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用于心血管疾病的 microRNA 治疗学:机遇与障碍。

MicroRNA therapeutics for cardiovascular disease: opportunities and obstacles.

机构信息

miRagen Therapeutics, 6200 Lookout Rd, Boulder, Colorado 80301, USA.

出版信息

Nat Rev Drug Discov. 2012 Nov;11(11):860-72. doi: 10.1038/nrd3864. Epub 2012 Oct 19.

Abstract

In recent years, prominent roles for microRNAs (miRNAs) have been uncovered in several cardiovascular disorders. The ability to therapeutically manipulate miRNA expression and function through systemic or local delivery of miRNA inhibitors, referred to as antimiRs, has triggered enthusiasm for miRNAs as novel therapeutic targets. Here, we focus on the pharmacokinetic and pharmacodynamic properties of current antimiR designs and their relevance to cardiovascular indications, and evaluate the opportunities and obstacles associated with this new therapeutic modality.

摘要

近年来,microRNAs(miRNAs)在几种心血管疾病中的重要作用已被揭示。通过全身或局部递送 miRNA 抑制剂(称为 antimiRs)来治疗性地操纵 miRNA 表达和功能的能力激发了人们对 miRNA 作为新型治疗靶点的热情。在这里,我们重点关注当前 antimiR 设计的药代动力学和药效学特性及其与心血管适应症的相关性,并评估这种新治疗模式的相关机会和障碍。

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