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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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| Autori principali: | , , , , , , , , , |
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| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2014
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4157757/ https://ncbi.nlm.nih.gov/pubmed/24681508 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/nbt.2884 |
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