The scientists 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. They show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, due to its ability to preferentially mediate homology-directed repair, resulted in a higher frequency of corrected clonal isolates.
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The scientists 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. They show that the Cas9 nuclease and nickase each resulted in gene correction, but the nickase, due to its ability to preferentially mediate homology-directed repair, resulted in a higher frequency of corrected clonal isolates.
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