An Observational Study from Long-Term AAV Re-administration in Two Hemophilia Dogs

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

Inflammation, Aav-factor Ix, Host Response +11

Adenovirus-Associated Virus Vector–Mediated Gene Transfer in Hemophilia B

Amit C. Nathwani, Edward G. D. Tuddenham, Savita Rangarajan et al. · New England Journal of Medicine · 2011 · 1.8K citations · Full text

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

Guangping Gao, Mauricio R. Alvira, Lili Wang et al. · Proceedings of the National Academy of Sciences · 2002 · 1.5K citations · Full text

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

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 · 736 citations · Full text