Mark A. Kay

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

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

Medicine, Hematology, Molecular Medicine +13

Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways

Dirk Grimm, Konrad L. Streetz, Catherine L. Jopling et al. · Nature · 2006 · 1.7K citations

Life Sciences, Genetics, Molecular Biology +8

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 · 1.3K citations · Full text

Medicine, Genetics, Molecular Genetics +17

RNA interference in adult mice

Anton P. McCaffrey, Leonard Meuse, Thu-Thao T. Pham et al. · Nature · 2002 · 1.1K citations

Life Sciences, Biology, Molecular Biology +4