SEARCH

SEARCH BY CITATION

References

  • 1
    Blaese RM, Culver KW, Miller AD, Carter CS, Fleisher T, Clerici M, Shearer G, Chang L, Chiang Y, Tolstoshev P, Greenblatt JJ, Rosenberg SA, Klein H, Berger M, Mullen CA, Ramsey WJ, Muul L, Morgan RA, Anderson WF. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995; 270: 47580.
  • 2
    Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A, Carlucci F, Eibl M, Aker M, Slavin S, Al-Mousa H, Al Ghonaium A, Ferster A, Duppenthaler A, Notarangelo L, et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med 2009; 360: 44758.
  • 3
    AMT submits its lead product Glybera® application for marketing authorisation to EMA. Press Release 2010. http://investors.amtpharma.com/phoenix.zhtml?c=212989&p=irol-mediaArticle&ID=1373195&highlight=. Accessed 13 June 2011.
  • 4
    Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I, Vidaud M, Abel U, Dal-Cortivo L, Caccavelli L, Mahlaoui N, Kiermer V, Mittelstaedt D, Bellesme C, Lahlou N, Lefrere F, Blanche S, Audit M, Payen E, Leboulch P, et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009; 326: 81823.
  • 5
    Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber’s congenital amaurosis. N Engl J Med 2008; 358: 22319.
  • 6
    Hauswirth WW, Aleman TS, Kaushal S, Cideciyan AV, Schwartz SB, Wang L, Conlon TJ, Boye SL, Flotte TR, Byrne BJ, Jacobson SG. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial. Hum Gene Ther 2008; 19: 97990.
  • 7
    Maguire AM, High KA, Auricchio A, Wright JF, Pierce EA, Testa F, Mingozzi F, Bennicelli JL, Ying GS, Rossi S, Fulton A, Marshall KA, Banfi S, Chung DC, Morgan JI, Hauck B, Zelenaia O, Zhu X, Raffini L, Coppieters F, et al. Age-dependent effects of RPE65 gene therapy for Leber’s congenital amaurosis: a phase 1 dose-escalation trial. Lancet 2009; 374: 1597605.
  • 8
    Hacein-Bey-Abina S, von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R, Forster A, Fraser P, Cohen JI, de Saint Basile G, Alexander I, Wintergerst U, Frebourg T, Aurias A, Stoppa-Lyonnet D, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 4159.
  • 9
    Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, et al. Successful transduction of liver in hemophilia by AAV-factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 3427.
  • 10
    Mingozzi F, High KA. Therapeutic in vivo gene transfer for genetic disease: progress and challenges. Nat Rev Genet 2011; 12: 34155.
  • 11
    Mendell JR, Campbell K, Rodino-Klapac L, Sahenk Z, Shilling C, Lewis S, Bowles D, Gray S, Li C, Galloway G, Malik V, Coley B, Clark KR, Li J, Xiao X, Samulski J, McPhee SW, Samulski RJ, Walker CM. Dystrophin immunity in Duchenne’s muscular dystrophy. N Engl J Med 2010; 363: 142937.
  • 12
    Franchini M, Favaloro EJ, Lippi G. Mild hemophilia A. J Thromb Haemost 2010; 8: 42132.
  • 13
    Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 1995; 10: 11921.
  • 14
    Lin HF, Maeda N, Smithies O, Straight DL, Stafford DW. A coagulation factor IX-deficient mouse model for human hemophilia B. Blood 1997; 90: 39626.
  • 15
    Wang L, Zoppe M, Hackeng TM, Griffin JH, Lee KF, Verma IM. A factor IX-deficient mouse model for hemophilia B gene therapy. Proc Natl Acad Sci U S A 1997; 94: 115636.
  • 16
    Yao SN, Farjo A, Roessler BJ, Davidson BL, Kurachi K. Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: evidence for prolonged stability and activity of the transgene in liver. Viral Immunol 1996; 9: 14153.
  • 17
    Evans JP, Brinkhous KM, Brayer GD, Reisner HM, High KA. Canine hemophilia B resulting from a point mutation with unusual consequences. Proc Natl Acad Sci U S A 1989; 86: 100959.
