RNA and RNA-Protein Complexes as Targets for Therapeutic Intervention

Published

May 1, 2002

Author(s)

E S. DeJong, B. Luy, John Marino

Abstract

Today, the majority of pharmaceuticals developed to treat cancers and viral/bacterial infections target cellular, bacterial or viral proteins known to be associated with a given pathology. Although proteins are the focus of most current drug discovery efforts, an increasing understanding of the central role of ribonucleic acid (RNA) and RNA-protein interactions in many biological processes and diseases, as well as a better understanding of RNA structure and an improvement in biophysical/biochemical techniques available to study RNA, have recently spurred exciting new research efforts aimed at exploiting RNAs and ribonucleoprotein (RNP) particles as novel and potentially powerful targets for pharmaceutical development. As for protein targets, recent genome sequencing efforts have also greatly accelerated the identification of human and microbial RNA transcripts for targeted drug discovery. With this explosion in the number of potential RNA targets, the effective development of specific small molecule RNA-based drugs now requires robust and general approaches for detecting and quantifying RNA-ligand interactions, which can be used as high-throughput screens (HTS) and for obtaining rapid structural information to guide rational drug design. In this review, an overview of the potential for therapeutic intervention based on RNA and RNP targets is presented, together with recent efforts to develop generally useful nuclear magnetic resonance (NMR) and fluorescence binding assays for screening and optimizing drugs aimed at RNA and RNPs.

Citation

Current Medicinal Chemistry

Volume

2

Issue

No. 3

Pub Type

Journals

Keywords

fluorescence, high-throughput screening, NMR, RNA

Citation

DeJong, E. , Luy, B. and Marino, J. (2002), RNA and RNA-Protein Complexes as Targets for Therapeutic Intervention, Current Medicinal Chemistry (Accessed October 13, 2025)