Gringeri A, Mantovani LG, Scalone L, Mannucci PM, Grp CS. Cost of care and quality of life for patients with hemophilia complicated by inhibitors: the COCIS Study Group. Blood. 2003;102:2358–63.
Roth DA, Tawa Jr NE, O’Brien JM, Treco DA, Selden RF. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med. 2001;344:1735–42.
Powell JS, Ragni MV, White 2nd GC, et al. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood. 2003;102:2038–45.
White 2nd GC. Gene therapy in hemophilia: clinical trials update. Thromb Haemost. 2001;86:172–7.
Doering CB, Gangadharan B, Dukart HZ, Spencer HT. Hematopoietic stem cells encoding porcine factor VIII induce pro-coagulant activity in hemophilia A mice with pre-existing factor VIII immunity. Mol Ther. 2007;15:1093–9.
Ide LM, Gangadharan B, Chiang KY, Doering CB, Spencer HT. Hematopoietic stem-cell gene therapy of hemophilia A incorporating a porcine factor VIII transgene and nonmyeloablative conditioning regimens. Blood. 2007;110:2855–63.
McCauslin CS, Wine J, Cheng L, et al. In vivo retroviral gene transfer by direct intrafemoral injection results in correction of the SCID phenotype in Jak3 knock-out animals. Blood. 2003;102:843–8.
Worsham DN, Schuesler T, von Kalle C, Pan D. In vivo gene transfer into adult stem cells in unconditioned mice by in situ delivery of a lentiviral vector. Mol Ther. 2006;14:514–24.
Pan D, Gunther R, Duan WM, et al. Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow. Mol Ther. 2002;6:19–29.
Wang X, Shin SC, Chiang AF, Khan I, Pan D, Rawlings DJ, Miao CH. Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A. Mol Ther. 2015;23:617–26.
Habi O, Girard J, Bourdages V, Delisle M-C, and Carreau M. Correction of Fanconi anemia group c hematopoietic stem cells following intrafemoral gene transfer. Anemia, 2010;2010.
Baum C, Dullmann J, Li ZX, Fehse B, Meyer J, Williams DA, von Kalle C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood. 2003;101:2099–114.
Kaufman RJ, Powell JS. Molecular approaches for improved clotting factors for hemophilia. Hematol Am Soc Hematol Educ Program. 2013;2013:30–6.
Gu JM, Ramsey P, Evans V, et al. Evaluation of the activated partial thromboplastin time assay for clinical monitoring of PEGylated recombinant factor VIII (BAY 94–9027) for haemophilia A. Haemophilia. 2014;20:593–600.
Mei B, Pan C, Jiang H, et al. Rational design of a fully active, long-acting PEGylated factor VIII for hemophilia A treatment. Blood. 2010;116:270–9.
Nolan B, Mahlangu J, Perry D, et al. Long-term safety and efficacy of recombinant factor VIII Fc fusion protein (rFVIIIFc) in subjects with haemophilia A. Haemophilia. 2016;22:72–80.
Schulte S. Innovative coagulation factors: albumin fusion technology and recombinant single-chain factor VIII. Thromb Res. 2013;131 Suppl 2:S2–6.
Uchida N, Sambe T, Yoneyama K, Fukazawa N, Kawanishi T, Kobayashi S, Shima M. A first-in-human phase 1 study of ACE910, a novel factor VIII-mimetic bispecific antibody, in healthy subjects. Blood. 2016;127:1633–41.
Shima M, Hanabusa H, Taki M, et al. Factor VIII-Mimetic Function of Humanized Bispecific Antibody in Hemophilia A. N Engl J Med. 2016;374:2044–53.
Monahan PE. Gene therapy in an era of emerging treatment options for hemophilia B. J Thromb Haemost. 2015;13 Suppl 1:S151–60.
Nathwani AC, Reiss UM, Tuddenham EG, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994–2004.
Nathwani AC, Tuddenham EGD, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med. 2011;365:2357–65.
Callan MB, Haskins ME, Wang P, Zhou S, High KA, Arruda VR. Successful phenotype improvement following gene therapy for severe hemophilia a in privately owned dogs. PLoS One. 2016;11:e0151800.
