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Volumn 108, Issue 5, 2011, Pages 2082-2087

Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII

Author keywords

Coagulation; Immune response

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8;

EID: 79952122629     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1015571108     Document Type: Article
Times cited : (45)

References (57)
  • 1
    • 0038383605 scopus 로고    scopus 로고
    • The epidemiology of inhibitors in haemophilia A: A systematic review
    • Wight J, Paisley S (2003) The epidemiology of inhibitors in haemophilia A: A systematic review. Haemophilia 9:418-435. (Pubitemid 36874391)
    • (2003) Haemophilia , vol.9 , Issue.4 , pp. 418-435
    • Wight, J.1    Paisley, S.2
  • 2
    • 8844263239 scopus 로고    scopus 로고
    • Pathogenic antibodies to coagulation factors. Part one: Factor VIII and factor IX
    • Lollar P (2004) Pathogenic antibodies to coagulation factors. Part one: Factor VIII and factor IX. J Thromb Haemost 2:1082-1095.
    • (2004) J Thromb Haemost , vol.2 , pp. 1082-1095
    • Lollar, P.1
  • 3
    • 44049094812 scopus 로고    scopus 로고
    • Gene delivery to the juvenile mouse liver using AAV2/8 vectors
    • DOI 10.1038/mt.2008.72, PII MT200872
    • Cunningham SC, Dane AP, Spinoulas A, Logan GJ, Alexander IE (2008) Gene delivery to the juvenile mouse liver using AAV2/8 vectors. Mol Ther 16:1081-1088. (Pubitemid 351737073)
    • (2008) Molecular Therapy , vol.16 , Issue.6 , pp. 1081-1088
    • Cunningham, S.C.1    Dane, A.P.2    Spinoulas, A.3    Alexander, I.E.4
  • 5
    • 77950859231 scopus 로고    scopus 로고
    • Neonatal gene transfer using lentiviral vector for murine Pompe disease: Long-term expression and glycogen reduction
    • Kyosen SO, et al. (2010) Neonatal gene transfer using lentiviral vector for murine Pompe disease: Long-term expression and glycogen reduction. Gene Ther 17:521-530.
    • (2010) Gene Ther , vol.17 , pp. 521-530
    • Kyosen, S.O.1
  • 8
    • 35348968497 scopus 로고    scopus 로고
    • Immunology of neonatal gene transfer
    • Ponder KP (2007) Immunology of neonatal gene transfer. Curr Gene Ther 7:403-410.
    • (2007) Curr Gene Ther , vol.7 , pp. 403-410
    • Ponder, K.P.1
  • 9
    • 0029970829 scopus 로고    scopus 로고
    • Long term correction of bilirubin-UDP-glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period
    • DOI 10.1074/jbc.271.43.26536
    • Takahashi M, et al. (1996) Long term correction of bilirubin-UDP- glucuronosyltransferase deficiency in Gunn rats by administration of a recombinant adenovirus during the neonatal period. J Biol Chem271:26536-26542. (Pubitemid 26359052)
    • (1996) Journal of Biological Chemistry , vol.271 , Issue.43 , pp. 26536-26542
    • Takahashi, M.1    Ilan, Y.2    Chowdhury, N.R.3    Guida, J.4    Horwitz, M.5    Chowdhury, J.R.6
  • 11
    • 0036932904 scopus 로고    scopus 로고
    • Evaluation of pathological manifestations of disease in mucopolysaccharidosisVIImice after neonatal hepatic genetherapy
    • Xu L, et al. (2002) Evaluation of pathological manifestations of disease in mucopolysaccharidosisVIImice after neonatal hepatic genetherapy.Mol Ther 6:745-758.
    • (2002) Mol Ther , vol.6 , pp. 745-758
    • Xu, L.1
  • 12
    • 0036792035 scopus 로고    scopus 로고
    • Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs
    • Ponder KP, et al. (2002) Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs. Proc Natl Acad Sci USA 99:13102-13107.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 13102-13107
    • Ponder, K.P.1
  • 14
    • 77749249680 scopus 로고    scopus 로고
    • Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN
    • Foust KD, et al. (2010) Rescue of the spinal muscular atrophy phenotype in a mouse model by early postnatal delivery of SMN. Nat Biotechnol 28:271-274.
    • (2010) Nat Biotechnol , vol.28 , pp. 271-274
    • Foust, K.D.1
  • 15
