메뉴 건너뛰기




Volumn 103, Issue 10, 2004, Pages 3700-3709

Targeting lentiviral vector expression to hepatocytes limits transgene-specific immune response and establishes long-term expression of human antihemophilic factor IX in mice

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBODY; BLOOD CLOTTING FACTOR 9; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR;

EID: 2342561729     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2003-09-3217     Document Type: Article
Times cited : (184)

References (48)
  • 1
    • 0035135747 scopus 로고    scopus 로고
    • Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics
    • Kay MA, Glorioso JC, Naldini L. Viral vectors for gene therapy: the art of turning infectious agents into vehicles of therapeutics. Nat Med. 2001; 7:33-40.
    • (2001) Nat Med , vol.7 , pp. 33-40
    • Kay, M.A.1    Glorioso, J.C.2    Naldini, L.3
  • 2
    • 0035676918 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Pasi KJ. Gene therapy for haemophilia. Br J Haematol. 2001;115:744-757.
    • (2001) Br J Haematol , vol.115 , pp. 744-757
    • Pasi, K.J.1
  • 3
    • 0037325357 scopus 로고    scopus 로고
    • Gene transfer as an approach to treating hemophilia
    • High KA. Gene transfer as an approach to treating hemophilia. Semin Thromb Hemost. 2003;29: 107-120.
    • (2003) Semin Thromb Hemost , vol.29 , pp. 107-120
    • High, K.A.1
  • 4
    • 0037103167 scopus 로고    scopus 로고
    • Dangerous liaisons: The role of "danger" signals in the immune response to gene therapy
    • Brown BD, Lillicrap D. Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy. Blood. 2002;100:1133-1140.
    • (2002) Blood , vol.100 , pp. 1133-1140
    • Brown, B.D.1    Lillicrap, D.2
  • 5
    • 0036776745 scopus 로고    scopus 로고
    • Antigen degradation or presentation by MHC class I molecules via classical and non-classical pathways
    • Gromme M, Neefjes J. Antigen degradation or presentation by MHC class I molecules via classical and non-classical pathways. Mol Immunol. 2002;39:181-202.
    • (2002) Mol Immunol , vol.39 , pp. 181-202
    • Gromme, M.1    Neefjes, J.2
  • 6
    • 0038446697 scopus 로고    scopus 로고
    • Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells
    • De Geest BR, Van Linthout SA, Collen D. Humoral immune response in mice against a circulating antigen induced by adenoviral transfer is strictly dependent on expression in antigen-presenting cells. Blood. 2003;101:2551-2556.
    • (2003) Blood , vol.101 , pp. 2551-2556
    • De Geest, B.R.1    Van Linthout, S.A.2    Collen, D.3
  • 7
    • 0035080378 scopus 로고    scopus 로고
    • Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice
    • Fields PA, Armstrong E, Hagstrom JN, et al. Intravenous administration of an E1/E3-deleted adenoviral vector induces tolerance to factor IX in C57BL/6 mice. Gene Ther. 2001;8:354-361.
    • (2001) Gene Ther , vol.8 , pp. 354-361
    • Fields, P.A.1    Armstrong, E.2    Hagstrom, J.N.3
  • 8
    • 0034151775 scopus 로고    scopus 로고
    • Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX
    • Fields PA, Kowalczyk DW, Arruda VR, et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX. Mol Ther. 2000;1:225-235.
    • (2000) Mol Ther , vol.1 , pp. 225-235
    • Fields, P.A.1    Kowalczyk, D.W.2    Arruda, V.R.3
  • 9
    • 0036376642 scopus 로고    scopus 로고
    • Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy
    • Herzog RW, Fields PA, Arruda VR, et al. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy. Hum Gene Ther. 2002;13:1281-1291.
    • (2002) Hum Gene Ther , vol.13 , pp. 1281-1291
    • Herzog, R.W.1    Fields, P.A.2    Arruda, V.R.3
  • 10
    • 0030999469 scopus 로고    scopus 로고
    • Adenovirus-mediated gene transfer: Influence of transgene, mouse strain and type of immune response on persistence of transgene expression
    • Michou AI, Santoro L, Christ M, Julliard V, Pavirani A, Mehtali M. Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression. Gene Ther. 1997;4:473-482.
    • (1997) Gene Ther , vol.4 , pp. 473-482
    • Michou, A.I.1    Santoro, L.2    Christ, M.3    Julliard, V.4    Pavirani, A.5    Mehtali, M.6
  • 11
    • 0036171327 scopus 로고    scopus 로고
    • Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors
    • Follenzi A, Sabatino G, Lombardo A, Boccaccio C, Naldini L. Efficient gene delivery and targeted expression to hepatocytes in vivo by improved lentiviral vectors. Hum Gene Ther. 2002;13:243-260.
