Here, the authors used TALEN technology to correct the genetic defect in Xeroderma pigmentosum (XP) cells. They showed that specifically-designed TALE-nuclease allowed to correct a 2 bp deletion in the XPC gene leading to patient's cells proficient for DNA repair and showing normal UV-sensitivity. This result could be a first step toward gene therapy in XP patients.
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Here, the authors used TALEN technology to correct the genetic defect in Xeroderma pigmentosum (XP) cells. They showed that specifically-designed TALE-nuclease allowed to correct a 2 bp deletion in the XPC gene leading to patient's cells proficient for DNA repair and showing normal UV-sensitivity. This result could be a first step toward gene therapy in XP patients.
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