The authors explore the potential of the recently described CRISPR-Cas9 site-specific nuclease system to encourage repair of the endogenous gene by enhancing the levels of homologous recombination. Using induced Pluripotent Stem cells derived from a Chronic Granulomatous Disease patient containing a single intronic mutation in the CYBB gene, they show that “footprintless” gene editing is a viable option to correct disease mutations.
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The authors explore the potential of the recently described CRISPR-Cas9 site-specific nuclease system to encourage repair of the endogenous gene by enhancing the levels of homologous recombination. Using induced Pluripotent Stem cells derived from a Chronic Granulomatous Disease patient containing a single intronic mutation in the CYBB gene, they show that “footprintless” gene editing is a viable option to correct disease mutations.
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