Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype

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....

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Main Authors: Yin, Hao (Contributor), Xue, Wen (Contributor), Chen, Sidi (Contributor), Benedetti, Eric (Author), Grompe, Markus (Author), Kotelianski, Victor E. (Author), Bogorad, Roman (Contributor), Sharp, Phillip A. (Contributor), Anderson, Daniel Griffith (Contributor), Jacks, Tyler E (Author)
Other Authors: Massachusetts Institute of Technology. Institute for Medical Engineering & Science (Contributor), Harvard University- (Contributor), Massachusetts Institute of Technology. Department of Biology (Contributor), Massachusetts Institute of Technology. Department of Chemical Engineering (Contributor), Koch Institute for Integrative Cancer Research at MIT (Contributor), Jacks, Tyler E. (Contributor)
Format: Article
Language:English
Published: Nature Publishing Group, 2015-06-05T17:19:53Z.
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