Research progress of small molecular drugs targeting RNA

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. © 2022, Chinese Pharmaceutical Association. All rights reserved."