Human stem cells and drug screening: opportunities and challenges
Human stem cells and drug screening: opportunities and challenges
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DOI：
10.1038/nrd3000
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发表时间：
2010-05-01
影响因子：
120.1
通讯作者：
Svendsen, Clive N.
中科院分区：
文献类型：
--
作者：
Ebert, Allison D.;Svendsen, Clive N.
高通量筛选技术广泛应用于药物发现的早期阶段，以快速评估数千种化合物的性质。然而，它们通常依赖于在高度增殖的永生化或癌细胞系上测试化合物文库，这不一定提供化合物在正常人细胞或所研究的特定细胞类型中的作用的准确指示。干细胞技术的最新进展有可能允许生产几乎无限供应的正常人类细胞，这些细胞可以分化成任何特定的细胞类型。此外，使用诱导多能干细胞技术，它们也可以从具有特定疾病特征的患者中产生，从而实现更相关的建模和药物筛选。本文讨论了干细胞在药物筛选中的机遇和挑战，重点是诱导多能干细胞。
High-throughput screening technologies are widely used in the early stages of drug discovery to rapidly evaluate the properties of thousands of compounds. However, they generally rely on testing compound libraries on highly proliferative immortalized or cancerous cell lines, which do not necessarily provide an accurate indication of the effects of compounds in normal human cells or the specific cell type under study. Recent advances in stem cell technology have the potential to allow production of a virtually limitless supply of normal human cells that can be differentiated into any specific cell type. Moreover, using induced pluripotent stem cell technology, they can also be generated from patients with specific disease traits, enabling more relevant modelling and drug screens. This article discusses the opportunities and challenges for the use of stem cells in drug screening with a focus on induced pluripotent stem cells.