Comparing ESC and iPSC—based models for human genetic disorders

Tomer Halevy, Achia Urbach

Research output: Contribution to journalReview articlepeer-review

56 Scopus citations


Traditionally, human disorders were studied using animal models or somatic cells taken from patients. Such studies enabled the analysis of the molecular mechanisms of numerous disorders, and led to the discovery of new treatments. Yet, these systems are limited or even irrelevant in modeling multiple genetic diseases. The isolation of human embryonic stem cells (ESCs) from diseased blastocysts, the derivation of induced pluripotent stem cells (iPSCs) from patients’ somatic cells, and the new technologies for genome editing of pluripotent stem cells have opened a new window of opportunities in the field of disease modeling, and enabled studying diseases that couldn’t be modeled in the past. Importantly, despite the high similarity between ESCs and iPSCs, there are several fundamental differences between these cells, which have important implications regarding disease modeling. In this review we compare ESC-based models to iPSC-based models, and highlight the advantages and disadvantages of each system. We further suggest a roadmap for how to choose the optimal strategy to model each specific disorder.

Original languageEnglish
Pages (from-to)1146-1162
Number of pages17
JournalJournal of Clinical Medicine
Issue number4
StatePublished - 24 Oct 2014

Bibliographical note

Publisher Copyright:
© 2014 by the authors; licensee MDPI, Basel, Switzerland.


  • Disease modeling
  • Embryonic stem cells (ESCs)
  • Induced pluripotent stem cells (iPSCs)


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