메뉴 건너뛰기




Volumn 10, Issue 22, 2003, Pages 1917-1925

Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction

Author keywords

Coagulation factor VIII; Hematopoietic stem cells; Hemophilia A; Lentiviral gene transfer; NOD SCID xenotransplantation

Indexed keywords

CD34 ANTIGEN; LENTIVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8;

EID: 0141962306     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302093     Document Type: Article
Times cited : (36)

References (49)
  • 1
    • 0035793325 scopus 로고    scopus 로고
    • Gene transfer as an approach to treating hemophilia
    • High KA. Gene transfer as an approach to treating hemophilia. Circ Res 2001; 88: 137-144.
    • (2001) Circ. Res. , vol.88 , pp. 137-144
    • High, K.A.1
  • 3
    • 0034923893 scopus 로고    scopus 로고
    • Gene therapy in hemophilia: Clinical trials update
    • White II GC. Gene therapy in hemophilia: clinical trials update. Thromb Haemost 2001; 86: 172-177.
    • (2001) Thromb. Haemost. , vol.86 , pp. 172-177
    • White II, G.C.1
  • 4
    • 0029850534 scopus 로고    scopus 로고
    • Complete short-term correction of canine hemophilia A by in vivo gene therapy
    • Connelly S et al. Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood 1996; 88: 3846-3853.
    • (1996) Blood , vol.88 , pp. 3846-3853
    • Connelly, S.1
  • 5
    • 0032080179 scopus 로고    scopus 로고
    • Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
    • Connelly S et al. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood 1998; 91: 3273-3281.
    • (1998) Blood , vol.91 , pp. 3273-3281
    • Connelly, S.1
  • 6
    • 0033539486 scopus 로고    scopus 로고
    • Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero
    • Lipshutz GS et al. Short-term correction of factor VIII deficiency in a murine model of hemophilia A after delivery of adenovirus murine factor VIII in utero. Proc Natl Acad Sci USA 1999; 96: 13324-13329.
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 13324-13329
    • Lipshutz, G.S.1
  • 7
    • 12944317288 scopus 로고    scopus 로고
    • Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector
    • Balagué C et al. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood 2000; 95: 820-828.
    • (2000) Blood , vol.95 , pp. 820-828
    • Balagué, C.1
  • 8
    • 0034630337 scopus 로고    scopus 로고
    • Partial correction of murine hemophilia A with neo-antigenic murine factor VIII
    • Sarkar R et al. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII. Hum Gene Ther 2000; 11: 881-894.
    • (2000) Hum. Gene Ther. , vol.11 , pp. 881-894
    • Sarkar, R.1
  • 9
    • 0034814009 scopus 로고    scopus 로고
    • Adenovirus-mediated factor VIII gene expression results in attenuated anti-factor VIII-specific immunity in hemophilia A mice compared with factor VIII protein infusion
    • Bristol JA et al. Adenovirus-mediated factor VIII gene expression results in attenuated anti-factor VIII-specific immunity in hemophilia A mice compared with factor VIII protein infusion. Hum Gene Ther 2001; 12: 1651-1661.
    • (2001) Hum. Gene Ther. , vol.12 , pp. 1651-1661
    • Bristol, J.A.1
  • 10
    • 0035174331 scopus 로고    scopus 로고
    • Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs
    • Gallo-Penn AM et al. Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs. Blood 2001; 97: 107-113.
    • (2001) Blood , vol.97 , pp. 107-113
    • Gallo-Penn, A.M.1
  • 11
    • 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 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 2002; 101: 1734-1743.
    • (2002) Blood , vol.101 , pp. 1734-1743
    • Chuah, M.K.1
  • 12
    • 0033607305 scopus 로고    scopus 로고
    • Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein