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina S, et al. (2008) Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118:3132-3142.
    • (2008) J Clin Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1
  • 17
    • 77952009127 scopus 로고    scopus 로고
    • Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression
    • Ginn SL, et al. (2010) Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and gammac overexpression. Mol Ther 18:965-976.
    • (2010) Mol Ther , vol.18 , pp. 965-976
    • Ginn, S.L.1
  • 18
    • 36348953133 scopus 로고    scopus 로고
    • Correction of murine hemophilia a and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors
    • DOI 10.1038/sj.mt.6300308, PII 6300308
    • Cerullo V, et al. (2007) Correction of murine hemophilia A and immunological differences of factor VIII variants delivered by helper-dependent adenoviral vectors. Mol Ther 15:2080-2087. (Pubitemid 350148922)
    • (2007) Molecular Therapy , vol.15 , Issue.12 , pp. 2080-2087
    • Cerullo, V.1    Seiler, M.P.2    Mane, V.3    Cela, R.4    Clarke, C.5    Kaufman, R.J.6    Pipe, S.W.7    Lee, B.8
  • 19
    • 0026635406 scopus 로고
    • Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B
    • Nilsson IM, Berntorp E, Löfqvist T, Pettersson H (1992) Twenty-five years' experience of prophylactic treatment in severe haemophilia A and B. J Intern Med 232:25-32.
    • (1992) J Intern Med , vol.232 , pp. 25-32
    • Nilsson, I.M.1    Berntorp, E.2    Löfqvist, T.3    Pettersson, H.4
  • 21
    • 0036588692 scopus 로고    scopus 로고
    • Paediatric care of the child with haemophilia
    • Ljung R (2002) Paediatric care of the child with haemophilia. Haemophilia 8:178-182.
    • (2002) Haemophilia , vol.8 , pp. 178-182
    • Ljung, R.1
  • 22
    • 0141482007 scopus 로고    scopus 로고
    • Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS study group
    • DOI 10.1182/blood-2003-03-0941
    • Gringeri A, Mantovani LG, Scalone L, Mannucci PM; COCIS Study Group (2003) Cost of care and quality of life for patients with hemophilia complicated by inhibitors: The COCIS Study Group. Blood 102:2358-2363. (Pubitemid 37193570)
    • (2003) Blood , vol.102 , Issue.7 , pp. 2358-2363
    • Gringeri, A.1    Mantovani, L.G.2    Scalone, L.3    Mannucci, P.M.4
  • 24
    • 0042626369 scopus 로고    scopus 로고
    • The incidence of inhibitor development according to specific mutations - and treatment?
    • Goodeve A (2003) The incidence of inhibitor development according to specific mutations - and treatment? Blood Coagul Fibrinolysis 14(Suppl 1):S17-S21.
    • (2003) Blood Coagul Fibrinolysis , vol.14 , Issue.SUPPL. 1
    • Goodeve, A.1
  • 27
    • 64749096663 scopus 로고    scopus 로고
    • Inhibitors of factor VIII in black patients with hemophilia
    • Viel KR, et al. (2009) Inhibitors of factor VIII in black patients with hemophilia. N Engl J Med 360:1618-1627.
    • (2009) N Engl J Med , vol.360 , pp. 1618-1627
    • Viel, K.R.1
  • 28
    • 0034994489 scopus 로고    scopus 로고
    • Incidence of factor VIII inhibitors in severe haemophilia: The importance of patient age
    • DOI 10.1046/j.1365-2141.2001.02828.x
    • Lorenzo JI, López A, Altisent C, Aznar JA (2001) Incidence of factor VIII inhibitors in severe haemophilia: The importance of patient age. Br J Haematol 113:600-603. (Pubitemid 32524377)
    • (2001) British Journal of Haematology , vol.113 , Issue.3 , pp. 600-603
    • Lorenzo, J.I.1    Lopez, A.2    Altisent, C.3    Aznar, J.A.4
  • 29
    • 30344434999 scopus 로고    scopus 로고
    • Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A
    • FVIII-LFB and Recombinant FVIII study groups
    • Goudemand J, et al.; FVIII-LFB and Recombinant FVIII study groups (2006) Influence of the type of factor VIII concentrate on the incidence of factor VIII inhibitors in previously untreated patients with severe hemophilia A. Blood 107:46-51.
    • (2006) Blood , vol.107 , pp. 46-51
    • Goudemand, J.1
  • 30
    • 0031598278 scopus 로고    scopus 로고
