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

Volume: 32, Issue: 6, Pages: 551 - 553
Published: Jun 1, 2014
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...
Paper Details
Title
Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
Published Date
Jun 1, 2014
Volume
32
Issue
6
Pages
551 - 553
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