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Regen Med. 2016 Dec;11(8):801-816. Epub 2016 Dec 1.

To CRISPR and beyond: the evolution of genome editing in stem cells.

Chen KY1,2,3, Knoepfler PS1,2,3.

Author information

Department of Cell Biology and Human Anatomy, University of California Davis School of Medicine, Davis, CA, USA.
Institute of Pediatric Regenerative Medicine, Shriners Hospital For Children Northern California, Sacramento, CA, USA.
Genome Center, University of California Davis, Davis, CA, USA.


The goal of editing the genomes of stem cells to generate model organisms and cell lines for genetic and biological studies has been pursued for decades. There is also exciting potential for future clinical impact in humans. While recent, rapid advances in targeted nuclease technologies have led to unprecedented accessibility and ease of gene editing, biology has benefited from past directed gene modification via homologous recombination, gene traps and other transgenic methodologies. Here we review the history of genome editing in stem cells (including via zinc finger nucleases, transcription activator-like effector nucleases and CRISPR-Cas9), discuss recent developments leading to the implementation of stem cell gene therapies in clinical trials and consider the prospects for future advances in this rapidly evolving field.


CRISPR-Cas9; TALEN; ZFN; gene therapy; genome editing; stem cells

[Indexed for MEDLINE]
Free PMC Article

Conflict of interest statement

Financial & competing interests disclosure This work was support by NIH grant 1R01GM116919-01 to PS Knoepfler and by NCI training fellowship T32CA108459 to KY Chen. The authors have no other relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript apart from those disclosed. No writing assistance was utilized in the production of this manuscript.

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