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Carbamoyl-phosphate synthetase 1 intronic transcript 1 (CPS1-IT1)

Target
CPS1-IT1
Molecular classification
Long non-coding RNA
01

Overview

Carbamoyl-phosphate synthetase 1 intronic transcript 1 (CPS1-IT1) is a long non-coding RNA (lncRNA) transcribed from the intronic region of the CPS1 gene, which encodes the rate-limiting enzyme of the urea cycle (NCBI Gene ID: 100506640). CPS1-IT1 acts as a potent tumor suppressor, and its expression is frequently reduced in various malignancies, most notably hepatocellular carcinoma (HCC), colorectal cancer, and lung cancer (Wang et al., 2014, J Exp Clin Cancer Res; Zhang et al., 2016, Tumour Biol). Mechanistically, CPS1-IT1 binds to heat shock protein 90 (Hsp90), interfering with its ability to stabilize client proteins like hypoxia-inducible factor 1-alpha (HIF-1α), which in turn suppresses tumor growth, epithelial-mesenchymal transition (EMT), and metastasis (Wang et al., 2014). Additionally, CPS1-IT1 may play a role in metabolic homeostasis by influencing the expression or activity of its host gene, CPS1, thereby affecting ammonia detoxification (Li et al., 2020, Eur Rev Med Pharmacol Sci). While no therapeutic agents currently target CPS1-IT1 in clinical settings, it is a significant focus of research as a diagnostic and prognostic biomarker, as well as a candidate for RNA-based therapeutic restoration in oncology.

Other names
CPS1-IT1CPS1 intronic transcript 1Carbamoyl-phosphate synthetase 1 regulatory RNAlncRNA-CPS1-IT1
02

Mechanism of action

Inhibition of the Hsp90/HIF-1α signaling pathway and potential modulation of CPS1 enzyme expression to regulate the urea cycle.

03

Biological functions

Tumor suppressionRegulation of gene expressionMetabolic regulationProtein-protein interaction
04

Disease associations

Hepatocellular carcinomaColorectal cancerLung cancerPancreatic cancer
05

Safety considerations

Off-target effects of RNA-based therapiesDelivery challenges to target tissuesPotential disruption of the urea cycle if overexpressed in non-target tissues
06

Biomarkers

CPS1-IT1 expression levelsHIF-1α protein levels

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