    • (2002) Hum Gene Ther , vol.13 , pp. 243-260
    • Follenzi, A.1    Sabatino, G.2    Lombardo, A.3    Boccaccio, C.4    Naldini, L.5
  • 12
    • 0034989411 scopus 로고    scopus 로고
    • Transduction of liver cells by lentiviral vectors: Analysis in living animals by fluorescence imaging
    • Pfeifer A, Kessler T, Yang M, et al. Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. Mol Ther. 2001; 3:319-322.
    • (2001) Mol Ther , vol.3 , pp. 319-322
    • Pfeifer, A.1    Kessler, T.2    Yang, M.3
  • 13
    • 0036682975 scopus 로고    scopus 로고
    • Lentiviral vectors containing the human immuno-deficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo
    • Vandendriessche T, Thorrez L, Naldiril L, et al. Lentiviral vectors containing the human immuno-deficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood. 2002; 100:813-822.
    • (2002) Blood , vol.100 , pp. 813-822
    • Vandendriessche, T.1    Thorrez, L.2    Naldiril, L.3
  • 14
    • 0036137639 scopus 로고    scopus 로고
    • Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector
    • Tsui LV, Kelly M, Zayek N, et al. Production of human clotting Factor IX without toxicity in mice after vascular delivery of a lentiviral vector. Nat Biotechnol. 2002;20:53-57.
    • (2002) Nat Biotechnol , vol.20 , pp. 53-57
    • Tsui, L.V.1    Kelly, M.2    Zayek, N.3
  • 15
    • 0034254320 scopus 로고    scopus 로고
    • Therapeutic levels of human factor VIII and IX using HIV-1-based lenti-viral vectors in mouse liver
    • Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lenti-viral vectors in mouse liver. Blood. 2000;96:1173-1176.
    • (2000) Blood , vol.96 , pp. 1173-1176
    • Park, F.1    Ohashi, K.2    Kay, M.A.3
  • 16
    • 0037853196 scopus 로고    scopus 로고
    • Efficient production of human FVIII in hemophilic mice using lentiviral vectors
    • Kootstra NA, Matsumura R, Verma IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther. 2003;7:623-631.
    • (2003) Mol Ther , vol.7 , pp. 623-631
    • Kootstra, N.A.1    Matsumura, R.2    Verma, I.M.3
  • 17
    • 0033694598 scopus 로고    scopus 로고
    • Universal GFP reporter for the study of vascular development
    • Motoike T, Loughna S, Perens E, et al. Universal GFP reporter for the study of vascular development. Genesis. 2000;28:75-81.
    • (2000) Genesis , vol.28 , pp. 75-81
    • Motoike, T.1    Loughna, S.2    Perens, E.3
  • 18
    • 0038644403 scopus 로고    scopus 로고
    • Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells
    • De Palma M, Venneri MA, Roca C, Naldini L. Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat Med. 2003;9:789-795.
    • (2003) Nat Med , vol.9 , pp. 789-795
    • De Palma, M.1    Venneri, M.A.2    Roca, C.3    Naldini, L.4
  • 19
    • 0035860585 scopus 로고    scopus 로고
    • Cytotoxic T cell polyepitope vaccines delivered by ISCOMs
    • Le TT, Drane D, Malliaros J, et al. Cytotoxic T cell polyepitope vaccines delivered by ISCOMs. Vaccine. 2001;19:4669-4675.
    • (2001) Vaccine , vol.19 , pp. 4669-4675
    • Le, T.T.1    Drane, D.2    Malliaros, J.3
  • 20
    • 0033203120 scopus 로고    scopus 로고
    • Selective transport of internalized antigens to the cytosol for MHC class I presentation in dendritic cells
    • Rodriguez A, Regnault A, Klaijmeer M, Ricciardi-Castagnoli P, Amigorena S. Selective transport of internalized antigens to the cytosol for MHC class I presentation in dendritic cells. Nat Cell Biol. 1999;1:362-366.
    • (1999) Nat Cell Biol , vol.1 , pp. 362-366
    • Rodriguez, A.1    Regnault, A.2    Klaijmeer, M.3    Ricciardi-Castagnoli, P.4    Amigorena, S.5
  • 21
    • 0037114698 scopus 로고    scopus 로고
    • Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice: Nonobese diabetic/severe combined immunodeficient
    • Placibello W, Bruno S, Sanavio F, et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice: nonobese diabetic/severe combined immunodeficient. Blood. 2002;100:4391-4400.