    • Burton M et al. Coexpression of factor VIII heavy and light chain adeno-associated viral vectors produces biologically active protein. Proc Natl Acad Sci USA 1999; 96: 12725-12730.
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 12725-12730
    • Burton, M.1
  • 13
    • 0034161486 scopus 로고    scopus 로고
    • Sustained expression of human factor VIII in mice using a parvovirus-based vector
    • Chao H, Mao L, Bruce AT, Walsh CE. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood 2000; 95: 1594-1599.
    • (2000) Blood , vol.95 , pp. 1594-1599
    • Chao, H.1    Mao, L.2    Bruce, A.T.3    Walsh, C.E.4
  • 14
    • 0035874523 scopus 로고    scopus 로고
    • Induction of tolerance to human factor VIII in mice
    • Chao H, Walsh CE. Induction of tolerance to human factor VIII in mice. Blood 2001; 97: 3311-3312.
    • (2001) Blood , vol.97 , pp. 3311-3312
    • Chao, H.1    Walsh, C.E.2
  • 15
    • 0035988996 scopus 로고    scopus 로고
    • Expression of human factor VIII by splicing between dimerized AAV vectors
    • Chao H et al. Expression of human factor VIII by splicing between dimerized AAV vectors. Mol Ther 2002; 5: 716-722.
    • (2002) Mol. Ther. , vol.5 , pp. 716-722
    • Chao, H.1
  • 16
    • 0036377825 scopus 로고    scopus 로고
    • Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector
    • Andrews JL et al. Evaluation of the duration of human factor VIII expression in nonhuman primates after systemic delivery of an adenoviral vector. Hum Gene Ther 2002; 13: 1331-1336.
    • (2002) Hum. Gene Ther. , vol.13 , pp. 1331-1336
    • Andrews, J.L.1
  • 17
    • 0033621126 scopus 로고    scopus 로고
    • Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice
    • VandenDriessche T et al. 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 USA 1999; 96: 10379-10384.
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 10379-10384
    • VandenDriessche, T.1
  • 18
    • 0034254320 scopus 로고    scopus 로고
    • Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver
    • Park F, Ohashi K, Kay MA. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral 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
  • 19
    • 0034960875 scopus 로고    scopus 로고
    • In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors
    • Stein CS et al. In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors. Mol Ther 2001; 3: 850-856.
    • (2001) Mol. Ther. , vol.3 , pp. 850-856
    • Stein, C.S.1
  • 20
    • 0036682975 scopus 로고    scopus 로고
    • Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo
    • VandenDriessche T et al. Lentiviral vectors containing the human immunodeficiency 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
  • 21
    • 0027283419 scopus 로고
    • Toward gene therapy for hemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice
    • Hoeben RC et al. Toward gene therapy for hemophilia A: long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice. Hum Gene Ther 1993; 4: 179-186.
    • (1993) Hum. Gene Ther. , vol.4 , pp. 179-186
    • Hoeben, R.C.1
  • 22
    • 0028842288 scopus 로고
    • Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice
    • Dwarki VJ et al. Gene therapy for hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 1995; 92: 1023-1027.
    • (1995) Proc. Natl. Acad. Sci. USA , vol.92 , pp. 1023-1027
    • Dwarki, V.J.1
  • 23
    • 0035822005 scopus 로고    scopus 로고
    • Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
    • Roth DA et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735-1742.
    • (2001) N. Engl. J. Med. , vol.344 , pp. 1735-1742
    • Roth, D.A.1
  • 24
    • 0032501988 scopus 로고    scopus 로고
    • Bone marrow stromal cells as targets for gene therapy of hemophilia A
    • Chuah MK et al. Bone marrow stromal cells as targets for gene therapy of hemophilia A. Hum Gene Ther 1998; 9: 353-365.
    • (1998) Hum. Gene Ther. , vol.9 , pp. 353-365