  • 18
    Lozier JN, Dutra A, Pak E, Zhou N, Zheng Z, Nichols TC, Bellinger DA, Read M, Morgan RA. The Chapel Hill hemophilia A dog colony exhibits a factor VIII gene inversion. Proc Natl Acad Sci U S A 2002; 99: 129916.
  • 19
    Mauser AE, Whitlark J, Whitney KM, Lothrop CD Jr. A deletion mutation causes hemophilia B in Lhasa Apso dogs. Blood 1996; 88: 34515.
  • 20
    Murphy SL, High KA. Gene therapy for haemophilia. Br J Haematol 2008; 140: 47987.
  • 21
    Hasbrouck NC, High KA. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther 2008; 15: 8705.
  • 22
    Powell JS, Ragni MV, White GC II, Lusher JM, Hillman-Wiseman C, Moon TE, Cole V, Ramanathan-Girish S, Roehl H, Sajjadi N, Jolly DJ, Hurst D. Phase 1 trial of FVIII gene transfer for severe hemophilia A using a retroviral construct administered by peripheral intravenous infusion. Blood 2003; 102: 203845.
  • 23
    VandenDriessche T, Vanslembrouck V, Goovaerts I, Zwinnen H, Vanderhaeghen ML, Collen D, Chuah MK. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc Natl Acad Sci U S A 1999; 96: 1037984.
  • 24
    Xu L, Gao C, Sands MS, Cai SR, Nichols TC, Bellinger DA, Raymer RA, McCorquodale S, Ponder KP. Neonatal or hepatocyte growth factor-potentiated adult gene therapy with a retroviral vector results in therapeutic levels of canine factor IX for hemophilia B. Blood 2003; 101: 392432.
  • 25
    Roth DA, Tawa NE Jr, 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: 173542.
  • 26
    Shi Q, Wilcox DA, Fahs SA, Weiler H, Wells CW, Cooley BC, Desai D, Morateck PA, Gorski J, Montgomery RR. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J Clin Invest 2006; 116: 197482.
  • 27
    Yarovoi H, Nurden AT, Montgomery RR, Nurden P, Poncz M. Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: lessons from platelet-expressed factor VIII. Blood 2005; 105: 46746.
  • 28
    Greene TK, Wang C, Hirsch JD, Zhai L, Gewirtz J, Thornton MA, Miao HZ, Pipe SW, Kaufman RJ, Camire RM, Arruda VR, Kowalska MA, Poncz M. In vivo efficacy of platelet-delivered, high specific activity factor VIII variants. Blood 2010; 116: 611422.
  • 29
    Brunetti-Pierri N, Ng P. Progress towards liver and lung-directed gene therapy with helper-dependent adenoviral vectors. Curr Gene Ther 2009; 9: 32940.
  • 30
    Brunetti-Pierri N, Stapleton GE, Law M, Breinholt J, Palmer DJ, Zuo Y, Grove NC, Finegold MJ, Rice K, Beaudet AL, Mullins CE, Ng P. Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther 2009; 17: 32733.
  • 31
    Cameron C, Notley C, Hoyle S, McGlynn L, Hough C, Kamisue S, Giles A, Lillicrap D. The canine factor VIII cDNA and 5′ flanking sequence. Thromb Haemost 1998; 79: 31722.
  • 32
    Kessler PD, Podsakoff GM, Chen X, McQuiston SA, Colosi PC, Matelis LA, Kurtzman GJ, Byrne BJ. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc Natl Acad Sci U S A 1996; 93: 140827.
  • 33
    Xiao X, Li J, Samulski RJ. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J Virol 1996; 70: 8098108.
  • 34
    Herzog RW, Hagstrom JN, Kung SH, Tai SJ, Wilson JM, Fisher KJ, High KA. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci U S A 1997; 94: 58049.
  • 35
    Herzog RW, Yang EY, Couto LB, Hagstrom JN, Elwell D, Fields PA, Burton M, Bellinger DA, Read MS, Brinkhous KM, Podsakoff GM, Nichols TC, Kurtzman GJ, High KA. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med 1999; 5: 5663.
  • 36
    Herzog RW, Mount JD, Arruda VR, High KA, Lothrop CD Jr. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation. Mol Ther 2001; 4: 192200.
  • 37
    Herzog RW, Fields PA, Arruda VR, Brubaker JO, Armstrong E, McClintock D, Bellinger DA, Couto LB, Nichols TC, High KA. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther 2002; 13: 128191.
