Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.

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Published in Mol Ther on January 18, 2011

Authors

Amit C Nathwani1, Cecilia Rosales, Jenny McIntosh, Ghasem Rastegarlari, Devhrut Nathwani, Deepak Raj, Sushmita Nawathe, Simon N Waddington, Roderick Bronson, Scott Jackson, Robert E Donahue, Katherine A High, Federico Mingozzi, Catherine Y C Ng, Junfang Zhou, Yunyu Spence, M Beth McCarville, Marc Valentine, James Allay, John Coleman, Susan Sleep, John T Gray, Arthur W Nienhuis, Andrew M Davidoff

Author Affiliations

1: Department of Hematology, University College London Cancer Institute, London, UK. a.nathwani@ucl.ac.uk

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