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Autor:
Michael Kaleko, Sheila Connelly, David Lillicrap, Cherie Cameron, Shawn Tinlin, Pamela S. Shirley, Angela M. Gallo-Penn, J. Andrews, Colleen Notley, Sandy Webster, Christine Hough
Publikováno v:
Blood. 97:107-113
Canine hemophilia A closely mimics the human disease and has been used previously in the development of factor VIII (FVIII) protein replacement products. FVIII-deficient dogs were studied to evaluate an in vivo gene therapy approach using an E1/E2a/E