Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Top Cited Papers
- 30 March 2014
- journal article
- research article
- Published by Springer Nature in Nature Biotechnology
- Vol. 32 (6) , 551-553
- https://doi.org/10.1038/nbt.2884
Abstract
CRISPR-Cas9-mediated genome editing corrects a hereditary tyrosinemia disease mutation in the liver of adult mice. 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.Keywords
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