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Nature Research, Nature Medicine, 12(17), p. 1570-1576, 2011

DOI: 10.1038/nm.2504

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Diseases in a dish: Modeling human genetic disorders using induced pluripotent cells

This paper is made freely available by the publisher.
This paper is made freely available by the publisher.

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Abstract

The derivation of induced pluripotent cells (iPSCs) from individuals suffering from genetic syndromes offers new opportunities for basic research into these diseases and the development of therapeutic compounds. iPSCs can self renew and can be differentiated to many cell types, offering a potentially unlimited source of material for study. In this review we discuss the conceptual and practical issues to consider when attempting to model genetic diseases using iPSCs.