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Int. J. Mol. Sci. 2015, 16(12), 28614-28634;

Induced Pluripotency and Gene Editing in Disease Modelling: Perspectives and Challenges

Epigenetics and Cell Fates Laboratory, A*STAR Institute of Molecular and Cell Biology, 61 Biopolis Drive Proteos, Singapore 138673, Singapore
Department of Biological Sciences, National University of Singapore, Singapore 117543
NUS Centre for BioImaging Sciences, National University of Singapore, Singapore 117543
These authors contributed equally to this work.
Author to whom correspondence should be addressed.
Academic Editors: Wenbin Deng and Mark L. Richter
Received: 15 October 2015 / Revised: 13 November 2015 / Accepted: 24 November 2015 / Published: 2 December 2015
(This article belongs to the Section Biochemistry)
Full-Text   |   PDF [1373 KB, uploaded 2 December 2015]   |  


Embryonic stem cells (ESCs) are chiefly characterized by their ability to self-renew and to differentiate into any cell type derived from the three main germ layers. It was demonstrated that somatic cells could be reprogrammed to form induced pluripotent stem cells (iPSCs) via various strategies. Gene editing is a technique that can be used to make targeted changes in the genome, and the efficiency of this process has been significantly enhanced by recent advancements. The use of engineered endonucleases, such as homing endonucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and Cas9 of the CRISPR system, has significantly enhanced the efficiency of gene editing. The combination of somatic cell reprogramming with gene editing enables us to model human diseases in vitro, in a manner considered superior to animal disease models. In this review, we discuss the various strategies of reprogramming and gene targeting with an emphasis on the current advancements and challenges of using these techniques to model human diseases. View Full-Text
Keywords: iPSCs; CRISPR; gene editing; disease modeling iPSCs; CRISPR; gene editing; disease modeling

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Seah, Y.F.S.; EL Farran, C.A.; Warrier, T.; Xu, J.; Loh, Y.-H. Induced Pluripotency and Gene Editing in Disease Modelling: Perspectives and Challenges. Int. J. Mol. Sci. 2015, 16, 28614-28634.

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