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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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Detalhes bibliográficos
Main Authors: Yin, Hao, Xue, Wen, Chen, Sidi, Bogorad, Roman L, Benedetti, Eric, Grompe, Markus, Koteliansky, Victor, Sharp, Phillip A, Jacks, Tyler, Anderson, Daniel G
Formato: Artigo
Idioma:Inglês
Publicado em: 2014
Assuntos:
Acesso em linha: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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