Corral P, Ruiz A J
Especialista en Medicina Interna, lipidólogo clínico, Facultad Medicina, Universidad FASTA, Departamento Farmacología, Mar del Plata, Buenos Aires, Argentina.
Departamento de Medicina Interna, Departamento de Epidemiología Clínica y Bioestadística, Facultad de Medicina, Pontificia Universidad Javeriana, Bogotá D.C., Colombia.
Hipertens Riesgo Vasc. 2017 Oct-Dec;34(4):176-183. doi: 10.1016/j.hipert.2017.06.001. Epub 2017 Jul 12.
The discovery of proprotein convertase subtilisin/kexin type 9 (PCSK9) in 2003 in families with familial hypercholesterolemia (HF) later generated the development of pharmacological strategies in order to inhibit this protein. Twelve years after this discovery, the first two biological compounds (monoclonal antibodies) were approved, which have been shown to substantially decrease LDL-C and other lipid subfractions. The objective of the present article is to review the history of the discovery of PCSK9, its physiology and pathophysiology and subsequent pharmacological development. The objectives and goals reached to date and the pending questions regarding the efficacy and safety of its clinical use are presented.
2003年，在患有家族性高胆固醇血症（HF）的家族中发现了前蛋白转化酶枯草杆菌蛋白酶/kexin 9型（PCSK9），这随后推动了旨在抑制该蛋白的药理学策略的发展。在这一发现后的十二年，首批两种生物化合物（单克隆抗体）获得批准，已证明它们能大幅降低低密度脂蛋白胆固醇（LDL-C）和其他脂质亚组分。本文的目的是回顾PCSK9的发现历程、其生理学和病理生理学以及随后的药理学发展。介绍了迄今为止已达成的目标以及关于其临床使用的疗效和安全性的悬而未决的问题。
