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Gene editing shows promise in patients with Hunter syndrome

Two patients with Hunter syndrome had significantly reduced biomarkers of the disease four months after receiving a medium dose of a corrective gene and zinc finger nucleases, while biomarkers were not reduced in two patients who received a low dose of the corrective gene and gene-editing compound. The results were presented at conference by study leader Joseph Muenzer of the University of North Carolina at Chapel Hill, who called them encouraging.

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