    • Inhibitors occur more frequently in African-American and Latino haemophiliacs
    • Aledort LM, Dimichele DM (1998) Inhibitors occur more frequently in African-American and Latino haemophiliacs. Haemophilia 4:68.
    • (1998) Haemophilia , vol.4 , pp. 68
    • Aledort, L.M.1    Dimichele, D.M.2
  • 31
    • 0027473752 scopus 로고
    • Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors
    • Kogenate Previously Untreated Patient Study Group
    • Lusher JM, Arkin S, Abildgaard CF, Schwartz RS; Kogenate Previously Untreated Patient Study Group (1993) Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. N Engl J Med 328:453-459.
    • (1993) N Engl J Med , vol.328 , pp. 453-459
    • Lusher, J.M.1    Arkin, S.2    Abildgaard, C.F.3    Schwartz, R.S.4
  • 32
    • 1342272118 scopus 로고    scopus 로고
    • Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A
    • Qian J, Borovok M, Bi L, Kazazian HH, Jr, Hoyer LW (1999) Inhibitor antibody development and T cell response to human factor VIII in murine hemophilia A. Thromb Haemost 81:240-244. (Pubitemid 29078310)
    • (1999) Thrombosis and Haemostasis , vol.81 , Issue.2 , pp. 240-244
    • Qian, J.1    Borovok, M.2    Bi, L.3    Kazazian Jr., H.H.4    Hoyer, L.W.5
  • 33
    • 0035874523 scopus 로고    scopus 로고
    • Induction of tolerance to human factor VIII in mice
    • Chao H, Walsh CE (2001) Induction of tolerance to human factor VIII in mice. Blood 97:3311-3312.
    • (2001) Blood , vol.97 , pp. 3311-3312
    • Chao, H.1    Walsh, C.E.2
  • 35
    • 0034630337 scopus 로고    scopus 로고
    • Partial correction of murine hemophilia A with neo-antigenic murine factor VIII
    • DOI 10.1089/10430340050015491
    • Sarkar R, Gao GP, Chirmule N, Tazelaar J, Kazazian HH, Jr. (2000) Partial correction of murine hemophilia A with neo-antigenic murine factor VIII. HumGene Ther 11:881-894. (Pubitemid 30202479)
    • (2000) Human Gene Therapy , vol.11 , Issue.6 , pp. 881-894
    • Sarkar, R.1    Gao, G.-P.2    Chirmule, N.3    Tazelaar, J.4    Kazazian Jr., H.H.5
  • 40
    • 0742307422 scopus 로고    scopus 로고
    • Acute Toxicity after High-Dose Systemic Injection of Helper-Dependent Adenoviral Vectors into Nonhuman Primates
    • DOI 10.1089/10430340460732445
    • Brunetti-Pierri N, et al. (2004) Acute toxicity after high-dose systemic injection of helper-dependent adenoviral vectors into nonhuman primates. Hum Gene Ther 15:35-46. (Pubitemid 38147187)
    • (2004) Human Gene Therapy , vol.15 , Issue.1 , pp. 35-46
    • Brunetti-Pierri, N.1    Palmer, D.J.2    Beaudet, A.L.3    Carey, K.D.4    Finegold, M.5    Ng, P.6
  • 42
    • 0033541522 scopus 로고    scopus 로고
    • Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo
    • DOI 10.1089/10430349950018364
    • Muruve DA, Barnes MJ, Stillman IE, Libermann TA (1999) Adenoviral gene therapy leads to rapid induction of multiple chemokines and acute neutrophil-dependent hepatic injury in vivo. Hum Gene Ther 10:965-976. (Pubitemid 29195426)
    • (1999) Human Gene Therapy , vol.10 , Issue.6 , pp. 965-976
    • Muruve, D.A.1    Barnes, M.J.2    Stillman, I.E.3    Libermann, T.A.4
  • 44
    • 0028814316 scopus 로고
    • Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep-vein thrombosis
    • Koster T, Blann AD, Briët E, Vandenbroucke JP, Rosendaal FR (1995) Role of clotting factor VIII in effect of von Willebrand factor on occurrence of deep-vein thrombosis. Lancet 345:152-155.
    • (1995) Lancet , vol.345 , pp. 152-155
    • Koster, T.1    Blann, A.D.2    Briët, E.3    Vandenbroucke, J.P.4    Rosendaal, F.R.5
  • 46
    • 36048956490 scopus 로고    scopus 로고
    • Absolute annual incidences of first events of venous thromboembolism and arterial vascular events in individuals with elevated FVIII:c A prospective family cohort study
    • DOI 10.1160/TH07-04-0293
