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Cells 2017, 6(1), 5; doi:10.3390/cells6010005

May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells

School of Biological and Health Systems Engineering, Arizona State University, 501 E. Tyler Mall, ECG 334A, Tempe, AZ 85287, USA
Author to whom correspondence should be addressed.
Academic Editor: Giovanni Amabile
Received: 18 December 2016 / Revised: 20 January 2017 / Accepted: 30 January 2017 / Published: 6 February 2017
(This article belongs to the Special Issue Ten Years of iPSCs: Current Status and Future Perspectives)
View Full-Text   |   Download PDF [655 KB, uploaded 6 February 2017]   |  


In the decade since Yamanaka and colleagues described methods to reprogram somatic cells into a pluripotent state, human induced pluripotent stem cells (hiPSCs) have demonstrated tremendous promise in numerous disease modeling, drug discovery, and regenerative medicine applications. More recently, the development and refinement of advanced gene transduction and editing technologies have further accelerated the potential of hiPSCs. In this review, we discuss the various gene editing technologies that are being implemented with hiPSCs. Specifically, we describe the emergence of technologies including zinc-finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), and clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 that can be used to edit the genome at precise locations, and discuss the strengths and weaknesses of each of these technologies. In addition, we present the current applications of these technologies in elucidating the mechanisms of human development and disease, developing novel and effective therapeutic molecules, and engineering cell-based therapies. Finally, we discuss the emerging technological advances in targeted gene editing methods. View Full-Text
Keywords: human induced pluripotent stem cells (hiPSCs); genome editing; homology-directed repair; ZFN; TALEN; CRISPR/Cas9 human induced pluripotent stem cells (hiPSCs); genome editing; homology-directed repair; ZFN; TALEN; CRISPR/Cas9

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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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Brookhouser, N.; Raman, S.; Potts, C.; Brafman, D.A. May I Cut in? Gene Editing Approaches in Human Induced Pluripotent Stem Cells. Cells 2017, 6, 5.

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