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针对心血管疾病的 miRNA 治疗药物的靶向递送:机遇与挑战。

Targeted delivery of miRNA therapeutics for cardiovascular diseases: opportunities and challenges.

机构信息

*Department of Physiology, VU University Medical Center, Van der Boechorststraat 7, 1081 BT, Amsterdam, The Netherlands.

†Experimental Cardiology Laboratory, Department of Cardiology, Division of Heart and Lungs, University Medical Center Utrecht, P.O. Box 85500, 3508 GA, Utrecht, The Netherlands.

出版信息

Clin Sci (Lond). 2014 Sep;127(6):351-65. doi: 10.1042/CS20140005.

DOI:10.1042/CS20140005
PMID:24895056
Abstract

Dysregulation of miRNA expression has been associated with many cardiovascular diseases in animal models, as well as in patients. In the present review, we summarize recent findings on the role of miRNAs in cardiovascular diseases and discuss the opportunities, possibilities and challenges of using miRNAs as future therapeutic targets. Furthermore, we focus on the different approaches that can be used to deliver these newly developed miRNA therapeutics to their sites of action. Since siRNAs are structurally homologous with the miRNA therapeutics, important lessons learned from siRNA delivery strategies are discussed that might be applicable to targeted delivery of miRNA therapeutics, thereby reducing costs and potential side effects, and improving efficacy.

摘要

miRNA 表达失调与动物模型以及患者中的许多心血管疾病有关。在本综述中,我们总结了 miRNA 在心血管疾病中的作用的最新发现,并讨论了将 miRNA 用作未来治疗靶点的机会、可能性和挑战。此外,我们还重点介绍了可用于将这些新开发的 miRNA 治疗药物递送至作用部位的不同方法。由于 siRNA 与 miRNA 治疗药物在结构上具有同源性,因此讨论了从 siRNA 递药策略中获得的重要经验教训,这些经验教训可能适用于 miRNA 治疗药物的靶向递药,从而降低成本和潜在的副作用,并提高疗效。

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