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Trends Biochem Sci. 2008 Sep;33(9):426-34. doi: 10.1016/j.tibs.2008.06.005. Epub 2008 Jul 30.

PCSK9 and LDL cholesterol: unravelling the target to design the bullet.

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INSERM, UMR915, L'Institut du Thorax, 1 Rue Gaston Veil, Nantes, France.


Gain-of-function mutations within proprotein convertase subtilisin kexin type 9 (PCSK9) are linked to familial autosomal dominant hypercholesterolaemia, a disease characterized by elevated plasma concentrations of cholesterol associated with low-density lipoproteins (LDLs). Conversely, PCSK9 loss-of-function mutations result in low levels of LDL cholesterol (LDLC) and protect against coronary heart disease. Although compelling evidence indicates that PCSK9 impairs the LDLR pathway, its role in cholesterol metabolism remains incompletely defined. In the past two years, several new biochemical findings, including the PCSK9 crystal structure and the identification of several transcriptional repressors, were reported. Moreover, new clinical and epidemiological data have revealed the correlation between plasma PCSK9 concentrations and LDLC levels.

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