    • Bank I, et al. (2007) Absolute annual incidences of first events of venous thromboembolism and arterial vascular events in individuals with elevated FVIII:c. A prospective family cohort study. Thromb Haemost 98:1040-1044. (Pubitemid 350091133)
    • (2007) Thrombosis and Haemostasis , vol.98 , Issue.5 , pp. 1040-1044
    • Bank, I.1    De Van, P.M.H.W.2    Coppens, M.3    Hamulyak, K.4    Prins, M.H.5    Van, D.M.J.6    Veeger, N.J.G.M.7    Middeldorp, S.8
  • 48
    • 0034655277 scopus 로고    scopus 로고
    • Persistence of physiological self antigen is required for the regulation of self tolerance
    • Garza KM, Agersborg SS, Baker E, Tung KS (2000) Persistence of physiological self antigen is required for the regulation of self tolerance. J Immunol 164:3982-3989.
    • (2000) J Immunol , vol.164 , pp. 3982-3989
    • Garza, K.M.1    Agersborg, S.S.2    Baker, E.3    Tung, K.S.4
  • 49
    • 0027532184 scopus 로고
    • Clonal anergy blocks in vivo growth of mature T cells and can be reversed in the absence of antigen
    • Rocha B, Tanchot C, Von Boehmer H (1993) Clonal anergy blocks in vivo growth of mature T cells and can be reversed in the absence of antigen. J Exp Med 177:1517-1521. (Pubitemid 23117145)
    • (1993) Journal of Experimental Medicine , vol.177 , Issue.5 , pp. 1517-1521
    • Rocha, B.1    Tanchot, C.2    Von, B.H.3
  • 50
    • 0001525604 scopus 로고
    • On the mechanism of immunologic self recognition
    • Triplett EL (1962) On the mechanism of immunologic self recognition. J Immunol 89:505-510.
    • (1962) J Immunol , vol.89 , pp. 505-510
    • Triplett, E.L.1
  • 51
    • 20844460836 scopus 로고    scopus 로고
    • Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors
    • DOI 10.1016/j.ymthe.2005.03.001, PII S1525001605000870
    • Brunetti-Pierri N, et al. (2005) Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol Ther 12:99-106. (Pubitemid 40862361)
    • (2005) Molecular Therapy , vol.12 , Issue.1 , pp. 99-106
    • Brunetti-Pierri, N.1    Palmer, D.J.2    Mane, V.3    Finegold, M.4    Beaudet, A.L.5    Ng, P.6
  • 52
    • 0346872991 scopus 로고    scopus 로고
    • Improved system for helper-dependent adenoviral vector production
    • DOI 10.1016/j.ymthe.2003.08.014
    • Palmer D, Ng P (2003) Improved system for helper-dependent adenoviral vector production. Mol Ther 8:846-852. (Pubitemid 37527461)
    • (2003) Molecular Therapy , vol.8 , Issue.5 , pp. 846-852
    • Palmer, D.1    Ng, P.2
  • 53
    • 4644252075 scopus 로고    scopus 로고
    • Physical and infectious titers of helper-dependent adenoviral vectors: A method of direct comparison to the adenovirus reference material
    • DOI 10.1016/j.ymthe.2004.06.1013, PII S1525001604013085
    • Palmer DJ, Ng P (2004) Physical and infectious titers of helper-dependent adenoviral vectors: A method of direct comparison to the adenovirus reference material. Mol Ther 10:792-798. (Pubitemid 39272868)
    • (2004) Molecular Therapy , vol.10 , Issue.4 , pp. 792-798
    • Palmer, D.J.1    Ng, P.2
  • 54
    • 0035988782 scopus 로고    scopus 로고
    • Preparation of helper-dependent adenoviral vectors
    • Ng P, Parks RJ, Graham FL (2002) Preparation of helper-dependent adenoviral vectors. Methods Mol Med 69:371-388.
    • (2002) Methods Mol Med , vol.69 , pp. 371-388
    • Ng, P.1    Parks, R.J.2    Graham, F.L.3
  • 55
    • 0029011723 scopus 로고
    • Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A
    • Bi L, et al. (1995) Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 10:119-121.
    • (1995) Nat Genet , vol.10 , pp. 119-121
    • Bi, L.1
  • 57
    • 0018745986 scopus 로고
    • Bleeding time in laboratory animals. II - A comparison of different assay conditions in rats
    • Dejana E, Callioni A, Quintana A, de Gaetano G (1979) Bleeding time in laboratory animals. II - A comparison of different assay conditions in rats. Thromb Res 15:191-197.
    • (1979) Thromb Res , vol.15 , pp. 191-197
    • Dejana, E.1    Callioni, A.2    Quintana, A.3    De Gaetano, G.4


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