Clinical applications of microRNAs.
Clinical applications of microRNAs.
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DOI：
10.12688/f1000research.2-136.v1
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发表时间：
2013-01-01
期刊：
影响因子：
--
通讯作者：
Badalian-Very, Gayane
中科院分区：
文献类型：
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作者：
Hydbring, Per;Badalian-Very, Gayane
MicroRNA 代表一类源自聚合酶 II 控制转录区域的小 RNA。初级转录物形成一个或多个凸出的双链发夹，它们被 Drosha 和 Dicer 加工成异源双链体。双链体的靶向 microRNA 链被整合到 RNA 诱导沉默复合物中，通过诱导 mRNA 降解或阻断蛋白质翻译，使多达数百个 mRNA 转录物沉默。除了参与多种生物过程之外，microRNA 还因其在疾病诊断和治疗方面的潜力而很早就被认识到。由于其稳定性，microRNA 可用作生物标志物。目前，有一些 microRNA 组合可以帮助医生确定播散性肿瘤中的癌症起源。事实证明，microRNA 疗法的开发更具挑战性，主要是由于递送问题。然而，一种药物已经进入临床试验，还有几种药物正在等待进入临床阶段。这篇综述总结了诊断性 microRNA 的临床前和临床公认的内容。此外，它还重点介绍了由四家领先的 microRNA 治疗公司驱动的运行平台中的单个 microRNA 药物。
MicroRNAs represent a class of small RNAs derived from polymerase II controlled transcriptional regions. The primary transcript forms one or several bulging double stranded hairpins which are processed by Drosha and Dicer into hetero-duplexes. The targeting microRNA strand of the duplex is incorporated into the RNA Induced Silencing Complex from where it silences up to hundreds of mRNA transcript by inducing mRNA degradation or blocking protein translation. Apart from involvement in a variety of biological processes, microRNAs were early recognized for their potential in disease diagnostics and therapeutics. Due to their stability, microRNAs could be used as biomarkers. Currently, there are microRNA panels helping physicians determining the origins of cancer in disseminated tumors. The development of microRNA therapeutics has proved more challenging mainly due to delivery issues. However, one drug is already in clinical trials and several more await entering clinical phases. This review summarizes what has been recognized pre-clinically and clinically on diagnostic microRNAs. In addition, it highlights individual microRNA drugs in running platforms driven by four leading microRNA-therapeutic companies.