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Showing 1 to 2 of 2 for “"Targeted Gene Repair"”.

  1. Gene Targeting in a Novel Mouse Model and the Chicken DT40 Cell Line

    Gene targeting has the power to create precise changes at specific sites within the genome. In the context of gene therapy, this technology may be used to treat patients with monogenic diseases by fixing mutations in disease causing genes, followed by transplanting the corrected cells back into the …

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  2. Optimizing Zinc Finger Nucleases for Use in Mammalian Cells

    … being explored as a potential way of performing gene therapy. However, the spontaneous rate of homologous recombination in human cells is too low (10-6) to be therapeutically useful. The most powerful way of stimulating homologous recombination is by introducing a double strand break within the …

    utswmed Repository record for Optimizing Zinc Finger Nucleases for Use in Mammalian Cells (opens in a new tab)