Valder R. Arruda

Safety and Efficacy of Gene Transfer for Leber's Congenital Amaurosis

Albert M. Maguire, Francesca Simonelli, Eric A. Pierce et al. · New England Journal of Medicine · 2008 · 2.1K citations · Full text

Medicine, Genetics, Ophthalmology +17

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

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

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

Federico Mingozzi, Yilin Liu, Eric Dobrzynski et al. · Journal of Clinical Investigation · 2003 · 395 citations · Full text

Life Sciences, Genetics, Immunology +17