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

Nature Medicine volume 17, pages 1570–1576 (2011)Cite this article

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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.

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Figure 1: An overview of the issues to consider when modeling genetic diseases with 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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Authors and Affiliations

  1. Centre of Regenerative Medicine in Barcelona, Barcelona, Spain

    Gustavo Tiscornia, Erica Lorenzo Vivas & Juan Carlos Izpisúa Belmonte

  2. Salk Institute for Biological Studies, La Jolla, California, USA

    Juan Carlos Izpisúa Belmonte

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  1. Gustavo Tiscornia
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  2. Erica Lorenzo Vivas
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  3. Juan Carlos Izpisúa Belmonte
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Correspondence to Juan Carlos Izpisúa Belmonte.

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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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