Journal
NATURE BIOTECHNOLOGY
Volume 32, Issue 6, Pages 551-553Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/nbt.2884
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Funding
- National Cancer Institute [2-PO1-CA42063, P30-CA14051]
- National Institutes of Health (NIH) [R01-CA133404]
- Howard Hughes Investigator
- NIH Centers for Cancer Nanotechnology Excellence and the Harvard-MIT Center of Cancer Nanotechnology Excellence [5-U51-CA151884-04]
- Damon Runyon Fellow [DRG2117- 12]
- [1K99CA169512]
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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 similar to 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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