RNA-Binding Small Molecules in Drug Discovery and Delivery: An Overview from Fundamentals,Journal of Medicinal Chemistry

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RNA-Binding Small Molecules in Drug Discovery and Delivery: An Overview from Fundamentals
Journal of Medicinal Chemistry ( IF 6.8 ) Pub Date : 2024-09-17 , DOI: 10.1021/acs.jmedchem.4c01330
Siyi Chen _{1,

2} , Qi Mao _{1,

2} , Hong Cheng _{1,

2} , Wanyi Tai _{1,

2}

Affiliation

RNA molecules, similar to proteins, fold into complex structures to confer diverse functions in cells. The intertwining of functions with RNA structures offers a new therapeutic opportunity for small molecules to bind and manipulate disease-relevant RNA pathways, thus creating a therapeutic realm of RNA-binding small molecules. The ongoing interest in RNA targeting and subsequent screening campaigns have led to the identification of numerous compounds that can regulate RNAs from splicing, degradation to malfunctions, with therapeutic benefits for a variety of diseases. Moreover, along with the rise of RNA-based therapeutics, RNA-binding small molecules have expanded their application to the modification, regulation, and delivery of RNA drugs, leading to the burgeoning interest in this field. This Perspective overviews the emerging roles of RNA-binding small molecules in drug discovery and delivery, covering aspects from their action fundamentals to therapeutic applications, which may inspire researchers to advance the field.

中文翻译：

药物发现和递送中的 RNA 结合小分子：基础概述

RNA 分子与蛋白质类似，折叠成复杂的结构，在细胞中赋予不同的功能。功能与 RNA 结构的交织为小分子结合和操纵疾病相关的 RNA 通路提供了新的治疗机会，从而创造了 RNA 结合小分子的治疗领域。对 RNA 靶向和后续筛选活动的持续兴趣导致鉴定出许多可以调节 RNA 从剪接、降解到功能障碍的化合物，对多种疾病具有治疗益处。此外，随着基于 RNA 的疗法的兴起，RNA 结合小分子已将其应用扩展到 RNA 药物的修饰、调节和递送，导致人们对该领域的兴趣日益浓厚。本展望概述了 RNA 结合小分子在药物发现和递送中的新兴作用，涵盖了从其作用基础到治疗应用的方面，这可能会激励研究人员推进该领域。

更新日期：2024-09-17

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