PCSK9 RNA interference

PCSK9 RNA interference refers to a therapeutic approach using RNA interference (RNAi) technology, specifically small interfering RNAs (siRNAs), to target and silence the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene. This mechanism occurs primarily in the liver, where the siRNA molecules are delivered (often via GalNAc conjugation) to hepatocytes. Once inside, they incorporate into the RNA-induced silencing complex (RISC) to catalyze the cleavage of PCSK9 mRNA, preventing the translation of the PCSK9 protein. Since PCSK9 protein normally binds to and promotes the lysosomal degradation of low-density lipoprotein receptors (LDLR), its reduction leads to increased LDLR recycling to the cell surface. This results in enhanced clearance of LDL cholesterol (LDL-C) from the bloodstream. The most prominent and currently approved drug utilizing this platform is inclisiran (Leqvio), developed by Alnylam Pharmaceuticals and Novartis, which is indicated for the treatment of primary hypercholesterolemia and mixed dyslipidemia.

Other names

PCSK9 siRNAPCSK-9 siRNAPCSK 9 siRNAPCSK9 RNAiPCSK-9 RNAiPCSK 9 RNAiPCSK9 RNA interference therapyPCSK-9 RNA interference therapyPCSK 9 RNA interference therapy