    • Chuah, M.K.1
  • 25
    • 0034689505 scopus 로고    scopus 로고
    • Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nomnyeloablated inummodeficient mice
    • Chuah MK et al. Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nomnyeloablated inummodeficient mice. Hum Gene Ther 2000; 11: 729-738.
    • (2000) Hum. Gene Ther. , vol.11 , pp. 729-738
    • Chuah, M.K.1
  • 26
    • 0026534020 scopus 로고
    • Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells
    • Hoeben RC et al. Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 1992; 67: 341-345.
    • (1992) Thromb. Haemost. , vol.67 , pp. 341-345
    • Hoeben, R.C.1
  • 27
    • 0032510756 scopus 로고    scopus 로고
    • Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A
    • 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 USA 1998; 95: 5734-5739.
    • (1998) Proc. Natl. Acad. Sci. USA , vol.95 , pp. 5734-5739
    • Evans, G.L.1    Morgan, R.A.2
  • 28
    • 0034839787 scopus 로고    scopus 로고
    • Tissue distribution of factor VIII gene expression in vivo - A closer look
    • Hollestelle MJ et al. Tissue distribution of factor VIII gene expression in vivo - a closer look. Thromb Haemost 2001; 86: 855-861.
    • (2001) Thromb. Haemost. , vol.86 , pp. 855-861
    • Hollestelle, M.J.1
  • 29
    • 0032578385 scopus 로고    scopus 로고
    • HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells
    • Uchida N et al. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells. Proc Natl Acad Sci USA 1998; 95: 11939-11944.
    • (1998) Proc. Natl. Acad. Sci. USA , vol.95 , pp. 11939-11944
    • Uchida, N.1
  • 30
    • 0033613827 scopus 로고    scopus 로고
    • Transduction of human CD34+ cells that mediate long-term engraftment of NCOD/SCID mice by HIV vectors
    • Miyoshi H et al. Transduction of human CD34+ cells that mediate long-term engraftment of NCOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
    • (1999) Science , vol.283 , pp. 682-686
    • Miyoshi, H.1
  • 31
    • 0033542427 scopus 로고    scopus 로고
    • Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors
    • Evans JT, Kelly PF, O'Neill E, Garcia JV. Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors. Hum Gene Ther 1999; 10: 1479-1489.
    • (1999) Hum. Gene Ther. , vol.10 , pp. 1479-1489
    • Evans, J.T.1    Kelly, P.F.2    O'Neill, E.3    Garcia, J.V.4
  • 32
    • 13044309472 scopus 로고    scopus 로고
    • Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors
    • Case SS et al. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci USA 1999; 96: 2988-2993.
    • (1999) Proc. Natl. Acad. Sci. USA , vol.96 , pp. 2988-2993
    • Case, S.S.1
  • 33
    • 0037079742 scopus 로고    scopus 로고
    • + NOD/SCID-repopulating cells
    • + NOD/SCID-repopulating cells. Blood 2002; 99: 709-712.
    • (2002) Blood , vol.99 , pp. 709-712
    • Scherr, M.1
  • 34
    • 0034646513 scopus 로고    scopus 로고
    • HIV-1 genome nuclear import is mediated by a central DNA flap
    • Zennou V et al. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 2000; 101: 173-185.
    • (2000) Cell , vol.101 , pp. 173-185
    • Zennou, V.1
  • 35
    • 0034672356 scopus 로고    scopus 로고
    • The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells
    • Sirven A et al. The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 2000; 96: 4103-4110.
    • (2000) Blood , vol.96 , pp. 4103-4110
    • Sirven, A.1
  • 36
    • 0035013429 scopus 로고    scopus 로고
    • The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain
    • Zennou V et al. The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain. Nat Biotechnol 2001; 19: 446-450.
    • (2001) Nat. Biotechnol. , vol.19 , pp. 446-450
    • Zennou, V.1
  • 37
    • 0034849315 scopus 로고    scopus 로고
    • Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo
