alexa Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.
Genetics & Molecular Biology

Genetics & Molecular Biology

Cloning & Transgenesis

Author(s): Yin H, Xue W, Chen S, Bogorad RL, Benedetti E,

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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.
This article was published in Nat Biotechnol and referenced in Cloning & Transgenesis

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