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Article

Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

Nature biotechnology - 1 Jun 2014

Yin Hao, Xue Wen, Chen Sidi, Bogorad Roman L, Benedetti Eric, Grompe Markus, Koteliansky Victor, Sharp Phillip A, Jacks Tyler, Anderson Daniel G

Abstract excerpt

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. Expansion of Fah-positive hepatocytes rescued the body weight loss phenotype. Our study indicates that...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 24681508
DOI: 10.1038/nbt.2884
PMCID: PMC4157757

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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