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Human stem cells and drug screening: opportunities and challenges

Abstract

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.

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Figure 1: Generation and lineage restriction of induced pluripotent stem cells derived from somatic cells can be adapted for high-throughput screening.
Figure 2: Drug treatment increases nuclear gems in induced pluripotent stem cells taken from a patient with spinal muscular atrophy.
Figure 3: Pluripotent stem cells could become an important factor in the drug discovery process.

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Correspondence to Clive N. Svendsen.

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DATABASES

OMIM

Alzheimer's disease

amyotrophic lateral sclerosis

Down's syndrome

familial dysautonomia

fragile X syndrome

Parkinson's disease

spinal muscular atrophy

United States Patent and Trademark Office 

20090011503

FURTHER INFORMATION

Allison D. Ebert's homepage

Wisconsin Alumni Research Foundation Technology — Stem Cells

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Ebert, A., Svendsen, C. Human stem cells and drug screening: opportunities and challenges. Nat Rev Drug Discov 9, 367–372 (2010). https://doi.org/10.1038/nrd3000

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