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Gene therapy used to replace skin of boy with genetic disorder

A case study in the journal Nature described a 7-year-old boy with junctional epidermolysis bullosa, a genetic disease causing extremely fragile skin prone to tears and blisters, who had 80% of his skin replaced with large skin grafts produced using experimental gene therapy on a patch of skin unaffected by the disease. The boy continues to have healthy skin and has been able to play soccer and live normally two years after treatment, researchers said.

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