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Targeting PCSK9 for Therapeutic Gains

  • Genetics (AJ Marian, Section Editor)
  • Published:
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Abstract

Even though it is only a little over a decade from the discovery of proprotein convertase subtilisin/kexin type 9 (PCSK9) as a plasma protein that associates with both hypercholesterolemia and low cholesterol syndromes, a rich literature has developed describing its unique physiology and the impact of antagonism of this molecule on cholesterol metabolism for therapeutic purposes. Indeed, the PCSK9 story is unfolding rapidly, with many answers and more questions. This review summarizes the most recent data from phase II/III clinical trials of PCSK9 inhibition with the three leading antibodies, highlights the clinical significance of the ongoing studies, and suggests future areas of investigation based on recent basic science discoveries on the physiology of PCSK9.

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Acknowledgments

This study was supported by the National Institutes of Health (National Heart, Lung, and Blood Institute) through grant R01-HL106845 to Dr. Fazio.

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Conflict of Interest

Michael Shapiro is a consultant to Abbott, LipoScience and Synageva. Sergio Fazio is a consultant to, received honoraria from, and had travel/accommodations expenses covered or reimbursed from Sanofi, Genzyme, ISIS. Hagai Tavori declares no conflict of interest.

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This article does not contain any studies with human or animal subjects performed by any of the authors.

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Correspondence to Sergio Fazio.

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This article is part of the Topical Collection on Genetics

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Shapiro, M.D., Fazio, S. & Tavori, H. Targeting PCSK9 for Therapeutic Gains. Curr Atheroscler Rep 17, 19 (2015). https://doi.org/10.1007/s11883-015-0499-4

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