Preclinical assessment of an optimized AAV-FVIII vector in mice and non-human primates for the treatment of hemophilia A

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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Inflammation, Aav-factor Ix, Host Response +11

Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA

F Liu, Young Song, D Liu · Gene Therapy · 1999 · 1.7K citations

Xenotransplantation, Engineering, Molecular Biology +10

Long-Term Safety and Efficacy of Factor IX Gene Therapy in Hemophilia B

Amit Nathwani, Ulreke M. Reiss, Edward G. D. Tuddenham et al. · New England Journal of Medicine · 2014 · 1.2K citations · Full text

Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep-vein thrombosis

Ted Koster, Jan P. Vandenbroucke, F. R. Rosendaal et al. · The Lancet · 1995 · 1.1K citations

Thrombosis, Venous Thrombosis, Von Willebrand Factor +10

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector

Mark A. Kay, Catherine S. Manno, Margaret V. Ragni et al. · Nature Genetics · 2000 · 1K citations

Factor Ix, Immunogenetics, Autoimmune Disease +11