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利用多能干细胞技术进行药物发现、筛选、安全性和毒理学评估。

Exploiting pluripotent stem cell technology for drug discovery, screening, safety, and toxicology assessments.

机构信息

Department of Cell Biology, Neurobiology and Anatomy, Medical College of Wisconsin, 8701 Watertown Plank Rd, Milwaukee, WI 53226, USA.

Department of Cell Biology, Neurobiology and Anatomy, Medical College of Wisconsin, 8701 Watertown Plank Rd, Milwaukee, WI 53226, USA.

出版信息

Adv Drug Deliv Rev. 2014 Apr;69-70:170-8. doi: 10.1016/j.addr.2013.11.012. Epub 2013 Dec 2.

Abstract

In order for the pharmaceutical industry to maintain a constant flow of novel drugs and therapeutics into the clinic, compounds must be thoroughly validated for safety and efficacy in multiple biological and biochemical systems. Pluripotent stem cells, because of their ability to develop into any cell type in the body and recapitulate human disease, may be an important cellular system to add to the drug development repertoire. This review will discuss some of the benefits of using pluripotent stem cells for drug discovery and safety studies as well as some of the recent applications of stem cells in drug screening studies. We will also address some of the hurdles that need to be overcome in order to make stem cell-based approaches an efficient and effective tool in the quest to produce clinically successful drug compounds.

摘要

为了使制药行业能够不断将新型药物和疗法推向临床,化合物必须在多个生物和生化系统中进行彻底的安全性和疗效验证。多能干细胞由于其能够发育成体内任何细胞类型并再现人类疾病的能力,可能是药物开发中增加的一个重要细胞系统。本文将讨论使用多能干细胞进行药物发现和安全性研究的一些好处,以及干细胞在药物筛选研究中的一些最新应用。我们还将讨论为了使基于干细胞的方法成为寻找具有临床成功的药物化合物的有效工具而需要克服的一些障碍。

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