Margaret V. Ragni

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

WFH Guidelines for the Management of Hemophilia, 3rd edition

Alok Srivastava, Elena Santagostino, Alison Dougall et al. · Haemophilia · 2020 · 1.9K citations · Full text

Medicine, Hematology, Pharmacotherapy +17

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

Life Sciences, Genetics, Molecular Biology +14

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

Lindsey A. George, Spencer K. Sullivan, Adam Giermasz et al. · New England Journal of Medicine · 2017 · 759 citations · Full text

Medicine, Hematology, Molecular Medicine +15

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