McIntosh J, Lenting PJ, Rosales C, et al. Therapeutic levels of FVIII following a single peripheral vein administration of rAAV vector encoding a novel human factor VIII variant. Blood. 2013;121:3335–44.
Siner JI, Iacobelli NP, Sabatino DE, et al. Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype. Blood. 2013;121:4396–403.
Adams B. BioMarin says hemophilia A gene therapy data ‘encouranging’. http://www.fiercebiotech.com/biotech/biomarin-says-hemophilia-a-gene-therapy-data-encouraging, 2016: April 20, 2016.
Zhou S, Mody D, DeRavin SS, et al. A self-inactivating lentiviral vector for SCID-X1 gene therapy that does not activate LMO2 expression in human T cells. Blood. 2010;116:900–8.
Knight S, Bokhoven M, Collins M, Takeuchi Y. Effect of the internal promoter on insertional gene activation by lentiviral vectors with an intact HIV long terminal repeat. J Virol. 2010;84:4856–9.
Aiuti A, Biasco L, Scaramuzza S, et al. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. Science. 2013;341:1233151.
Biffi A, Montini E, Lorioli L, et al. Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy. Science. 2013;341:1233158.
Shi Q, Wilcox DA, Fahs SA, et al. Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A. J Thromb Haemost. 2007;5:352–61.
Wang CX, Sather BD, Wang X, et al. Rapamycin relieves lentiviral vector transduction resistance in human and mouse hematopoietic stem cells. Blood. 2014;124:913–23.
Kuether E, Schroeder J, Fahs S, et al. Lentivirus-mediated platelet gene therapy of murine hemophilia A with pre-existing ant-factor VIII immunity. J Thromb Haemost. 2012;10:1570–80.
Shi QZ, Wilcox DA, Fahs SA, et al. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J Clin Investig. 2006;116:1974–82.
Yarovoi HV, Kufrin D, Eslin DE, et al. Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment. Blood. 2003;102:4006–13.
Damon AL, Scudder LE, Gnatenko DV, Sitaraman V, Hearing P, Jesty J, Bahou WF. Altered bioavailability of platelet-derived factor VIII during thrombocytosis reverses phenotypic efficacy in haemophilic mice. Thromb Haemost. 2008;100:1111–22.
Ohmori T, Mimuro J, Takano K, et al. Efficient expression of a transgene in platelets using simian immunodeficiency virus-based vector harboring glycoprotein Ib alpha promoter: in vivo model for platelet-targeting gene therapy. FASEB J. 2006;20:1522–4.
Neyman M, Gewirtz J, Poncz M. Analysis of the spatial and temporal characteristics of platelet-delivered factor VHI-based clots. Blood. 2008;112:1101–8.
Ward NJ, Buckley SM, Waddington SN, et al. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood. 2011;117:798–807.
Scott DW, Pratt KP, Miao CH. Progress toward inducing immunologic tolerance to factor VIII. Blood. 2013;121:4449–56.
Matsui H, Shibata M, Brown B, et al. A murine model for induction of long-term immunologic tolerance to factor VIII does not require persistent detectable levels of plasma factor VIII and involves contributions from Foxp3(+) T regulatory cells. Blood. 2009;114:677–85.
Sack BK, Merchant S, Markusic DM, Nathwani AC, Davidoff AM, Byrne BJ, Herzog RW. Transient B cell depletion or improved transgene expression by codon optimization promote tolerance to factor VIII in gene therapy. PLoS One. 2012;7:e37671.
Shi Q, Fahs SA, Wilcox DA, et al. Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. Blood. 2008;112:2713–21.
Brown BD, Sitia G, Annoni A, et al. In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood. 2007;109:2797–805.
Agudo J, Ruzo A, Kitur K, Sachidanandam R, Blander JM, Brown BD. A TLR and non-TLR mediated innate response to lentiviruses restricts hepatocyte entry and can be ameliorated by pharmacological blockade. Mol Ther. 2012;20:2257–67.
Akira S, Uematsu S, Takeuchi O. Pathogen recognition and innate immunity. Cell. 2006;124:783–801.
Wang X, Lyle MJ, Fu R, Miao CH. Enhancing factor VIII expression in platelets of hemophilia A mice following intraosseous delivery of lentiviral vectors. New Orleans: American Society of Gene and Cell Therapy 18th Annual Meeting; 2015. p. 245.