“Genome Editing With Cas9 in Adult Mice Corrects a Disease Mutation and Phenotype”, 2014 (; backlinks; similar):
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.