Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
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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 ~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.
Date issued
2014-03Department
Massachusetts Institute of Technology. Institute for Medical Engineering & Science; Harvard University--MIT Division of Health Sciences and Technology; Massachusetts Institute of Technology. Department of Biology; Massachusetts Institute of Technology. Department of Chemical Engineering; Koch Institute for Integrative Cancer Research at MITJournal
Nature Biotechnology
Publisher
Nature Publishing Group
Citation
Yin, Hao, Wen Xue, Sidi Chen, Roman L Bogorad, Eric Benedetti, Markus Grompe, Victor Koteliansky, Phillip A Sharp, Tyler Jacks, and Daniel G Anderson. “Genome Editing with Cas9 in Adult Mice Corrects a Disease Mutation and Phenotype.” Nature Biotechnology 32, no. 6 (March 30, 2014): 551–553.
Version: Author's final manuscript
ISSN
1087-0156
1546-1696