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....
| Main Authors: | , , , , , , , , , |
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| Other Authors: | |
| Format: | Article |
| Language: | en_US |
| Published: |
Nature Publishing Group
2015
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| 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 |