| Bioactivity | SPC5001 is a locked nucleic acid (LNA)-modifed antisense oligonucleotide (ASO) complementary to human PCSK9 (proprotein convertase subtilisin/kexin type 9) mRNA. SPC5001 can be used for the research of hypercholesterolemia. SPC5001 sequence: 5′-TGmCTACAAAACmCmCA-3′[1]. |
| Invitro | SPC5001 (0.16-40 µM; 72 h) induces the downregulation of PCSK9 mRNA targets but cytotoxicity is not observed at any concentration in 2D human renal proximal tubule epithelial cells (HRPTEC) cultures[1].SPC5001 (5 µM; 48 h or 20 d) downregulates PCSK9 mRNA in chip-cultured HRPTEC, with 74.1% and 93.7% for 48 h and 20 days, respectively[1].SPC5001 (5 µM; 20 d) induces cytotoxicity and increases perfusate levels of biomarkers KIM-1, NGAL, clusterin, osteopontin and VEGF in chip-cultured HRPTEC[1]. |
| Name | SPC5001 |
| Molar Mass | 4689.85 |
| Appearance | Solid |
| Transport | Room temperature in continental US; may vary elsewhere. |
| Storage | -20°C, sealed storage, away from moisture *In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture) |
| Reference | [1]. Nieskens TTG, et, al. Nephrotoxic antisense oligonucleotide SPC5001 induces kidney injury biomarkers in a proximal tubule-on-a-chip. Arch Toxicol. 2021 Jun;95(6):2123-2136. [2]. Lindholm MW, Elmén J, Fisker N, et al. PCSK9 LNA antisense oligonucleotides induce sustained reduction of LDL cholesterol in nonhuman primates. Mol Ther. 2012;20(2):376-381. |