Publication
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
Amit C Nathwani, 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 YC 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
Molecular Therapy, January 2011, Nature
DOI: 10.1038/mt.2010.274