REVIEW article
Front. Chem.
Sec. Medicinal and Pharmaceutical Chemistry
Volume 13 - 2025 | doi: 10.3389/fchem.2025.1649692
This article is part of the Research TopicRecent Advances in the Design of Heterocyclic Modulators of Druggable EnzymesView all 3 articles
Targeting RNA-binding proteins with small molecules: perspectives and challenges
Provisionally accepted
- University of Kinshasa, Kinshasa, Democratic Republic of Congo
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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 emerging 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.
Keywords: Drug Discovery, RNA-Binding Proteins, small molecules, RNA-protein interactions, PROTACs, Natural Products, molecular glues
Received: 19 Jun 2025; Accepted: 02 Sep 2025.
Copyright: © 2025 Konde, Balela and Tshitenge. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Tania Bishola Tshitenge, University of Kinshasa, Kinshasa, Democratic Republic of Congo
Disclaimer: All claims expressed in this article are solely those of the authors and do not necessarily represent those of their affiliated organizations, or those of the publisher, the editors and the reviewers. Any product that may be evaluated in this article or claim that may be made by its manufacturer is not guaranteed or endorsed by the publisher.