Abstract
Human pluripotent stem cells comprise induced pluripotent and embryonic stem cells, which have tremendous potential for biological and therapeutic applications. The development of efficient technologies for the targeted genome alteration of stem cells in disease models is a prerequisite for utilizing stem cells to their full potential. Genome editing of stem cells is possible with the help of synthetic nucleases that facilitate site-specific modification of a gene of interest. Recent advances in genome editing techniques have improved the efficiency and speed of the development of stem cells for human disease models. Zinc finger nucleases, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated system are powerful tools for editing DNA at specific loci. Here, we discuss recent technological advances in genome editing with site-specific nucleases in human stem cells.
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Acknowledgements
We would like to thank all of Suri’s laboratory members for their helpful discussions. Our work is supported by the National Research Foundation of Korea (2015R1C1A1A01054482 and 2013R1A1A1075992).
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Chandrasekaran, A.P., Song, M. & Ramakrishna, S. Genome editing: a robust technology for human stem cells. Cell. Mol. Life Sci. 74, 3335–3346 (2017). https://doi.org/10.1007/s00018-017-2522-0
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DOI: https://doi.org/10.1007/s00018-017-2522-0