Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype
2014; Nature Portfolio; Volume: 32; Issue: 6 Linguagem: Inglês
10.1038/nbt.2884
ISSN1546-1696
AutoresHao Yin, Wen Xue, Sidi Chen, Roman L. Bogorad, Eric Benedetti, Markus Grompe, Victor Koteliansky, Phillip A. Sharp, Tyler Jacks, Daniel G. Anderson,
Tópico(s)Genetics, Aging, and Longevity in Model Organisms
ResumoCRISPR-Cas9-mediated genome editing corrects a hereditary tyrosinemia disease mutation in the liver of adult mice. 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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