Carbamoyl-phosphate synthetase 1-associated regulatory RNA (CPS1-IT1)

Carbamoyl-phosphate synthetase 1 (CPS1)-associated regulatory RNAs, primarily the long non-coding RNA CPS1-IT1, are essential epigenetic regulators of liver metabolism and oncogenesis. CPS1-IT1 is transcribed from the intronic region of the CPS1 gene, which encodes the rate-limiting enzyme of the urea cycle (Wang et al., 2014). In hepatocellular carcinoma (HCC), CPS1-IT1 is frequently downregulated, and its loss is associated with poor prognosis and increased metastasis (Li et al., 2015). The primary biological function of these RNAs involves the modulation of the CPS1 enzyme and the sequestration of proteins like Hsp90. By binding to Hsp90, CPS1-IT1 prevents the stabilization of HIF-1α, thereby inhibiting the epithelial-mesenchymal transition and tumor growth (Wang et al., 2014). These regulatory RNAs also play a role in maintaining ammonia homeostasis by influencing the urea cycle's efficiency. Although no clinical drugs currently target these RNAs, they are being explored as therapeutic targets for RNA-based interventions such as antisense oligonucleotides or gene therapy. Restoring CPS1-IT1 levels represents a potential strategy to suppress liver cancer progression and manage metabolic dysfunction (Zhang et al., 2020). They also serve as promising diagnostic and prognostic biomarkers in liver-related malignancies.

Other names

CPS1-IT1Carbamoyl-phosphate synthetase 1 intronic transcript 1lncRNA CPS1-IT1CPS1-associated lncRNA

Therapeutic strategies involve the restoration of CPS1-IT1 expression via gene therapy or viral vectors to suppress oncogenic signaling, or the use of RNA-based tools to modulate CPS1 enzyme activity and urea cycle flux.