Targeting RNA‐binding proteins with small molecules: Perspectives, pitfalls and bifunctional molecules

Abstract

RNA‐binding proteins (RBPs) play vital roles in organisms through binding with RNAs to regulate their functions. Small molecules affecting the function of RBPs have been developed, providing new avenues for drug discovery. Herein, we describe the perspectives on developing small molecule regulators of RBPs. The following types of small molecule modulators are of great interest in drug discovery: small molecules binding to RBPs to affect interactions with RNA molecules, bifunctional molecules binding to RNA or RBP to influence their interactions, and other types of molecules that affect the stability of RNA or RBPs. Moreover, we emphasize that the bifunctional molecules may play important roles in small molecule development to overcome the challenges encountered in the process of drug discovery.

Fibroblast-Specific Depletion of Human Antigen R Alleviates Myocardial Fibrosis Induced by Cardiac Stress

“…However, in recent years, several small molecules have been screened that inhibit RBP function by modulating RBP-RNA interaction and dynamics of RBPs. 56 These small molecule inhibitors could be valuable tools for developing novel therapeutics for various diseases. Several small molecules have also been investigated to inhibit HuR function in various diseases, especially in cancer.…”