微小 RNA 及其他非编码 RNA 作为抗癌药物研发的靶点。
MicroRNAs and other non-coding RNAs as targets for anticancer drug development.
机构信息
1] Experimental Therapeutics and Leukemia Department, MD Anderson Cancer Center, University of Texas, Houston, Texas 77030, USA. [2].
出版信息
Nat Rev Drug Discov. 2013 Nov;12(11):847-65. doi: 10.1038/nrd4140.
Abstract
The first cancer-targeted microRNA (miRNA) drug - MRX34, a liposome-based miR-34 mimic - entered Phase I clinical trials in patients with advanced hepatocellular carcinoma in April 2013, and miRNA therapeutics are attracting special attention from both academia and biotechnology companies. Although miRNAs are the most studied non-coding RNAs (ncRNAs) to date, the importance of long non-coding RNAs (lncRNAs) is increasingly being recognized. Here, we summarize the roles of miRNAs and lncRNAs in cancer, with a focus on the recently identified novel mechanisms of action, and discuss the current strategies in designing ncRNA-targeting therapeutics, as well as the associated challenges.
摘要
首个针对癌症的 microRNA（miRNA）药物——MRX34，一种基于脂质体的 miR-34 模拟物，于 2013 年 4 月进入晚期肝细胞癌的 I 期临床试验，miRNA 疗法正引起学术界和生物技术公司的特别关注。尽管 miRNA 是迄今为止研究最多的非编码 RNA（ncRNA），但长非编码 RNA（lncRNA）的重要性也日益受到重视。在这里，我们总结了 miRNA 和 lncRNA 在癌症中的作用，重点介绍了最近发现的新作用机制，并讨论了设计 ncRNA 靶向治疗的当前策略以及相关挑战。
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