  • 38
    Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi Y, Mingozzi F, Xiao W, Couto LB, High KA. Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1. Blood 2004; 103: 8592.
  • 39
    Su LT, Gopal K, Wang Z, Yin X, Nelson A, Kozyak BW, Burkman JM, Mitchell MA, Low DW, Bridges CR, Stedman HH. Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector. Circulation 2005; 112: 17808.
  • 40
    Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA. Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B. Blood 2010; 115: 467888.
  • 41
    Powers W, Fan Z, Kocis K, Valley R, Chopra M, Howard J, Muenzer J. Safety and feasibility of transvenous limb perfusion with saline in human muscular dystrophy. Mol Ther 2010; 18(Suppl. 1): S126.
  • 42
    Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA. Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 2000; 24: 25761.
  • 43
    Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood 2003; 101: 296372.
  • 44
    Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med 1999; 5: 6470.
  • 45
    Mount JD, Herzog RW, Tillson DM, Goodman SA, Robinson N, McCleland ML, Bellinger D, Nichols TC, Arruda VR, Lothrop CD Jr, High KA. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 2002; 99: 26706.
  • 46
    Mingozzi F, Liu YL, Dobrzynski E, Kaufhold A, Liu JH, Wang Y, Arruda VR, High KA, Herzog RW. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest 2003; 111: 134756.
  • 47
    Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA. CD8(+) T-cell responses to adeno-associated virus capsid in humans. Nat Med 2007; 13: 41922.
  • 48
    Pien GC, Basner-Tschakarjan E, Hui DJ, Mentlik AN, Finn JD, Hasbrouck NC, Zhou S, Murphy SL, Maus MV, Mingozzi F, Orange JS, High KA. Capsid antigen presentation flags human hepatocytes for destruction after transduction by adeno-associated viral vectors. J Clin Invest 2009; 119: 168895.
  • 49
    Li C, Goudy K, Hirsch M, Asokan A, Fan Y, Alexander J, Sun J, Monahan P, Seiber D, Sidney J, Sette A, Tisch R, Frelinger J, Samulski RJ. Cellular immune response to cryptic epitopes during therapeutic gene transfer. Proc Natl Acad Sci U S A 2009; 106: 107704.
  • 50
    Report on immune responses to adeno-associated virus (AAV) vectors. Office of Biotechnology Activities, NIH Recombinant DNA Advisory Committee Meeting, June 19, 2007. 2007. http://oba.od.nih.gov/rdna_rac/rac_meetings.html. Accessed 13 June 2011.
  • 51
    Vandenberghe LH, Wang L, Somanathan S, Zhi Y, Figueredo J, Calcedo R, Sanmiguel J, Desai RA, Chen CS, Johnston J, Grant RL, Gao G, Wilson JM. Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid. Nat Med 2006; 12: 96771.
  • 52
    Gene transfer for subjects with hemophilia B factor IX deficiency. Clinical Trials.gov Identifier: NCT00515710. 2007. http://clinicaltrials.gov/ct2/show/NCT00515710?term=Hemophilia+B+AND+ Philadelphia&rank=1. Accessed 13 June 2011.
  • 53
    Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, Edmonson SA, Hui DJ, Sabatino DE, Zhou S, Wright JF, Jiang H, Pierce GF, Arruda VR, High KA. Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver. Blood 2007; 110: 233441.
  • 54
    Gaudet D, de Wal J, Tremblay K, Dery S, van Deventer S, Freidig A, Brisson D, Methot J. Review of the clinical development of alipogene tiparvovec gene therapy for lipoprotein lipase deficiency. Atheroscler Suppl 2010; 11: 5560.
  • 55
    Gao GP, Alvira MR, Wang L, Calcedo R, Johnston J, Wilson JM. Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci U S A 2002; 99: 118549.
  • 56
    McCarty DM, Fu H, Monahan PE, Toulson CE, Naik P, Samulski RJ. Adeno-associated virus terminal repeat (TR) mutant generates self-complementary vectors to overcome the rate-limiting step to transduction in vivo. Gene Ther 2003; 10: 21128.
  • 57
    Nathwani AC, Gray JT, Ng CY, Zhou J, Spence Y, Waddington SN, Tuddenham EG, Kemball-Cook G, McIntosh J, Boon-Spijker M, Mertens K, Davidoff AM. 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: 265361.
