Induced pluripotent stem cells as a model for accelerated patient- and disease-specific drug discovery.
Induced pluripotent stem cells as a model for accelerated patient- and disease-specific drug discovery.
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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。
DOI：
10.2174/092986710790514480
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发表时间：
2010
影响因子：
4.1
通讯作者：
Sachinidis A
中科院分区：
文献类型：
--
作者：
Gunaseeli I;Doss MX;Antzelevitch C;Hescheler J;Sachinidis A
人类诱导多能干（iPS）细胞在治疗多种退行性疾病方面具有巨大前景，例如缺血性心力衰竭、帕金森病、阿尔茨海默病、糖尿病、镰状细胞性贫血和亨廷顿病。它们还有可能通过 3 种方式加速药物发现。第一个涉及对患者血细胞或活组织检查中的细胞进行有效重编程的化学成分的描述，从而消除了细胞传递重编程外源转基因的需要，从而将患有退行性疾病的患者的希望变成现实。基于 iPS 细胞的细胞替代疗法对多种退行性疾病的临床适用性将使全世界患者受益。第二个是利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方式发现新药的潜力。第三是它们对药物毒理学测试和环境因素的适用性。本综述重点关注 iPS 细胞的这些潜在应用，特别强调 iPS 细胞研究的最新进展，有助于加速药物发现。
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.
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