Efficacy and safety of long-term prophylaxis in severe hemophilia a dogs following liver gene therapy using AAV vectors

Denise E. Sabatino, Amy M. Lange, Ekaterina S. Altynova, Rita Sarkar, Shangzhen Zhou, Elizabeth P. Merricks, Helen G. Franck, Timothy C. Nichols, Valder R. Arruda, Haig H. Kazazian Jr