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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Bibliographic Details
Main Authors: Yin, Hao, Xue, Wen, Chen, Sidi, Benedetti, Eric, Grompe, Markus, Kotelianski, Victor E., Bogorad, Roman, Sharp, Phillip A., Anderson, Daniel Griffith, Jacks, Tyler E
Other Authors: Massachusetts Institute of Technology. Institute for Medical Engineering & Science
Format: Article
Language:en_US
Published: Nature Publishing Group 2015
Online Access:http://hdl.handle.net/1721.1/97197
https://orcid.org/0000-0001-5785-8911
https://orcid.org/0000-0003-1465-1691
https://orcid.org/0000-0001-5629-4798
https://orcid.org/0000-0001-6898-3793

Internet

http://hdl.handle.net/1721.1/97197
https://orcid.org/0000-0001-5785-8911
https://orcid.org/0000-0003-1465-1691
https://orcid.org/0000-0001-5629-4798
https://orcid.org/0000-0001-6898-3793

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