Targeting RNA-binding proteins with small molecules: perspectives and challenges.

IF 4.2 3区化学 Q2 CHEMISTRY, MULTIDISCIPLINARY

Frontiers in Chemistry Pub Date : 2025-10-02 eCollection Date: 2025-01-01 DOI:10.3389/fchem.2025.1649692

Olivier Mpungi Konde, Williams Balela Balela, Tania Bishola Tshitenge

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Abstract

RNA-binding proteins (RBPs) play critical roles in numerous biological processes because they regulate RNA function by directly interacting with RNA molecules. In recent years, researchers have developed small molecules that can affect the function of RBPs, opening up promising new directions for drug discovery. While several reviews have already explored this topic, here we aim to provide additional perspectives and highlight emerging challenges in the area of targeting RBPs. There are several types of small molecule modulators that are particularly developing in this field. These include molecules that bind directly to RBPs and alter their interaction with RNA, bifunctional molecules that associate to either RNA or RBPs to disrupt or enhance their interaction, and other compounds that affect the stability of either the RNA or the RBP itself. Among these, bifunctional molecules stand out as especially promising, as they offer potential solutions to some of the common challenges faced in developing drugs targeting RBPs.

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靶向小分子rna结合蛋白：观点和挑战。

RNA结合蛋白（rbp）通过直接与RNA分子相互作用来调节RNA的功能，在许多生物过程中起着至关重要的作用。近年来，研究人员已经开发出可以影响rbp功能的小分子，为药物发现开辟了有希望的新方向。虽然已经有几篇综述探讨了这一主题，但在这里，我们的目标是提供额外的观点，并强调针对rbp领域的新挑战。有几种类型的小分子调节剂在这一领域得到了特别的发展。这些包括直接与RBP结合并改变其与RNA相互作用的分子，与RNA或RBP结合以破坏或增强其相互作用的双功能分子，以及影响RNA或RBP本身稳定性的其他化合物。其中，双功能分子尤其有前景，因为它们为开发针对rbp的药物所面临的一些共同挑战提供了潜在的解决方案。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Frontiers in Chemistry Chemistry-General Chemistry

CiteScore

8.50

自引率

3.60%

发文量

1540

审稿时长

12 weeks

期刊介绍： Frontiers in Chemistry is a high visiblity and quality journal, publishing rigorously peer-reviewed research across the chemical sciences. Field Chief Editor Steve Suib at the University of Connecticut is supported by an outstanding Editorial Board of international researchers. This multidisciplinary open-access journal is at the forefront of disseminating and communicating scientific knowledge and impactful discoveries to academics, industry leaders and the public worldwide. Chemistry is a branch of science that is linked to all other main fields of research. The omnipresence of Chemistry is apparent in our everyday lives from the electronic devices that we all use to communicate, to foods we eat, to our health and well-being, to the different forms of energy that we use. While there are many subtopics and specialties of Chemistry, the fundamental link in all these areas is how atoms, ions, and molecules come together and come apart in what some have come to call the “dance of life”. All specialty sections of Frontiers in Chemistry are open-access with the goal of publishing outstanding research publications, review articles, commentaries, and ideas about various aspects of Chemistry. The past forms of publication often have specific subdisciplines, most commonly of analytical, inorganic, organic and physical chemistries, but these days those lines and boxes are quite blurry and the silos of those disciplines appear to be eroding. Chemistry is important to both fundamental and applied areas of research and manufacturing, and indeed the outlines of academic versus industrial research are also often artificial. Collaborative research across all specialty areas of Chemistry is highly encouraged and supported as we move forward. These are exciting times and the field of Chemistry is an important and significant contributor to our collective knowledge.