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2)Snyder RO, Miao CH, Patijn GA, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet. 1997; 16: 270-6
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7)Mimuro J, Mizukami H, Hishikawa S, et al. Minimizing the inhibitory effect of neutralizing antibody for efficient gene expression in the liver with adeno-associated virus 8 vectors. Mol Ther. 2013; 21: 318-23
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9)Mimuro J, Mizukami H, Shima M, et al. The prevalence of neutralizing antibodies against adeno-associated virus capsids is reduced in young Japanese individuals. J Med Virol. 2014; 86: 1990-7
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11)Lisowski L, Dane AP, Chu K, et al. Selection and evaluation of clinically relevant AAV variants in a xenograft liver model. Nature. 2014; 506: 382-6
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12)Roth DA, Tawa NE, Jr, OʼBrien JM, et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med. 2001; 344: 1735-42
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14)Everett LA, Cleuren AC, Khoriaty RN, et al. Murine coagulation factor VIII is synthesized in endothelial cells. Blood. 2014; 123: 3697-705
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15)Lin Y, Chang L, Solovey A, et al. Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood. 2002; 99: 457-62
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16)Matsui H, Shibata M, Brown B, et al. Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells. 2007; 25: 2660-9
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17)Ozelo MC, Vidal B, Brown C, et al. Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response. Blood. 2014; 123: 4045-53
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18)Tatsumi K, Sugimoto M, Lillicrap D, et al. A novel cell-sheet technology that achieves durable factor VIII delivery in a mouse model of hemophilia A. PLoS One. 2013; 8: e83280
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19)Xu D, Alipio Z, Fink LM, et al. Phenotypic correction of murine hemophilia A using an iPS cell-based therapy. Proc Natl Acad Sci U S A. 2009; 106: 808-13
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20)Yakura Y, Ishihara C, Kurosaki H, et al. An induced pluripotent stem cell-mediated and integration-free factor VIII expression system. Biochem Biophys Res Commun. 2013; 431: 336-41
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21)Park CY, Kim J, Kweon J, et al. Targeted inversion and reversion of the blood coagulation factor 8 gene in human iPS cells using TALENs. Proc Natl Acad Sci U S A. 2014; 111: 9253-8
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22)Kashiwakura Y, Ohmori T, Mimuro J, et al. Production of functional coagulation factor VIII from iPSCs using a lentiviral vector. Haemophilia. 2014; 20: e40-4
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23)Kashiwakura Y, Ohmori T, Mimuro J, et al. Intra-articular injection of mesenchymal stem cells expressing coagulation factor ameliorates hemophilic arthropathy in factor VIII-deficient mice. J Thromb Haemost. 2012; 10: 1802-13
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24)Dhadwar SS, Kiernan J, Wen J, et al. Repeated oral administration of chitosan/DNA nanoparticles delivers functional FVIII with the absence of antibodies in hemophilia A mice. J Thromb Haemost. 2010; 8: 2743-50
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25)Matsui H, Fujimoto N, Sasakawa N, et al. Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A. PLoS One. 2014; 9: e104957
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26)Kashiwakura Y, Mimuro J, Onishi A, et al. Porcine model of hemophilia A. PLoS One. 2012; 7: e49450
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27)Peters RT, Toby G, Lu Q, et al. Biochemical and functional characterization of a recombinant monomeric factor VIII-Fc fusion protein. J Thromb Haemost. 2013; 11: 132-41
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28)Powell JS, Josephson NC, Quon D, et al. Safety and prolonged activity of recombinant factor VIII Fc fusion protein in hemophilia A patients. Blood. 2012; 119: 3031-7
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29)Mahlangu J, Powell JS, Ragni MV, et al. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014; 123: 317-25
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30)Coyle TE, Reding MT, Lin JC, et al. Phase I study of BAY 94-9027, a PEGylated B-domain-deleted recombinant factor VIII with an extended half-life, in subjects with hemophilia A. J Thromb Haemost. 2014; 12: 488-96
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31)Tiede A, Brand B, Fischer R, et al. Enhancing the pharmacokinetic properties of recombinant factor VIII: first-in-human trial of glycoPEGylated recombinant factor VIII in patients with hemophilia A. J Thromb Haemost. 2013; 11: 670-8
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32)Zollner S, Raquet E, Claar P, et al. Non-clinical pharmacokinetics and pharmacodynamics of rVIII-SingleChain, a novel recombinant single-chain factor VIII. Thromb Res. 2014; 134: 125-31
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33)Negrier C, Knobe K, Tiede A, et al. Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B. Blood. 2011; 118: 2695-701
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34)Santagostino E, Negrier C, Klamroth R, et al. Safety and pharmacokinetics of a novel recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in hemophilia B patients. Blood. 2012; 120: 2405-11
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35)Peters RT, Low SC, Kamphaus GD, et al. Prolonged activity of factor IX as a monomeric Fc fusion protein. Blood. 2010; 115: 2057-64
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36)Powell JS, Pasi KJ, Ragni MV, et al. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013; 369: 2313-23
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37)Golor G, Bensen-Kennedy D, Haffner S, et al. Safety and pharmacokinetics of a recombinant fusion protein linking coagulation factor VIIa with albumin in healthy volunteers. J Thromb Haemost. 2013; 11: 1977-85
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38)Karpf DM, Sorensen BB, Hermit MB, et al. Prolonged half-life of glycoPEGylated rFVIIa variants compared to native rFVIIa. Thromb Res. 2011; 128: 191-5
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39)Shirahata A, Fukutake K, Takamatsu J, et al. A Phase II clinical trial of a mixture of plasma-derived factor VIIa and factor X (MC710) in haemophilia patients with inhibitors: haemostatic efficacy, safety and pharmacokinetics/pharmacodynamics. Haemophilia. 2013; 19: 853-60
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40)Kitazawa T, Igawa T, Sampei Z, et al. A bispecific antibody to factors IXa and X restores factor VIII hemostatic activity in a hemophilia A model. Nat Med. 2012; 18: 1570-4
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41)Sampei Z, Igawa T, Soeda T, et al. Identification and multidimensional optimization of an asymmetric bispecific IgG antibody mimicking the function of factor VIII cofactor activity. PLoS One. 2013; 8: e57479
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42)Shima M, Hermans C, de Moerloose P. Novel products for haemostasis. Haemophilia. 2014; 20 Suppl 4: 29-35
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