作　　者： ; ; ; (王江煌）; (于暕辰）;

机构地区： 中山大学中山医学院

出　　处： 《中国科学：生命科学》 2016年第12期1354-1369,共16页

摘　　要： 药物靶点的研究一直是新药研发中最重要的内容之一,microRNA(miRNA)作为调节基因与蛋白之间相互关系的纽带无疑是药物靶点研究的重要对象,以它们作为靶点的药物设计和药物研发工作也在不断深入,并获得了一系列新发现.其中,对细胞内miRNA的表达或者功能具有调控活性的化学小分子的研究很值得关注.这些化学小分子不但可以用于研究细胞内miRNA的调控网络,而且基于其对重大疾病,如肿瘤、心脏疾病、神经性疾病等相关的miRNA的调控,可能在将来的疾病治疗方面有着潜在的重要作用.本文围绕miRNA化学小分子抑制剂及其筛选方法进行综述,通过总结归纳现有的研究进展,希望对今后的miRNA特异性小分子抑制剂的发现和研究提供借鉴. MicroRNAs （miRNAs） are a type of non-coding RNA （ncRNA） transcripts and regarded as potential targets for the development of novel drugs, because they are critical regulators of gene expression by hindering translation in pathological conditions. Development of efficient miRNA-inhibiting strategies has become a promising challenge and significant advances has been made in this decade. Among these achievements, small molecule inhibitors of miRNAs are the noteworthy findings, which contribute to the studies of modulation of miRNAs regulatory networks and therapeutic strategies for serious diseases, such as cancer, angiocardiopathy and neurologic disease. In this review, we present the development of research on small molecule inhibitors of miRNAs and screening strategies for novel small molecule inhibitors. We hope it will improve the discovery of small molecule inhibitors of miRNAs and facilitate their clinical translation into disease therapeutics.

关 键 词： 化学小分子抑制剂 荧光素酶报告系统 分子信标 计算机辅助药物设计 二维组合筛选

领　　域： [生物学]