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Blood|February 22, 2013
Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variantJenny McIntosh, Peter J Lenting, Cecilia Rosales, et al.Science Translational Medicine|April 22, 2016
Lentiviral hematopoietic stem cell gene therapy for X-linked severe combined immunodeficiencySuk See De Ravin, Xiaolin Wu, Susan Moir, et al.The New England Journal of Medicine|November 20, 2014
Long-term safety and efficacy of factor IX gene therapy in hemophilia BAmit C Nathwani, Ulreke M Reiss, Edward G D Tuddenham, et al.The New England Journal of Medicine|December 14, 2011
Adenovirus-associated virus vector-mediated gene transfer in hemophilia BAmit C Nathwani, Edward G D Tuddenham, Savita Rangarajan, et al.Pageof 4