| First Author | Yin H | Year | 2014 |
| Journal | Nat Biotechnol | Volume | 32 |
| Issue | 6 | Pages | 551-3 |
| PubMed ID | 24681508 | Mgi Jnum | J:207452 |
| Mgi Id | MGI:5558873 | Doi | 10.1038/nbt.2884 |
| Citation | Yin H, et al. (2014) Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nat Biotechnol 32(6):551-3 |
| abstractText | 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 approximately 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. |
