Current approaches for efficient genetic editing in human pluripotent stem cells

Abstract

Human pluripotent stem cells have been much anticipated as a powerful system to study developmental events, model genetic disorders, and serve as a source of autologous cells for cell therapy in genetic disorders. Precise genetic manipulation is crucial to all these applications, and many recent advances have been made in site specific nuclease systems like zinc finger nucleases, TALENs, and CRISPR/Cas. In this review, we address the importance of site-specific genome modification and how this technology can be applied to manipulate human pluripotent stem cells.

Keywords

gene targeting / human pluripotent stem cells / TALEN / ZFN / CRISPR/Cas

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Acknowledgements

We would like to thank members of the Lee laboratory for valuable discussions on the manuscript. The Robertson Investigator Award from the New York Stem Cell Foundation (G.L.) and the Maryland Stem Cell Research Fund (G.L.) supported experiments in the Lee laboratory.

Competing financial interests

The authors declare no competing financial interests.

RIGHTS & PERMISSIONS

2014 Higher Education Press and Springer-Verlag Berlin Heidelberg