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Updated: Jul 26, 2025

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Sequence-specific and Selective Recognition of Double-stranded RNAs over Single-stranded RNAs by Chemically Modified Peptide Nucleic Acids

Published on: September 21, 2017

Targeted Degradation of Structured RNAs via Ribonuclease-Targeting Chimeras (RiboTacs).

Salma Haj-Yahia¹, Arijit Nandi¹, Raphael I Benhamou¹

¹The Institute for Drug Research of the School of Pharmacy, Faculty of Medicine, The Hebrew University of Jerusalem, Jerusalem, Israel.

Expert Opinion on Drug Discovery

|June 12, 2023

Summary

This summary is machine-generated.

Ribonuclease-Targeting Chimeras (RiboTaCs) are a novel drug discovery approach that uses small molecules to degrade disease-associated RNA structures. This targeted RNA degradation strategy shows promise for treating various diseases.

Keywords:

RNA RNase L RiboTaC Targeted degradation Therapeutics

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Method for the Isolation and Identification of mRNAs, microRNAs and Protein Components of Ribonucleoprotein Complexes from Cell Extracts using RIP-Chip

Method for the Isolation and Identification of mRNAs, microRNAs and Protein Components of Ribonucleoprotein Complexes from Cell Extracts using RIP-Chip

Published on: September 29, 2012

Area of Science:

Biochemistry
Molecular Biology
Drug Discovery

Background:

RNA structural motifs are crucial for biological recognition and regulation.
Dysfunctional RNA structures are implicated in numerous diseases.
Targeting RNA with small molecules is an emerging therapeutic strategy.

Purpose of the Study:

To review the evolution and mechanism of Ribonuclease-Targeting Chimeras (RiboTaCs).
To summarize disease-associated RNAs targeted by RiboTaCs and their therapeutic outcomes.
To discuss the potential and challenges of RiboTaC technology in drug discovery.

Main Methods:

Review of existing literature on RiboTaC strategy.
Analysis of in vitro and in vivo validation studies.
Summary of disease-associated RNA targets and their degradation effects.

Main Results:

RiboTaCs selectively degrade structured RNA targets.
Degradation of disease-associated RNAs by RiboTaCs alleviates disease phenotypes in vitro and in vivo.
The RiboTaC strategy has demonstrated efficacy in preclinical models.

Conclusions:

RiboTaCs represent a promising targeted degradation strategy for RNA-based therapeutics.
Further development is needed to address challenges for clinical translation.
RiboTaC technology holds potential to revolutionize treatment for diverse diseases.