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

Nat Med. 2011 Dec;17(12):1570-6. doi: 10.1038/nm.2504.


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.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Cell Differentiation
  • Disease Models, Animal*
  • Genetic Diseases, Inborn / genetics*
  • Genetic Diseases, Inborn / pathology*
  • Genome, Human
  • Humans
  • Induced Pluripotent Stem Cells / cytology*
  • Induced Pluripotent Stem Cells / metabolism
  • Phenotype