  • 58
    Nathwani AC, Tuddenham E, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, High KA, Mingozzi F, Gray JT, Reiss UM, Nienhuis AW, Davidoff A. Early clinical trial results following administration of a low dose of a novel self complementary adeno-associated viral vector encoding human Factor IX in two subjects with severe hemophilia B. Blood 2010; 116: 114.
  • 59
    Calcedo R, Vandenberghe LH, Gao G, Lin J, Wilson JM. Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses. J Infect Dis 2009; 199: 38190.
  • 60
    Basner-Tschakarjan E, Mingozzi F, Chen Y, Nathwani AC, Tuddenham EGD, Rosales C, McIntosh J, Riddell A, Rustagi P, Glader B, Kay M, Allay J, Coleman J, Sleep S, Gray J, Reiss U, Nienhuis AW, Davidoff AM, High KA. Dose-dependent activation of capsid-specific T cells after AAV serotype 8 vector administration in a clinical study for hemophilia. Mol Ther 2011; 19(Suppl 1): S230.
  • 61
    Ponder KP. Hemophilia gene therapy: a holy grail found. Mol Ther 2011; 19: 4278.
  • 62
    McIntosh J, Lenting PJ, Tuddenham E, Sontannde M, Waddington SN, Gray JT, Davidoff A, Nathwani A. Stable high level coagulation Factor VIII expressin in vivo follwing gene transfer using a novel expression cassette encoding a more potent FVIII variant. Blood 2010; 116: 115.
  • 63
    Simioni P, Tormene D, Tognin G, Gavasso S, Bulato C, Iacobelli NP, Finn JD, Spiezia L, Radu C, Arruda VR. X-linked thrombophilia with a mutant factor IX (factor IX Padua). N Engl J Med 2009; 361: 16715.
  • 64
    Ward NJ, Buckley SM, Waddington SN, Vandendriessche T, Chuah MK, Nathwani AC, McIntosh J, Tuddenham EG, Kinnon C, Thrasher AJ, McVey JH. Codon optimization of human factor VIII cDNAs leads to high-level expression. Blood 2011; 117: 798807.
  • 65
    Donsante A, Miller DG, Li Y, Vogler C, Brunt EM, Russell DW, Sands MS. AAV vector integration sites in mouse hepatocellular carcinoma. Science 2007; 317: 477.
  • 66
    Li H, Malani N, Hamilton SR, Schlachterman A, Bussadori G, Edmonson SE, Shah R, Arruda VR, Mingozzi F, Wright JF, Bushman FD, High KA. Assessing the potential for AAV vector genotoxicity in a murine model. Blood 2011; 117: 33119.
  • 67
    Goedert JJ, Brown DL, Hoots K, Sherman KE. Human immunodeficiency and hepatitis virus infections and their associated conditions and treatments among people with haemophilia. Haemophilia 2004; 10(Suppl. 4): 20510.
  • 68
    Intraobserver and interobserver variations in liver biopsy interpretation in patients with chronic hepatitis C. The French METAVIR Cooperative Study Group. Hepatology 1994; 20: 1520.
  • 69
    Miller DG, Trobridge GD, Petek LM, Jacobs MA, Kaul R, Russell DW. Large-scale analysis of adeno-associated virus vector integration sites in normal human cells. J Virol 2005; 79: 1143442.
  • 70
    Nakai H, Wu X, Fuess S, Storm TA, Munroe D, Montini E, Burgess SM, Grompe M, Kay MA. Large-scale molecular characterization of adeno-associated virus vector integration in mouse liver. J Virol 2005; 79: 360614.
  • 71
    Donsante A, Vogler C, Muzyczka N, Crawford JM, Barker J, Flotte T, Campbell-Thompson M, Daly T, Sands MS. Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors. Gene Ther 2001; 8: 13436.
  • 72
    Buchlis G, Podsakoff GM, Radu A, Flake AW, Mingozzi F, High KA. Factor IX expressoin in muscle of a severe hemophilia B patient 10 years after AAV gene transfer. Mol Ther 2011; 19(Suppl 1): S314.
  • 73
    Jiang H, Pierce GF, Ozelo MC, de Paula EV, Vargas JA, Smith P, Sommer J, Luk A, Manno CS, High KA, Arruda VR. Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B. Mol Ther 2006; 14: 4525.
