Fanconi anemia gene editing by the CRISPR/Cas9 system.

  title={Fanconi anemia gene editing by the CRISPR/Cas9 system.},
  author={Mark J. Osborn and Richard Gabriel and Beau R Webber and Anthony P. DeFeo and Amber N. McElroy and Jordan Jarjour and Colby Starker and John E Wagner and J. Keith Joung and Daniel F. Voytas and Christof von Kalle and Manfred Schmidt and Bruce R Blazar and Jakub Tolar},
  journal={Human gene therapy},
  volume={26 2},
Genome engineering with designer nucleases is a rapidly progressing field, and the ability to correct human gene mutations in situ is highly desirable. We employed fibroblasts derived from a patient with Fanconi anemia as a model to test the ability of the clustered regularly interspaced short palindromic repeats/Cas9 nuclease system to mediate gene correction. We show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, because of its ability to preferentially… CONTINUE READING
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