    • Park F, Kay MA. Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol Ther 2001; 4: 164-173.
    • (2001) Mol. Ther. , vol.4 , pp. 164-173
    • Park, F.1    Kay, M.A.2
  • 38
    • 0034663381 scopus 로고    scopus 로고
    • Lentiviral-mediated gene transfer into human lymphocytes: Role of HIV-1 accessory proteins
    • Chinnasamy D et al. Lentiviral-mediated gene transfer into human lymphocytes: role of HIV-1 accessory proteins. Blood 2000; 96: 1309-1316.
    • (2000) Blood , vol.96 , pp. 1309-1316
    • Chinnasamy, D.1
  • 39
    • 0028201732 scopus 로고
    • Tolerance, danger, and the extended family
    • Matzinger P. Tolerance, danger, and the extended family. Annu Rev Immunol 1994; 12: 991-1045.
    • (1994) Annu. Rev. Immunol. , vol.12 , pp. 991-1045
    • Matzinger, P.1
  • 40
    • 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
  • 41
    • 0034199170 scopus 로고    scopus 로고
    • Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model
    • Heim DA et al. Introduction of a xenogeneic gene via hematopoietic stem cells leads to specific tolerance in a rhesus monkey model. Mol Ther 2000; 1: 533-544.
    • (2000) Mol. Ther. , vol.1 , pp. 533-544
    • Heim, D.A.1
  • 42
    • 0034809177 scopus 로고    scopus 로고
    • In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting
    • Kang E et al. In vivo persistence of retrovirally transduced murine long-term repopulating cells is not limited by expression of foreign gene products in the fully or minimally myeloablated setting. Hum Gene Ther 2001; 12: 1663-1672.
    • (2001) Hum. Gene Ther. , vol.12 , pp. 1663-1672
    • Kang, E.1
  • 43
    • 0031884476 scopus 로고    scopus 로고
    • The canine factor VIII cDNA and 5′ flanking sequence
    • Cameron C et al. The canine factor VIII cDNA and 5′ flanking sequence. Thromb Haemost 1998; 79: 317-322.
    • (1998) Thromb. Haemost. , vol.79 , pp. 317-322
    • Cameron, C.1
  • 44
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1
  • 45
    • 0029993858 scopus 로고    scopus 로고
    • Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector
    • Naldini L et al. Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc Natl Acad Sci USA 1996; 93: 11382-11388.
    • (1996) Proc. Natl. Acad. Sci. USA , vol.93 , pp. 11382-11388
    • Naldini, L.1
  • 46
    • 0030819379 scopus 로고    scopus 로고
    • Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
    • Zufferey R et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
    • (1997) Nat. Biotechnol. , vol.15 , pp. 871-875
    • Zufferey, R.1
  • 47
    • 0034857455 scopus 로고    scopus 로고
    • Quantitative determination of lentiviral vector particle numbers by real-time PCR
    • Scherr M et al. Quantitative determination of lentiviral vector particle numbers by real-time PCR. Biotechniques 2001; 31: 520-526.
    • (2001) Biotechniques , vol.31 , pp. 520-526
    • Scherr, M.1
  • 48
    • 0032845686 scopus 로고    scopus 로고
    • Monitoring of BCR-ABL expression using real-time RT-PCR in CML after bone marrow or peripheral blood stem cell transplantation
    • Eder M et al. Monitoring of BCR-ABL expression using real-time RT-PCR in CML after bone marrow or peripheral blood stem cell transplantation. Leukemia 1999; 13: 1383-1389.
    • (1999) Leukemia , vol.13 , pp. 1383-1389
    • Eder, M.1
  • 49
    • 0342470476 scopus 로고    scopus 로고
    • Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice
    • Schiedlmeier B et al. Quantitative assessment of retroviral transfer of the human multidrug resistance 1 gene to human mobilized peripheral blood progenitor cells engrafted in nonobese diabetic/severe combined immunodeficient mice. Blood 2000; 95: 1237-1248.
    • (2000) Blood , vol.95 , pp. 1237-1248
    • Schiedlmeier, B.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.