Research progress of small molecular drugs targeting RNA

VernacularTitle:以RNA为靶标的小分子药物的研究进展
Author: Xiao-li DU ; Hui-hui CHEN ; Xiang-yang YE ; Tian XIE ; Xing-rui HE
Publication Type:Research Article
Keywords: RNA; small molecular drug; targeted RNA small molecular drug; screening method; novel coronavirus
From: Acta Pharmaceutica Sinica 2022;57(10):2902-2913
CountryChina
Language:Chinese
Abstract: At present, majority of the small molecular drugs used in clinics target proteins, they exert the efficacy through the binding to specific sites on the target protein. However, the "druggable" protein targets account for a small portion of the total number of proteins, and "non-druggable" proteins account for 80%, because of not having suitable drug binding sites. In the central rule, RNA is located in the upstream of proteins and controls the transcription of proteins. The research of small molecule drugs targeting RNA can solve the problem of protein "undruggable proteins" in some extent. This review summarizes the representative research achievements of small molecular drugs targeting RNA in recent years, and the screening methods applied to this field, with the focuses on the latest progress of small molecular drugs targeting novel coronavirus RNA.