4.8 Review

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

Journal

NATURE MEDICINE
Volume 17, Issue 12, Pages 1570-1576

Publisher

NATURE PORTFOLIO
DOI: 10.1038/nm.2504

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Funding

  1. Ramon y Cajal
  2. Helmsley Charitable Trust
  3. Sanofi-Aventis
  4. G. Harold and Leila Y. Mathers Charitable Foundation
  5. Fundacion Cellex

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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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