Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Nature biotechnology, 2014•nature.com
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 …
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 …
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.
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