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
系统性给药编码人FIX的自互补AAV载体（该载体假型化了血清型5和8的衣壳蛋白）后的长期安全性和有效性。
期刊:Molecular Therapy
影响因子:12
doi:10.1038/mt.2010.274
Nathwani, Amit C; Rosales, Cecilia; McIntosh, Jenny; Rastegarlari, Ghasem; Nathwani, Devhrut; Raj, Deepak; Nawathe, Sushmita; Waddington, Simon N; Bronson, Roderick; Jackson, Scott; Donahue, Robert E; High, Katherine A; Mingozzi, Federico; Ng, Catherine Y C; Zhou, Junfang; Spence, Yunyu; McCarville, M Beth; Valentine, Marc; Allay, James; Coleman, John; Sleep, Susan; Gray, John T; Nienhuis, Arthur W; Davidoff, Andrew M