  • 74
    Russell KE, Olsen EH, Raymer RA, Merricks EP, Bellinger DA, Read MS, Rup BJ, Keith JC Jr, McCarthy KP, Schaub RG, Nichols TC. Reduced bleeding events with subcutaneous administration of recombinant human factor IX in immune-tolerant hemophilia B dogs. Blood 2003; 102: 43938.
  • 75
    Yarovoi HV, Kufrin D, Eslin DE, Thornton MA, Haberichter SL, Shi Q, Zhu H, Camire R, Fakharzadeh SS, Kowalska MA, Wilcox DA, Sachais BS, Montgomery RR, Poncz M. Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment. Blood 2003; 102: 400613.
  • 76
    Shi Q, Wilcox DA, Fahs SA, Fang J, Johnson BD, Du LM, Desai D, Montgomery RR. Lentivirus-mediated platelet-derived factor VIII gene therapy in murine haemophilia A. J Thromb Haemost 2007; 5: 35261.
  • 77
    Gewirtz J, Thornton MA, Rauova L, Poncz M. Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors. J Thromb Haemost 2008; 6: 11606.
  • 78
    Shi Q, Fahs SA, Wilcox DA, Kuether EL, Morateck PA, Mareno N, Weiler H, Montgomery RR. 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: 271321.
  • 79
    Neyman M, Gewirtz J, Poncz M. Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots. Blood 2008; 112: 11018.
  • 80
    Chang AH, Stephan MT, Sadelain M. Stem cell-derived erythroid cells mediate long-term systemic protein delivery. Nat Biotechnol 2006; 24: 101721.
  • 81
    Margaritis P, Arruda VR, Aljamali M, Camire RM, Schlachterman A, High KA. Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII. J Clin Invest 2004; 113: 102531.
  • 82
    Aljamali MN, Margaritis P, Schlachterman A, Tai SJ, Roy E, Bunte R, Camire RM, High KA. Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality. J Clin Invest 2008; 118: 182534.
  • 83
    Margaritis P, Roy E, Aljamali MN, Downey HD, Giger U, Zhou S, Merricks E, Dillow A, Ezban M, Nichols TC, High KA. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood 2009; 113: 36829.
  • 84
    Margaritis P, Roy E, Faella A, Downey HD, Ivanciu L, Pavani G, Zhou S, Bunte RM, High KA. Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo. Blood 2011; 117: 397482.
  • 85
    Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, Cheng SH. AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice. Mol Ther 2004; 9: 23140.
  • 86
    Evans GL, Morgan RA. Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc Natl Acad Sci U S A 1998; 95: 57349.
  • 87
    Finn JD, Ozelo MC, Sabatino DE, Franck HW, Merricks EP, Crudele JM, Zhou S, Kazazian HH, Lillicrap D, Nichols TC, Arruda VR. Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy. Blood 2010; 116: 58428.
  • 88
    Urnov FD, Miller JC, Lee YL, Beausejour CM, Rock JM, Augustus S, Jamieson AC, Porteus MH, Gregory PD, Holmes MC. Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 2005; 435: 64651.
  • 89
    Li H, Haurigot V, Doyon Y, Li J, Bhagwat A, Wong S, Anguela X, Sharma R, Ivanciu L, Murphy S, Zhou Z, Paschon D, Rebar E, Gregory PD, Holmes MC, High KA. Phenotypic Correction of a Mouse Model of Hemophilia B by In Vivo Genetic Correction of the F9 Gene. 52nd ASH Annual Meeting and Exposition. Orlando, FL, 2010.
  • 90
    Autologous T-cells genetically modified at the CCR5 gene by zinc finger nucleases SB-728 for HIV (Zinc-Finger). Clinical Trials.gov Identifier: NCT00842634. 2009. http://clinicaltrials.gov/ct2/show/NCT00842634?term=Zinc+Finger&rank=1. Accessed 13 June 2011.
  • 91
    Brunetti-Pierri N, Nichols TC, McCorquodale S, Merricks E, Palmer DJ, Beaudet AL, Ng P. Sustained phenotypic correction of canine hemophilia B after systemic administration of helper-dependent adenoviral vector. Hum Gene Ther 2005; 16: 81120.