Infection Medicine, University of Edinburgh, The Chancellor's Building, 49 Little France Crescent, Edinburgh EH16 4SB, UK.
Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, 718 East Haizhou Rd., Haining 314400, China.
Int J Mol Sci. 2020 Apr 23;21(8):2996. doi: 10.3390/ijms21082996.
RNA-binding proteins (RBPs) are involved in regulating all aspects of RNA metabolism, including processing, transport, translation, and degradation. Dysregulation of RNA metabolism is linked to a plethora of diseases, such as cancer, neurodegenerative diseases, and neuromuscular disorders. Recent years have seen a dramatic shift in the knowledge base, with RNA increasingly being recognised as an attractive target for precision medicine therapies. In this article, we are going to review current RNA-targeted therapies. Furthermore, we will scrutinise a range of drug discoveries targeting protein-RNA interactions. In particular, we will focus on the interplay between Lin28 and let-7, splicing regulatory proteins and survival motor neuron (SMN) pre-mRNA, as well as HuR, Musashi, proteins and their RNA targets. We will highlight the mechanisms RBPs utilise to modulate RNA metabolism and discuss current high-throughput screening strategies. This review provides evidence that we are entering a new era of RNA-targeted medicine.
RNA 结合蛋白（RBPs）参与调节 RNA 代谢的各个方面，包括加工、运输、翻译和降解。RNA 代谢的失调与多种疾病有关，如癌症、神经退行性疾病和神经肌肉疾病。近年来，知识基础发生了巨大转变，RNA 越来越被认为是精准医学治疗的一个有吸引力的靶点。在本文中，我们将回顾当前的 RNA 靶向治疗方法。此外，我们还将仔细研究一系列针对蛋白-RNA 相互作用的药物发现。特别是，我们将重点关注 Lin28 和 let-7、剪接调节蛋白和运动神经元存活（SMN）前体 RNA 以及 HuR、Musashi 蛋白及其 RNA 靶标之间的相互作用。我们将强调 RBPs 用于调节 RNA 代谢的机制，并讨论当前的高通量筛选策略。这篇综述提供的证据表明，我们正在进入 RNA 靶向医学的新时代。
