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RNA Interference

RNA Interference

RNA interference (RNAi) is a process in which a small non-coding RNA molecule blocks the post-transcriptional expression of a gene by binding to its messenger RNA (mRNA) and preventing the protein from being translated.
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RNA Structure

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RNA Editing

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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...

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RNA Polymerase (RNAP) is conserved in all animals, with bacterial, archaeal, and eukaryotic RNAPs sharing significant sequence, structural, and functional similarities. Among the three eukaryotic RNAPs, RNA Polymerase II is most similar to bacterial RNAP in terms of both structural organization and folding topologies of the enzyme subunits. However, these similarities are not reflected in their mechanism of action.
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Updated: Feb 3, 2026

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes

RNA Catalyst as a Reporter for Screening Drugs against RNA Editing in Trypanosomes

Published on: July 22, 2014

Drugging the RNA World.

Matthew D Disney¹, Brendan G Dwyer¹, Jessica L Childs-Disney¹

¹Department of Chemistry, The Scripps Research Institute, Jupiter, Florida 33458.

Cold Spring Harbor Perspectives in Biology

|November 3, 2018

Summary

This summary is machine-generated.

Designing small molecules to target RNA, though challenging, is now achievable. This review explores methods for creating RNA-targeting compounds and probes for RNA biology, focusing on inhibiting RNA-protein complexes.

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Identification of Footprints of RNA:Protein Complexes via RNA Immunoprecipitation in Tandem Followed by Sequencing RIPiT-Seq

Identification of Footprints of RNA:Protein Complexes via RNA Immunoprecipitation in Tandem Followed by Sequencing RIPiT-Seq

Published on: July 10, 2019

Area of Science:

Biochemistry
Molecular Biology
Medicinal Chemistry

Background:

The field of drug discovery predominantly focuses on proteins, overlooking RNA as a therapeutic target.
Developing selective small molecules for RNA is a significant challenge due to RNA's complex structure and function.
RNA plays crucial roles in gene regulation and cellular processes, making it a promising target for novel therapeutics.

Purpose of the Study:

To demystify the design of small molecules that target RNA.
To review various approaches for designing RNA-targeting small molecules and their applications in RNA biology.
To highlight the potential of small molecules in inhibiting human RNA-protein complexes and modulating disease-causing RNAs.

Main Methods:

Library-versus-library screening approach to identify selective RNA-small-molecule binding partners.
Application of designed small molecules to disease-causing RNAs, including noncoding oncogenic RNAs and expanded RNA repeats.
Development of novel small-molecule probes for deciphering RNA functions in cellular contexts.

Main Results:

Demonstrated success in designing selective small molecules that bind to specific RNA targets.
Modulation of disease-associated RNA biology in cellular and animal models using these small molecules.
Successful application of screening approaches to identify RNA-small-molecule interactions.

Conclusions:

Small molecule-based targeting of RNA is a feasible and powerful strategy in drug discovery.
The presented approaches offer new avenues for developing therapeutics against RNA-related diseases.
Further development of RNA-targeting small molecules and probes will unlock new possibilities in understanding and manipulating RNA biology.