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Comparing ESC and iPSC—Based Models for Human Genetic Disorders

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 multipl...

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Detalles Bibliográficos
Autores principales: Halevy, Tomer, Urbach, Achia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2014
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4470175/
https://www.ncbi.nlm.nih.gov/pubmed/26237596
http://dx.doi.org/10.3390/jcm3041146
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author Halevy, Tomer
Urbach, Achia
author_facet Halevy, Tomer
Urbach, Achia
author_sort Halevy, Tomer
collection PubMed
description 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.
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spelling pubmed-44701752015-07-28 Comparing ESC and iPSC—Based Models for Human Genetic Disorders Halevy, Tomer Urbach, Achia J Clin Med Review 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. MDPI 2014-10-24 /pmc/articles/PMC4470175/ /pubmed/26237596 http://dx.doi.org/10.3390/jcm3041146 Text en © 2014 by the authors; licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Halevy, Tomer
Urbach, Achia
Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title_full Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title_fullStr Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title_full_unstemmed Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title_short Comparing ESC and iPSC—Based Models for Human Genetic Disorders
title_sort comparing esc and ipsc—based models for human genetic disorders
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4470175/
https://www.ncbi.nlm.nih.gov/pubmed/26237596
http://dx.doi.org/10.3390/jcm3041146
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