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′.
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].
















