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Updated: Feb 8, 2026
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A Rapid and Quantitative Fluorimetric Method for Protein-Targeting Small Molecule Drug Screening
Published on: October 16, 2015
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Principles for targeting RNA with drug-like small molecules
Katherine Deigan Warner1, Christine E Hajdin1, Kevin M Weeks2
1Ribometrix, Durham, NC, USA.
Nature Reviews. Drug Discovery
|July 7, 2018
Summary
Small molecules can target RNA for disease treatment, offering new therapeutic avenues. Focusing on disease-causing RNAs with druggable pockets makes RNA drug discovery feasible.
Area of Science:
- Molecular Biology
- Medicinal Chemistry
- Drug Discovery
Background:
- RNA molecules are crucial for cellular functions like information transfer and gene regulation.
- Structured elements within RNAs are vital for their biological roles.
- RNAs are implicated in various human diseases, presenting therapeutic targets.
Purpose of the Study:
- To discuss principles for discovering small-molecule drugs that target RNA.
- To highlight the challenge of identifying suitable RNA target structures.
- To propose that focusing on druggable binding sites in disease-causing RNAs can enable effective small-molecule drug discovery.
Main Methods:
- Review of current literature on small-molecule RNA ligands.
- Analysis of RNA structure-function relationships.
- Discussion of physicochemical properties relevant to drug design.
Main Results:
- Currently, only one class of small molecules (linezolid antibiotics) targets RNA clinically.
- Numerous small-molecule RNA ligands are being identified, increasing field interest.
- Disease-causing RNAs with high information content possess druggable binding pockets.
Conclusions:
- Targeting RNA with small molecules offers potential for modulating cellular processes and addressing 'undruggable' protein targets.
- Identifying appropriate, druggable binding sites in disease-causing RNAs is the primary challenge.
- RNA drug discovery can become as feasible as protein drug discovery by focusing on these specific binding sites.
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