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.
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Acknowledgements
The authors thank R. Vassena and M. Barragan Monasterio for their critical reading of the manuscript. Work in the laboratory of J.C.I.B. was funded by Ramon y Cajal, The Helmsley Charitable Trust, Sanofi-Aventis, The G. Harold and Leila Y. Mathers Charitable Foundation and Fundacion Cellex.
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Tiscornia, G., Vivas, E. & Belmonte, J. Diseases in a dish: modeling human genetic disorders using induced pluripotent cells. Nat Med 17, 1570–1576 (2011). https://doi.org/10.1038/nm.2504
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DOI: https://doi.org/10.1038/nm.2504
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