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Pasi, K. John; Rangarajan, Savita; Mitchell, Nina; Lester, Will; Symington, Emily; Madan, Bella; Laffan, Michael; Russell, Chris B; Li, Mingjin; Pierce, Glenn F; Wong, Wing Y
The New England journal of medicine, 01/2020, Letnik: 382, Številka: 1Journal Article
In 15 participants with hemophilia A treated with a single infusion of AAV5 vector containing the factor VIII gene, 7 who received 6×10 13 vector genomes per kilogram had a median factor VIII level of 20 IU per deciliter, and 6 who received 4×10 13 vg per kilogram had a median level of 13 IU per deciliter. Factor VIII levels decreased in years 2 and 3 of follow-up, but the median annualized rate of bleeding events among these 13 participants remained zero.
