Description
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′-TGmCTACAAAppearance:SolidIn Vitro: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. SPC5001 (5 µM; 48 h or 20 d) downregulates PCSK9 mBiological Activity: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′-TGmCTACAA