    • (2002) Blood , vol.100 , pp. 4391-4400
    • Placibello, W.1    Bruno, S.2    Sanavio, F.3
  • 23
    • 0034853102 scopus 로고    scopus 로고
    • Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9
    • Fields PA, Arruda VR, Armstrong E, et al. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9. Mol Ther. 2001;4:201-210.
    • (2001) Mol Ther , vol.4 , pp. 201-210
    • Fields, P.A.1    Arruda, V.R.2    Armstrong, E.3
  • 24
    • 0035704168 scopus 로고    scopus 로고
    • The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice
    • Skelton D, Satake N, Kohn DB. The enhanced green fluorescent protein (eGFP) is minimally immunogenic in C57BL/6 mice. Gene Ther. 2001;8: 1813-1814.
    • (2001) Gene Ther , vol.8 , pp. 1813-1814
    • Skelton, D.1    Satake, N.2    Kohn, D.B.3
  • 26
    • 0028914687 scopus 로고
    • Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: Comparisons between immunocompetent and immunodeficient inbred strains
    • Barr D, Tubb J, Ferguson D, et al. Strain related variations in adenovirally mediated transgene expression from mouse hepatocytes in vivo: comparisons between immunocompetent and immunodeficient inbred strains. Gene Ther. 1995;2: 151-155.
    • (1995) Gene Ther , vol.2 , pp. 151-155
    • Barr, D.1    Tubb, J.2    Ferguson, D.3
  • 27
    • 0031969428 scopus 로고    scopus 로고
    • Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers
    • Jooss K, Yang Y, Fisher KJ, Wilson JM. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. Proc Natl Acad Sci U S A. 1998; 72:4212-4223.
    • (1998) Proc Natl Acad Sci U S A , vol.72 , pp. 4212-4223
    • Jooss, K.1    Yang, Y.2    Fisher, K.J.3    Wilson, J.M.4
  • 28
    • 0036288752 scopus 로고    scopus 로고
    • Efficient transduction of dendritic cells and induction of a T-cell response by third-generation lentivectors
    • Esslinger C, Romero P, MacDonald HR. Efficient transduction of dendritic cells and induction of a T-cell response by third-generation lentivectors. Hum Gene Ther. 2002;13:1091-1100.
    • (2002) Hum Gene Ther , vol.13 , pp. 1091-1100
    • Esslinger, C.1    Romero, P.2    MacDonald, H.R.3
  • 29
    • 0032812877 scopus 로고    scopus 로고
    • Immune response to green fluorescent protein: Implications for gene therapy
    • Stripecke R, Carmen Villacres M, et al. Immune response to green fluorescent protein: implications for gene therapy. Gene Ther. 1999;6:1305-1312.
    • (1999) Gene Ther , vol.6 , pp. 1305-1312
    • Stripecke, R.1    Carmen Villacres, M.2
  • 30
    • 0037066427 scopus 로고    scopus 로고
    • The danger model: A renewed sense of self
    • Matzinger P. The danger model: a renewed sense of self. Science. 2002;296:301-305.
    • (2002) Science , vol.296 , pp. 301-305
    • Matzinger, P.1
  • 31
    • 0038819053 scopus 로고    scopus 로고
    • In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses
    • Esslinger C, Chapatte L, Finke D, et al. In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8(+) T cell responses. J Clin Invest. 2003;111:1673-1681.
    • (2003) J Clin Invest , vol.111 , pp. 1673-1681
    • Esslinger, C.1    Chapatte, L.2    Finke, D.3
  • 32
    • 0032605547 scopus 로고    scopus 로고
    • Expanded-capacity adenoviral vectors-the helper-dependent vectors
    • Morsy MA, Caskey CT. Expanded-capacity adenoviral vectors-the helper-dependent vectors. Mol Med Today. 1999;5:18-24.
    • (1999) Mol Med Today , vol.5 , pp. 18-24
    • Morsy, M.A.1    Caskey, C.T.2
  • 33
    • 0033587482 scopus 로고    scopus 로고
    • Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors
    • Pastore L, Morral N, Zhou H, et al. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors. Hum Gene Ther. 1999;10:1773-1781.
    • (1999) Hum Gene Ther , vol.10 , pp. 1773-1781
    • Pastore, L.1    Morral, N.2    Zhou, H.3
  • 34
    • 0037988905 scopus 로고    scopus 로고
    • Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer
    • Mingozzi F, Liu YL, Dobrzynski E, et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J Clin Invest. 2003;111:1347-1356.
    • (2003) J Clin Invest , vol.111 , pp. 1347-1356
    • Mingozzi, F.1    Liu, Y.L.2    Dobrzynski, E.3
  • 35
    • 0036592086 scopus 로고    scopus 로고
    • A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo
    • Ehrhardt A, Kay MA. A new adenoviral helper-dependent vector results in long-term therapeutic levels of human coagulation factor IX at low doses in vivo. Blood. 2002;99:3923-3930.
