PCSK9：从基础科学发现到临床试验。
PCSK9: From Basic Science Discoveries to Clinical Trials.
机构信息
From the Center for Preventive Cardiology, Knight Cardiovascular Institute, Oregon Health & Science University, Portland.
出版信息
Circ Res. 2018 May 11;122(10):1420-1438. doi: 10.1161/CIRCRESAHA.118.311227.
Abstract
Unknown 15 years ago, PCSK9 (proprotein convertase subtilisin/kexin type 9) is now common parlance among scientists and clinicians interested in prevention and treatment of atherosclerotic cardiovascular disease. What makes this story so special is not its recent discovery nor the fact that it uncovered previously unknown biology but rather that these important scientific insights have been translated into an effective medical therapy in record time. Indeed, the translation of this discovery to novel therapeutic serves as one of the best examples of how genetic insights can be leveraged into intelligent target drug discovery. The PCSK9 saga is unfolding quickly but is far from complete. Here, we review major scientific understandings as they relate to the role of PCSK9 in lipoprotein metabolism and atherosclerotic cardiovascular disease and the impact that therapies designed to inhibit its action are having in the clinical setting.
摘要
15 年前还不为人知的 PCSK9（前蛋白转化酶枯草溶菌素 9），现在已经成为了对动脉粥样硬化性心血管疾病的预防和治疗感兴趣的科学家和临床医生的常用语。这个故事之所以如此特别，并不是因为它是最近才被发现的，也不是因为它揭示了以前未知的生物学，而是因为这些重要的科学发现已经在创纪录的时间内转化为有效的医学治疗方法。事实上，这一发现转化为新型治疗方法，是如何利用遗传见解来进行智能靶向药物发现的最好例子之一。PCSK9 的故事正在迅速展开，但远未结束。在这里，我们回顾了与 PCSK9 在脂蛋白代谢和动脉粥样硬化性心血管疾病中的作用以及旨在抑制其作用的治疗方法在临床环境中的影响相关的主要科学认识。
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