Target intelligence / Profile preview

Structured viral RNA

Molecular classification
RNA, Viral genetic element, Non-coding RNA structure
01

Overview

Structured viral RNA refers to the complex secondary and tertiary folds within viral genomes, such as the HIV-1 Trans-activation response (TAR) element or the Hepatitis C Virus (HCV) Internal Ribosome Entry Site (IRES), which are essential for viral replication and protein synthesis (Hermann, 2016). These structures serve as critical scaffolds for the assembly of viral and host protein complexes, making them attractive therapeutic targets for small molecules and antisense oligonucleotides (Butcher & Pyle, 2011). By binding to these specific RNA motifs, drugs can sterically hinder essential interactions or stabilize non-functional conformations, thereby inhibiting the viral life cycle (Donlic & Hargrove, 2018). In the context of drug discovery, these well-characterized viral RNAs are often used as surrogates to study the selectivity and affinity of small molecules toward RNA in general. This approach helps in identifying chemical scaffolds capable of discriminating between different RNA structures, which is a major challenge in developing RNA-targeted therapies (Warner et al., 2018).

Other names
Viral RNA secondary structureStructured RNA motifsRNA regulatory elementsViral RNA tertiary structureRNA-targeted therapeutic motifs
02

Mechanism of action

Small molecule binding to specific RNA secondary or tertiary structures to sterically hinder protein-RNA interactions or induce conformational changes that inhibit viral replication or translation (Hermann, 2016; Donlic & Hargrove, 2018).

03

Biological functions

Viral replicationTranslation initiationRibosomal frameshiftingGenome packagingTranscription regulation
04

Disease associations

InfectionViral disease (e.g., HIV/AIDS, Hepatitis C, COVID-19)
05

Safety considerations

Off-target binding to host cellular RNA (rRNA, mRNA, tRNA)High mutation rates leading to rapid drug resistanceSystemic toxicity due to lack of specificityDelivery challenges for RNA-binding molecules
06

Interacting drugs

Risdiplam

4 more in the full profile.

07

Biomarkers

Viral loadViral RNA titersViral protein expression levels

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