Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia a following gene therapy with high-capacity adenoviral vectors

Marinee K.L. Chuah, Gudrun Schiedner, Lieven Thorrez, Brian Brown, Marion Johnston, Veerle Gillijns, Sabine Hertel, Nico Van Rooijen, David Lillicrap, Désiré Collen, Thierry VandenDriessche, Stefan Kochanek

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