Yang Ming, Mattes Joerg
Centre for Asthma and Respiratory Diseases, School of Biomedical Sciences, Faculty of Health, University of Newcastle and Hunter Medical Research Institute, Callaghan, NSW 2300, Australia.
Pharmacol Ther. 2008 Jan;117(1):94-104. doi: 10.1016/j.pharmthera.2007.08.004. Epub 2007 Sep 7.
The discovery of small RNA molecules as regulators of posttranscriptional gene silencing has paved the way to specifically target any given protein via the RNA interference (RNAi) pathway. An endogenous class of these molecules, the microRNA (miRNA), is proposed to control expression of up to one third of all genes and may be utilized as diagnostic and prognostic marker for diseases. In addition the recent employment of antagomirs that specifically inhibit function of a given miRNA represents a powerful tool to determine the role of these molecules in disease pathogenesis. Here, we describe our current understanding of the structure, biogenesis and function of small RNA, as well as their potential and limitation as novel therapeutic approaches.
小RNA分子作为转录后基因沉默的调节因子的发现,为通过RNA干扰(RNAi)途径特异性靶向任何给定蛋白质铺平了道路。这类分子中的内源性一类,即微小RNA(miRNA),被认为可控制多达三分之一的所有基因的表达,并可用作疾病的诊断和预后标志物。此外,最近使用的特异性抑制给定miRNA功能的抗miR分子是确定这些分子在疾病发病机制中作用的有力工具。在这里,我们描述了我们目前对小RNA的结构、生物发生和功能的理解,以及它们作为新型治疗方法的潜力和局限性。