Long-term safety and efficacy of factor IX gene therapy in hemophilia B
凝血因子IX基因疗法治疗B型血友病的长期安全性和有效性
期刊:New England Journal of Medicine
影响因子:78.5
doi:10.1056/NEJMoa1407309
Nathwani, Amit C; Reiss, Ulreke M; Tuddenham, Edward G D; Rosales, Cecilia; Chowdary, Pratima; McIntosh, Jenny; Della Peruta, Marco; Lheriteau, Elsa; Patel, Nishal; Raj, Deepak; Riddell, Anne; Pie, Jun; Rangarajan, Savita; Bevan, David; Recht, Michael; Shen, Yu-Min; Halka, Kathleen G; Basner-Tschakarjan, Etiena; Mingozzi, Federico; High, Katherine A; Allay, James; Kay, Mark A; Ng, Catherine Y C; Zhou, Junfang; Cancio, Maria; Morton, Christopher L; Gray, John T; Srivastava, Deokumar; Nienhuis, Arthur W; Davidoff, Andrew M