz Zs, Kaucsár T, Hamar P
Semmelweis University Institute of Pathophysiology, Faculty of Medicine, Budapest, Hungary.
Acta Physiol Hung. 2011 Sep;98(3):243-51. doi: 10.1556/APhysiol.98.2011.3.1.
MicroRNAs (miRNAs) are a recently discovered class of small, non-coding RNAs which do not code proteins. MiRNAs regulate gene expression by inhibiting protein translation from the messenger RNA. MiRNAs may function in networks, forming a complex relationship with diseases. Furthermore, specific miRNAs have significant correlation with diseases of divergent origin. After identification of disease-associated miRNAs, their tissue expression could be altered in a beneficial way by inhibiting or mimicking their effects. Thus, modifying the expression of miRNAs is a potential future gene-therapeutic tool to influence post-transcriptional regulation of multiple genes in a single therapy. In this review we introduce the biogenesis, mechanism of action and future aspects of miRNAs. Research on the post-transcriptional regulation of gene expression by miRNA may reshape our understanding of diseases and consequently may bring new diagnostic markers and therapeutic agents. Therapeutic use of miRNAs is already under clinical investigation in RNA interference trials.
微小RNA(miRNA)是最近发现的一类小型非编码RNA,它们不编码蛋白质。miRNA通过抑制信使核糖核酸的蛋白质翻译来调节基因表达。miRNA可能在网络中发挥作用,与疾病形成复杂的关系。此外,特定的miRNA与多种起源的疾病具有显著相关性。在鉴定出与疾病相关的miRNA后,通过抑制或模拟它们的作用,可以以有益的方式改变其组织表达。因此,改变miRNA的表达是一种潜在的未来基因治疗工具,可在单一治疗中影响多个基因的转录后调控。在本综述中,我们介绍了miRNA的生物合成、作用机制和未来展望。关于miRNA对基因表达的转录后调控的研究可能会重塑我们对疾病的理解,从而可能带来新的诊断标志物和治疗药物。miRNA的治疗用途已经在RNA干扰试验中进行临床研究。