Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.
basic_science · Level V
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- Record sourced from PubMed, PMID 24681508.
- Also identified by DOI 10.1038/nbt.2884 and PMC identifier 4157757.
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Abstract
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 CRISPR-Cas9-mediated genome editing is possible in adult animals and has potential for correction of human genetic diseases.
Medical subject headings
- CRISPR-Associated Proteins
- CRISPR-Cas Systems
- Gene Editing
- Genetic Therapy
- Hydrolases
- Tyrosinemias