Ru Zhang;Qiang Wang;Lin Zhang;Saijuan Chen ;

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Ru Zhang;Qiang Wang;Lin Zhang;Saijuan Chen ; Optimized human factor IX expression cassettes for hepatic-directed gene therapy of hemophilia B Ru Zhang;Qiang Wang;Lin Zhang;Saijuan Chen ; State Key Laboratory for Medical Genomics, Shanghai Institute of Hematology, Ruijin Hospital Affiliated to Shanghai Jiao Tong University School of Medicine, Shanghai 200025, China ; Fig.4 Comparison of hFIX coagulant activity and expression level mediated by transgene cassettes in HB mice after hydrodynamic injection. FIX-SJ: scAAV-LP1-hFIXco-SJ; FIX-SJ-M: scAAV-LP1-hFIXco-SJ-R338L; FIX-SIH: scAAV-LP1-hFIXco-SIH; and FIX-SIH-M: scAAV-LP1-hFIXco-SIH-R338L. A+ B Chromogenic assay to determine hFIX activity in individual mice in A is presented as mean±SD in B. The activity is calculated as a percentage of normal hFIX activity in the pooled plasma. The average hFIX activities mediated by FIX-SJ, FIX-SJ-M, FIX-SIH, and FIX-SIH-M were about 60%±24%, 406%±32%, 66%±16%, and 479%±37%, respectively. C+ D ELISA results of hFIX expression in individual mice in C are presented as mean±SD in D. The expression of codon-optimized h FIX with or without R338L mutation showed no significant difference. Frontiers of Medicine,2015,9(1),90-99. Doi:10.1007/s11684-015-0390-2