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. 2008 Jan 4;82(1):113–124. doi: 10.1016/j.ajhg.2007.09.007

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Level of Transduction Necessary to Obtain a Complete Correction of the Phenotype

CEP mice were transplanted with HSCs transduced with the therapeutic ESpUROS-EFpGFP vector (same experiments as in Figure 4C), and the percentages of fluorocytes and GFP⁺ WBCs were determined for each mouse at 4 and 12 weeks. In the right part, skin photosensitivity was assayed 20 weeks after transplantation: (◆), mice with severe skin photosensitivity; (), mice with mild photosensitivity; and (◇) mice without any macroscopic or microscopic lesions.

Inline graphic — Level of Transduction Necessary to Obtain a Complete Correction of the Phenotype

CEP mice were transplanted with HSCs transduced with the therapeutic ESpUROS-EFpGFP vector (same experiments as in Figure 4C), and the percentages of fluorocytes and GFP⁺ WBCs were determined for each mouse at 4 and 12 weeks. In the right part, skin photosensitivity was assayed 20 weeks after transplantation: (◆), mice with severe skin photosensitivity; (), mice with mild photosensitivity; and (◇) mice without any macroscopic or microscopic lesions.