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心血管疾病中针对RNA的疗法:时机已至。

RNA-targeted therapeutics in cardiovascular disease: the time is now.

作者信息

Krychtiuk Konstantin A, Rader Daniel J, Granger Christopher B

机构信息

Duke Clinical Research Institute, Duke University School of Medicine, Durham, NC 27701, USA.

Division of Translational Medicine and Human Genetics, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA.

出版信息

Eur Heart J Cardiovasc Pharmacother. 2022 Dec 15;9(1):94-99. doi: 10.1093/ehjcvp/pvac052.

Abstract

Ribonucleic acid (RNA)-targeted therapeutics, including antisense oligonucleotide technologies as well as small interfering RNAs (siRNAs), represent a new class of medications that may overcome several of the disadvantages of small molecule drugs or monoclonal antibodies. Specifically, upstream targeting at the messenger RNA (mRNA) level renders any disease-related protein a potential target, even those pathways previously deemed 'undruggable'. Additional advantages include the comparably simple and cost-effective way of manufacturing and the long dosing intervals. A few agents are already approved and a wide array of cardiovascular drugs is in development, aimed at hypercholesterolaemia, hypertension, myocardial storage diseases, and the coagulation system. Here, we provide an update on the current status of RNA-targeted therapeutics in the cardiovascular arena.

摘要

靶向核糖核酸(RNA)的疗法,包括反义寡核苷酸技术以及小干扰RNA(siRNA),是一类新型药物,可能克服小分子药物或单克隆抗体的一些缺点。具体而言,在信使核糖核酸(mRNA)水平进行上游靶向可使任何与疾病相关的蛋白质成为潜在靶点,即使是那些以前被认为“不可成药”的途径。其他优点包括相对简单且经济高效的生产方式以及较长的给药间隔。一些药物已获批准,并且正在开发一系列用于治疗高胆固醇血症、高血压、心肌贮积病和凝血系统的心血管药物。在此,我们提供靶向RNA疗法在心血管领域的现状更新。

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