    • (2002) Blood , vol.99 , pp. 3923-3930
    • Ehrhardt, A.1    Kay, M.A.2
  • 36
    • 0141593513 scopus 로고    scopus 로고
    • A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia
    • Ehrhardt A, Xu H, Dillow AM, Bellinger DA, Nichols TC, Kay MA. A gene-deleted adenoviral vector results in phenotypic correction of canine hemophilia B without liver toxicity or thrombocytopenia. Blood. 2003;102:2403-2411.
    • (2003) Blood , vol.102 , pp. 2403-2411
    • Ehrhardt, A.1    Xu, H.2    Dillow, A.M.3    Bellinger, D.A.4    Nichols, T.C.5    Kay, M.A.6
  • 37
    • 0037089331 scopus 로고    scopus 로고
    • Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
    • Mount JD, Herzog RW, Tillson DM, et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood. 2002;99:2670-2676.
    • (2002) Blood , vol.99 , pp. 2670-2676
    • Mount, J.D.1    Herzog, R.W.2    Tillson, D.M.3
  • 39
    • 0030997346 scopus 로고    scopus 로고
    • Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
    • 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;16:270-276.
    • (1997) Nat Genet , vol.16 , pp. 270-276
    • Snyder, R.O.1    Miao, C.H.2    Patijn, G.A.3
  • 40
    • 17444387438 scopus 로고    scopus 로고
    • Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector
    • Herzog RW, Yang EY, Couto LB, et al. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med. 1999; 5:56-63.
    • (1999) Nat Med , vol.5 , pp. 56-63
    • Herzog, R.W.1    Yang, E.Y.2    Couto, L.B.3
  • 41
    • 17444372024 scopus 로고    scopus 로고
    • Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors
    • Snyder RO, Miao C, Meuse L, et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med. 1999;5:64-70.
    • (1999) Nat Med , vol.5 , pp. 64-70
    • Snyder, R.O.1    Miao, C.2    Meuse, L.3
  • 42
    • 0032762405 scopus 로고    scopus 로고
    • Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity
    • Lozier JN, Metzger ME, Donahue RE, Morgan RA. Adenovirus-mediated expression of human coagulation factor IX in the rhesus macaque is associated with dose-limiting toxicity. Blood. 1999;94:3968-3975.
    • (1999) Blood , vol.94 , pp. 3968-3975
    • Lozier, J.N.1    Metzger, M.E.2    Donahue, R.E.3    Morgan, R.A.4
  • 43
    • 0034137797 scopus 로고    scopus 로고
    • Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver
    • Wang L, Nichols TC, Read MS, Bellinger DA, Verma IM. Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver. Mol Ther. 2000;1: 154-158.
    • (2000) Mol Ther , vol.1 , pp. 154-158
    • Wang, L.1    Nichols, T.C.2    Read, M.S.3    Bellinger, D.A.4    Verma, I.M.5
  • 44
    • 0034050904 scopus 로고    scopus 로고
    • Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector
    • 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;24:257-261.
    • (2000) Nat Genet , vol.24 , pp. 257-261
    • Kay, M.A.1    Manno, C.S.2    Ragni, M.V.3
  • 45
    • 0013442744 scopus 로고    scopus 로고
    • AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
    • Manno CS, Chew AJ, Hutchison S, et al. AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B. Blood. 2003;101:2963-2972.
    • (2003) Blood , vol.101 , pp. 2963-2972
    • Manno, C.S.1    Chew, A.J.2    Hutchison, S.3
  • 46
    • 0034849318 scopus 로고    scopus 로고
    • Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation
    • 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;4:192-200.
    • (2001) Mol Ther , vol.4 , pp. 192-200
    • Herzog, R.W.1    Mount, J.D.2    Arruda, V.R.3    High, K.A.4    Lothrop Jr., C.D.5
  • 47
    • 13044304200 scopus 로고    scopus 로고
    • Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
    • Morral N, O'Neal W, Rice K, et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci U S A. 1999;96:12816-12821.
    • (1999) Proc Natl Acad Sci U S A , vol.96 , pp. 12816-12821
    • Morral, N.1    O'Neal, W.2    Rice, K.3
  • 48
    • 0037372398 scopus 로고    scopus 로고
    • Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors
    • Chuah MK, Schiedner G, Thorrez L, et al. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood. 2003;101:1734-1743.
    • (2003) Blood , vol.101 , pp. 1734-1743
    • Chuah, M.K.1    Schiedner, G.2    Thorrez, L.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.