hepatic gene

GSK4112 (SR6452) is a Rev-erbα agonist with an EC50 of 0.4 μM and serves as a small molecule chemical probe for studying the cell biology of the nuclear heme receptor Rev-erbα.

HPG1860, an agonist of the farnesoid X receptor (FXR), induces luminescence in reporter gene assays utilizing HEK293T cells expressing human FXR, with an EC50 value of 18 nM. In vivo studies demonstrate that HPG1860 (administered at 1, 3, or 10 mg/kg daily) reduces serum alanine aminotransferase (ALT) and total cholesterol levels in a mouse model of non-alcoholic steatohepatitis (NASH), which was induced by a high-fat diet and carbon tetrachloride (CCl4). Additionally, this compound decreases hepatic inflammation, fat levels, and fibrosis.

4-Epidoxycycline is a hepatic metabolite of the antibiotic doxycycline and lacks antibiotic properties in mice. In both in vitro assays and in vivo mouse models, 4-Epidoxycycline regulates HER2 gene expression similarly to doxycycline and exhibits comparable efficacy in tumor tissues, achieving over 95% tumor regression rates.

Anti-NASH agent 2 (compound 21) is an inhibitor of neolipogenesis activity and α-SMA gene expression. It improves hepatic steatosis, edema, inflammatory infiltration, and liver fibrosis in NASH mouse models.

Plozasiran, previously called ARO-APOC3, is a first-in-class investigational RNA interference (RNAi) therapeutic that is specifically designed to actively suppress hepatic production of apolipoprotein C-III (APOC3), a critical structural and functional component of triglyceride-rich lipoproteins (TRLs) and a central regulator of systemic triglyceride metabolism, thereby addressing dyslipidemia at the level of gene expression through targeted post-transcriptional silencing mechanisms.

Givinostat (ITF-2357) is an HDAC inhibitor with IC50 values of 198 nM for HDAC1 and 157 nM for HDAC3.

Patisiran sodium is a double-stranded small interfering RNA (siRNA) that selectively binds a defined sequence within transthyretin (TTR) messenger RNA. Patisiran sodium possess potent and specific suppression of hepatic synthesis of both mutant and wild-type TTR protein, and its mechanism-based activity underpins its significant application in hereditary TTR amyloidosis research, gene silencing therapeutics development, and RNA interference–based translational medicine.

Lumasiran (ALN-G01) is a small interfering RNA therapeutic that targets hepatic glycolate oxidase mRNA, silences enzyme expression, reduces oxalate biosynthesis. Lumasiran addresses the pathogenic metabolic defect underlying primary hyperoxaluria type 1, supporting its use in translational and mechanistic disease research.

SBI993 is an analog of SBI-477 and can be used as a biomarker to confirm the expected action of MondoA target gene expression in vivo. SBI993 reduces muscle TAG levels and hepatic steatosis.

3,7-Dimethylfumarate (3,7-DMF) serves as an oral inhibitor of transforming growth factor-beta 1 (TGF-β1)-mediated hepatic stellate cell (HSC) activation. By inducing antioxidant gene expression and scavenging reactive oxygen species (ROS), 3,7-DMF is useful for researching liver fibrosis [1].