Target intelligence / Profile preview

Carbamoyl-phosphate synthetase 1 regulatory RNA (CPS1-regRNA)

Target
CPS1-regRNA
Molecular classification
Non-coding RNA, Long non-coding RNA, MicroRNA
01

Overview

Carbamoyl-phosphate synthetase 1 (CPS1) regulatory RNAs, including long non-coding RNAs (lncRNAs) like CPS1-IT1 and various microRNAs, are essential modulators of the CPS1 gene, which encodes the rate-limiting enzyme of the urea cycle (Summar et al., 2013). These regulatory molecules control the conversion of toxic ammonia into urea, thereby maintaining nitrogen homeostasis in the liver. The lncRNA CPS1-IT1 specifically acts as a tumor suppressor in hepatocellular carcinoma; its downregulation reduces CPS1 expression, leading to ammonia accumulation and accelerated tumor cell growth (Wang et al., 2014). Additionally, microRNAs such as miR-885-5p have been shown to post-transcriptionally repress CPS1, further influencing metabolic flux and disease progression (NCBI Gene). Dysregulation of these RNA networks is implicated in both urea cycle disorders and various cancers, making them attractive targets for precision medicine. While no specific drugs are currently approved to target these RNAs, experimental approaches using antisense oligonucleotides and miRNA mimics are under investigation to restore CPS1 function (Matsui & Corey, 2017). Targeting these regulatory RNAs provides a unique opportunity to address metabolic reprogramming in liver diseases and oncology.

Other names
CPS1-IT1CPS1 intronic transcript 1lncRNA-CPS1-IT1CPS1-AS1miR-885-5pmiR-34aCarbamoyl-phosphate synthetase 1 antisense RNA 1
02

Mechanism of action

Regulatory RNAs modulate CPS1 gene expression through various mechanisms: long non-coding RNAs like CPS1-IT1 can act as transcriptional activators or scaffolds for chromatin modifiers, while microRNAs like miR-885-5p typically bind to the 3' UTR of CPS1 mRNA to induce degradation or inhibit translation (Wang et al., 2014; NCBI Gene).

03

Biological functions

Regulation of gene expressionUrea cycle regulationNitrogen metabolismCell proliferation
04

Disease associations

Hepatocellular carcinomaUrea cycle disorderHyperammonemiaCancer
05

Safety considerations

Off-target RNA interactionsPotential for inducing hyperammonemia if downregulatedLiver-specific delivery challenges for RNA therapeutics
06

Biomarkers

CPS1-IT1 expression levelPlasma ammonia levelCPS1 protein expression

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