rAAV9 combined with renal vein injection is optimal for kidney-targeted gene delivery: conclusion of a comparative study

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

Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

James Bainbridge, Alexander J. Smith, Susie S. Barker et al. · New England Journal of Medicine · 2008 · 1.9K citations · Full text

Medicine, Genetics, Ophthalmology +15

Prevalence of Serum IgG and Neutralizing Factors Against Adeno-Associated Virus (AAV) Types 1, 2, 5, 6, 8, and 9 in the Healthy Population: Implications for Gene Therapy Using AAV Vectors

Sylvie Boutin, Virginie Monteilhet, P. Véron et al. · Human Gene Therapy · 2010 · 947 citations

Life Sciences, Immunology, Molecular Biology +15

Worldwide Epidemiology of Neutralizing Antibodies to Adeno‐Associated Viruses

Roberto Calcedo, Luk H. Vandenberghe, Guangping Gao et al. · The Journal of Infectious Diseases · 2009 · 792 citations · Full text

Life Sciences, Infectious Diseases, Molecular Biology +14