Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Author(s)Yin, Hao; Xue, Wen; Chen, Sidi; Benedetti, Eric; Grompe, Markus; Kotelianski, Victor E.; Bogorad, Roman; Sharp, Phillip A.; Jacks, Tyler E.; Anderson, Daniel Griffith; ... Show more Show less
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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.
DepartmentInstitute for Medical Engineering and Science; David H. Koch Institute for Integrative Cancer Research at MIT; Harvard University--MIT Division of Health Sciences and Technology; Massachusetts Institute of Technology. Department of Biology; Massachusetts Institute of Technology. Department of Chemical Engineering
Nature Publishing Group
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.
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