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97197 |
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|a dc
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|a Yin, Hao
|e author
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|a Massachusetts Institute of Technology. Institute for Medical Engineering & Science
|e contributor
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|a Harvard University-
|e contributor
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|a Massachusetts Institute of Technology. Department of Biology
|e contributor
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|a Massachusetts Institute of Technology. Department of Chemical Engineering
|e contributor
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|a Koch Institute for Integrative Cancer Research at MIT
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|a Yin, Hao
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|a Xue, Wen
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|a Chen, Sidi
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|a Bogorad, Roman
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|a Sharp, Phillip A.
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|a Jacks, Tyler E.
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|a Anderson, Daniel Griffith
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|a Xue, Wen
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|a Chen, Sidi
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|a Benedetti, Eric
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|a Grompe, Markus
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|a Kotelianski, Victor E.
|e author
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|a Bogorad, Roman
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|a Sharp, Phillip A.
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|a Anderson, Daniel Griffith
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|a Jacks, Tyler E
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|a Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
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|b Nature Publishing Group,
|c 2015-06-05T17:19:53Z.
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|z Get fulltext
|u http://hdl.handle.net/1721.1/97197
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|a We demonstrate CRISPR-Cas9-mediated correction of a Fah mutation in hepatocytes in a mouse model of the human disease hereditary tyrosinemia. Delivery of components of the CRISPR-Cas9 system by hydrodynamic injection resulted in initial expression of the wild-type Fah protein in ~1/250 liver cells. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that CRISPR-Cas9-mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases.
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|a National Cancer Institute (U.S.) (Grant 2-PO1-CA42063)
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|a National Cancer Institute (U.S.) (Core Grant P30-CA14051)
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|a National Institutes of Health (U.S.) (Grant R01-CA133404)
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|a David H. Koch Institute for Integrative Cancer Research at MIT (Marie D. and Pierre Casimir-Lambert Fund)
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|a National Institutes of Health (U.S.) (Centers for Cancer Nanotechnology Excellence 5-U54-CA151884-04)
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|a MIT-Harvard Center of Cancer Nanotechnology Excellence
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|a National Institutes of Health (U.S.) (1K99CA169512)
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|a en_US
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|a Article
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|t Nature Biotechnology
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