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2) Hacein-Bey-Abina S, Garrigue A, Wang GP, el at. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest. 2008; 118: 3132-42
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3) Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003; 302: 415-9
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4) Frank KM, Hogarth DK, Miller JL, et al. Investigation of the cause of death in a gene-therapy trial. N Engl J Med. 2009; 361: 161-9
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5) 三室 淳. 血友病の遺伝子治療. In: 高久史麿, 他編. Annual Review血液 2002. 東京: 中外医学社; 2002. p.207-14
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6) 三室 淳. 血友病の遺伝子治療—最新の進歩. In: 高久史麿, 他編. Annual Review 血液. 2005. 東京: 中外医学社; 2005. p.295-302
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7) Arruda VR, Favaro P, Finn JD. Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther. 2009; 17: 1492-503
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8) Miao HZ, Sirachainan N, Palmer L, et al. Bioengineering of coagulation factor VIII for improved secretion. Blood. 2004; 103: 3412-9
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9) Dooriss KL, Denning G, Gangadharan B, et al. Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of Hemophilia A. Hum Gene Ther. 2009; 20: 465-78
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10) Margaritis P, Roy E, Aljamali MN, et al. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood. 2009; 113: 3682-9
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11) Ishiwata A, Mimuro J, Kashiwakura Y, et al. Phenotype correction of hemophilia A mice with adeno-associated virus vectors carrying the B domain-deleted canine factor VIII gene. Thromb Res. 2006; 118: 627-35
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12) Ishiwata A, Mimuro J, Mizukami H, et al. Liver-restricted expression of the canine factor VIII gene facilitates prevention of inhibitor formation in factor VIII-deficient mice. J Gene Med. 2009; 11: 1020-9
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13) Bank A, Dorazio R, Leboulch P. A phase I/II clinical trial of beta-globin gene therapy for beta-thalassemia. Ann NY Acad Sci. 2005; 1054: 308-16
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14) Kaiser J. Gene therapy. Beta-thalassemia treatment succeeds, with a caveat. Science. 2009; 326: 1468-9
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15) Kikuchi J, Mimuro J, Ogata K, et al. Sustained transgene expression by human cord blood derived CD34+ cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice. J Gene Med. 2004; 6: 1049-60
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16) Ohmori T, Mimuro J, Takano K, et al. Efficient expression of a transgene in platelets using simian immunodeficiency virus-based vector harboring glycoprotein Ibα promoter: in vivo model for platelet-targeting gene therapy. FASEB J. 2006; 20: 1522-4
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17) Ohmori T, Ishiwata A, Kashiwakura Y, et al. Phenotypic correction of hemophilia A by ectopic expression of activated factor VII in platelets. Mol Ther. 2008; 16: 1359-65
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18) Ogata K, Mimuro J, Kikuchi J, et al. Expression of human coagulation factor VIII in adipocytes transduced with the simian immunodeficiency virus agmTYO1-based vector for hemophilia A gene therapy. Gene Ther. 2004; 11: 253-9
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19) Viiala NO, Larsen SR, Rasko JE. Gene therapy for hemophilia: clinical trials and technical tribulations. Semin Thromb Hemost. 2009; 35: 81-92
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20) Kren BT, Unger GM, Sjeklocha L, et al. Nanocapsule-delivered sleeping beauty mediates therapeutic factor VIII expression in liver sinusoidal endothelial cells of hemophilia A mice. J Clin Invest. 2009; 119: 2086-99
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21) Ishiwata A, Mimuro J, Mizukami H, et al. Mutant Macaque factor IX T262A: A Tool for Hemophilia B Gene Therapy Studies in Macaques. Thromb Res. 2010; 125: 533-7
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22) Kay MA, Manno CS, Ragni MV, et al. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet. 2000; 24: 257-61
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23) Wang Z, Ma HI, Li J, et al. Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo. Gene Ther. 2003; 10: 2105-11
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24) Nathwani AC, Gray JT, Ng CY, et al. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood. 2006; 107: 2653-61
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25) Nathwani AC, Gray JT, McIntosh J, et al. Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates. Blood. 2007; 109: 1414-21
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26) Arruda VR, Stedman HH, Haurigot V, et al. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood. 2010; 115: 4678-88
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27) Calcedo R, Vandenberghe LH, Gao G, et al. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis. 2009; 199: 381-90
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