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

Hao Yin, Wen Xue, Sidi Chen, Roman L. Bogorad, Eric Benedetti, Markus Grompe, Victor Koteliansky, Phillip A. Sharp, Tyler Jacks, Daniel G. Anderson

Research output: Contribution to journalArticle

552 Scopus citations

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 ~1/41/250 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.

Original languageEnglish (US)
Pages (from-to)551-553
Number of pages3
JournalNature biotechnology
Volume32
Issue number6
DOIs
StatePublished - Jun 2014

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ASJC Scopus subject areas

  • Biotechnology
  • Bioengineering
  • Applied Microbiology and Biotechnology
  • Molecular Medicine
  • Biomedical Engineering

Cite this

Yin, H., Xue, W., Chen, S., Bogorad, R. L., Benedetti, E., Grompe, M., Koteliansky, V., Sharp, P. A., Jacks, T., & Anderson, D. G. (2014). Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype. Nature biotechnology, 32(6), 551-553. https://doi.org/10.1038/nbt.2884