Margareth C. Ozelo

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

Catherine S. Manno, Glenn F. Pierce, Valder R. Arruda et al. · Nature Medicine · 2006 · 2.1K citations

Life Sciences, Hematology, Molecular Genetics +9

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Catherine S. Manno, Ah Keng Chew, S Hutchison et al. · Blood · 2003 · 747 citations · Full text

Medicine, Genetics, Hematology +11

ASH ISTH NHF WFH 2021 guidelines on the management of von Willebrand disease

Nathan T. Connell, Veronica H. Flood, Romina Brignardello‐Petersen et al. · Blood Advances · 2021 · 367 citations · Full text

Medicine, Hematology, Hematopathology +16

Phase 3 Study of Recombinant Factor IX Fc Fusion Protein in Hemophilia B

Jerry S. Powell, John Pasi, Margaret V. Ragni et al. · New England Journal of Medicine · 2013 · 352 citations · Full text

Medicine, Hematology, Immunology +14

Long-Term Follow-Up of the First in Human Intravascular Delivery of AAV for Gene Transfer: AAV2-hFIX16 for Severe Hemophilia B

Lindsey A. George, Margaret V. Ragni, John E.J. Rasko et al. · Molecular Therapy · 2020 · 209 citations · Full text

Medicine, Hematology, Immunology +13