Target intelligence / Profile preview

Cytidine residues on target RNA (RNA-C)

Target
RNA-C
Molecular classification
Nucleic acid, RNA component, Substrate
01

Overview

Cytidine residues on target RNA are fundamental components of the transcriptome, serving as building blocks for messenger RNA (mRNA), transfer RNA (tRNA), and ribosomal RNA (rRNA). Beyond their structural role, these residues are specific sites for post-transcriptional modifications, most notably the deamination of cytidine to uridine (C-to-U editing). This process is naturally catalyzed by the APOBEC (Apolipoprotein B mRNA editing enzyme, catalytic polypeptide-like) family of enzymes, which plays a vital role in physiological processes such as lipid metabolism and innate immunity (Lerner et al., 2018). However, aberrant cytidine modification or APOBEC overactivity is frequently associated with mutational signatures in various cancers and the progression of viral infections (Swanton et al., 2015). In modern biotechnology, specific cytidine residues are targeted by RNA base editing technologies, such as RESCUE (RNA Editing for Specific C-to-U Exchange), which utilize CRISPR-Cas systems fused to deaminase domains (Abudayyeh et al., 2019). These therapeutic tools allow for the precise correction of pathogenic mutations at the RNA level, offering a transient and potentially safer alternative to DNA editing. By targeting specific cytidine residues, researchers can restore protein function or modulate splicing patterns to treat genetic diseases like muscular dystrophy or cystic fibrosis (Cox et al., 2017). The interaction between these therapeutic agents and the target cytidine is governed by guide RNA complementarity, ensuring high specificity for the intended residue within a complex cellular environment.

Other names
RNA cytidineCytosine in RNAC-residuesRibocytidineTargeted RNA cytidine
02

Mechanism of action

Site-specific hydrolytic deamination of the C4 position of the cytidine pyrimidine ring to form uridine, thereby altering the genetic information at the transcript level (C-to-U editing).

03

Biological functions

Genetic codingProtein synthesisRNA splicing regulationSubstrate for RNA editingRNA stabilityInnate immune signaling
04

Disease associations

CancerGenetic disordersViral infectionNeurological disordersMetabolic diseases
05

Safety considerations

Off-target RNA editingInnate immune response to RNA-targeting agentsUnintended splicing alterationsSaturation of endogenous RNA processing machineryDelivery vehicle toxicity
06

Interacting drugs

RNA base editors (e.g., RESCUE)

4 more in the full profile.

07

Biomarkers

C-to-U editing efficiencyRNA expression levelsTarget transcript sequence variantsAPOBEC expression levels

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