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诱导多能干细胞作为加速患者和疾病特异性药物发现的模型。

Induced pluripotent stem cells as a model for accelerated patient- and disease-specific drug discovery.

机构信息

Center of Physiology and Pathophysiology, Institute of Neurophysiology, and Center of Molecular Medicine, University of Cologne (CMMC), Robert-Koch Str. 39, Cologne, Germany.

出版信息

Curr Med Chem. 2010;17(8):759-66. doi: 10.2174/092986710790514480.

Abstract

Human induced pluripotent stem (iPS) cells hold great promise for therapy of a number of degenerative diseases such as ischemic heart failure, Parkinson's disease, Alzheimer's disease, diabetes mellitus, sickle cell anemia and Huntington disease. They also have the potential to accelerate drug discovery in 3 ways. The first involves the delineation of chemical components for efficient reprogramming of patient's blood cells or cells from biopsies, obviating the need for cellular delivery of reprogramming exogenous transgenes, thereby converting hope into reality for patients suffering from degenerative diseases. Patients worldwide stand to benefit from the clinical applicability of iPS cell-based cell replacement therapy for a number of degenerative diseases. The second is the potential for discovering novel drugs in a high throughput manner using patient-specific iPS cell-derived somatic cells possessing the etiology of the specific disease. The third is their suitability for toxicological testing of drugs and environmental factors. This review focuses on these potential applications of iPS cells with special emphasis on recent updates of iPS cell research contributing to the accelerated drug discovery.

摘要

人类诱导多能干细胞(iPS 细胞)在治疗多种退行性疾病方面具有巨大的潜力,如缺血性心力衰竭、帕金森病、阿尔茨海默病、糖尿病、镰状细胞贫血和亨廷顿病。它们还具有通过以下 3 种方式加速药物发现的潜力。第一种方法涉及到化学成分的描绘,以便有效地对患者的血细胞或活检细胞进行重新编程,从而避免了外源性转基因的细胞传递,从而为患有退行性疾病的患者将希望变为现实。世界各地的患者都将受益于 iPS 细胞为基础的细胞替代疗法在多种退行性疾病中的临床适用性。第二种方法是使用具有特定疾病病因的患者特异性 iPS 细胞衍生的体细胞,以高通量的方式发现新的药物的潜力。第三种方法是它们适合于药物和环境因素的毒理学测试。这篇综述重点介绍了 iPS 细胞的这些潜在应用,特别强调了最近的 iPS 细胞研究更新,这些更新有助于加速药物发现。

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