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Cellular & Molecular Biology Letters

Open Access

Proprotein convertase subtilisin/kexin type 9: A new target molecule for gene therapy

Cellular & Molecular Biology LettersAn International Journal201217:6

Received: 25 May 2011

Accepted: 2 February 2012

Published: 7 February 2012


Proprotein convertase subtilisin/kexin type 9 (PCSK9) has emerged as a novel target for controlling plasma levels of low-density lipoprotein cholesterol (LDL-C) and decreasing the risk of cardiovascular diseases. At present it is clear that the major classes of commonly prescribed lipid-lowering medications increase serum PCSK9 levels and fail to protect a significant percentage of patients from cardiovascular events. Therefore development of new LDL-C lowering medications that either do not increase circulating PCSK9 levels or work through inhibition of PCSK9 expression and protease activity is a highly desirable approach to overcome hypercholesterolemia. Since there are several agents which are being evaluated in human preclinical and clinical trials, this review summarizes current therapeutic strategies targeting PCSK9, including specific antibodies, antisense oligonucleotides, small interfering RNAs (siRNAs) and other small-molecule inhibitors.

Key words

PCSK9LDL cholesterolLDL receptor degradationStatinsFibratesEzetimibeAni-PCSK9 antibodyAntisense oligonucleotidesRNAiHypercholesterolemia