Engineered nucleases have advanced the field of gene therapy with the promise of targeted genome modification as a treatment for human diseases. Here we discuss why engineered nucleases are an exciting research tool for gene editing and consider their applications to a range of biological questions.
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Acknowledgements
M.P. thanks the Burroughs-Wellcome Fund, the Stanford School of Medicine Laurie Kraus Lacob Faculty Scholar Fund, the Amon Carter Fund and the National Institutes of Health Nanomedicine Development Center for Nucleoprotein Machines for their support. M.A.M. thanks the Stanford University Dean's Post-Doctoral Fellow Award for its support. M.R. thanks the Northwest Genome Engineering Consortium funded by the National Institutes of Health for its support.
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McMahon, M., Rahdar, M. & Porteus, M. Gene editing: not just for translation anymore. Nat Methods 9, 28–31 (2012). https://doi.org/10.1038/nmeth.1811
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DOI: https://doi.org/10.1038/nmeth.1811
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