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.
Gene editing shows promise in patients with Hunter syndrome
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