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诱导多能干细胞作为疾病建模和药物筛选平台。

Induced pluripotent stem cells as a disease modeling and drug screening platform.

机构信息

Division of Cardiology, Departments of Medicine, Stanford University, Stanford, CA 94305-5454, USA.

出版信息

J Cardiovasc Pharmacol. 2012 Oct;60(4):408-16. doi: 10.1097/FJC.0b013e318247f642.

DOI:10.1097/FJC.0b013e318247f642
PMID:22240913
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC3343213/
Abstract

Induced pluripotent stem cells (iPSCs) hold great hopes for therapeutic application in various diseases. Although ongoing research is dedicated to achieving clinical translation of iPSCs, further understanding of the mechanisms that underlie complex pathogenic conditions is required. Compared with other classical models for studying diseases, iPSCs provide considerable advantages. A newly emerging application of iPSCs is in vitro disease modeling, which can significantly improve the never-ending search for new pharmacological cures. Here, we will discuss current efforts to create iPSC-dependent patient-specific disease models. Furthermore, we will review the use of iPSCs for development and testing of new therapeutic agents and the implications for high-throughput drug screening.

摘要

诱导多能干细胞 (iPSCs) 在各种疾病的治疗应用中具有巨大的希望。尽管正在进行的研究致力于实现 iPSCs 的临床转化,但需要进一步了解复杂致病条件背后的机制。与其他用于研究疾病的经典模型相比,iPSCs 具有很大的优势。iPSCs 的一个新出现的应用是体外疾病建模,它可以显著改善对新的药理学治疗方法的无休止的探索。在这里,我们将讨论目前创建基于 iPSC 的患者特异性疾病模型的努力。此外,我们还将回顾 iPSCs 在新型治疗药物的开发和测试中的应用,以及其对高通量药物筛选的影响。

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本文引用的文献

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Defining the nature of human pluripotent stem cell progeny.定义人类多能干细胞后代的性质。
Cell Res. 2012 Jan;22(1):178-93. doi: 10.1038/cr.2011.133. Epub 2011 Aug 16.
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Efficient generation of transgene-free human induced pluripotent stem cells (iPSCs) by temperature-sensitive Sendai virus vectors.温度敏感型仙台病毒载体高效生成无转基因的人类诱导多能干细胞(iPSCs)。
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Development of patient-specific neurons in schizophrenia using induced pluripotent stem cells.利用诱导多能干细胞生成精神分裂症患者特异性神经元
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Modeling neuronal defects associated with a lysosomal disorder using patient-derived induced pluripotent stem cells.使用患者来源的诱导多能干细胞对与溶酶体相关的神经元缺陷进行建模。
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