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J. Clin. Med. 2014, 3(4), 1146-1162; doi:10.3390/jcm3041146

Comparing ESC and iPSC—Based Models for Human Genetic Disorders

1
Stem Cell Unit, Department of Genetics, Institute of Life Sciences, The Hebrew University of Jerusalem, Jerusalem 91904, Israel
2
Mina and Everard Goodman Faculty of Life Sciences, Bar-Ilan University, Ramat Gan 5290002, Israel
*
Author to whom correspondence should be addressed.
Received: 10 February 2014 / Revised: 29 September 2014 / Accepted: 30 September 2014 / Published: 24 October 2014
(This article belongs to the Special Issue iPS Cells for Modelling and Treatment of Human Diseases)
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Abstract

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. View Full-Text
Keywords: embryonic stem cells (ESCs); induced pluripotent stem cells (iPSCs); disease modeling embryonic stem cells (ESCs); induced pluripotent stem cells (iPSCs); disease modeling
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This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. (CC BY 4.0).

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Halevy, T.; Urbach, A. Comparing ESC and iPSC—Based Models for Human Genetic Disorders. J. Clin. Med. 2014, 3, 1146-1162.

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