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In Utero Transfer of Adeno-Associated Viral Vectors Produces Long-Term Factor IX Levels in a Cynomolgus Macaque Model

Citra N. Z. Mattar,Irene Gil-Farina,Cecilia Rosales,Nuryanti Johana,Yvonne Yi Wan Tan,Jenny McIntosh,Christine Kaeppel,Simon N. Waddington,Arijit Biswas,Mahesh Choolani,Manfred Schmidt,Amit C. Nathwani,Jerry K. Y. Chan

Molecular Therapy（2017）

Cited 32|Views50
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Key words
intrauterine gene transfer,adeno-associated viral vector,vector integration,non-human primate,immune tolerance,long-term expression,non-responder
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