메뉴 건너뛰기




Volumn 5, Issue 3, 1999, Pages 160-168

Efficient adenoviral vector transduction and expression of functional human factor VIII in cultured primary human hepatocytes

Author keywords

Adenovirus; Factor VIII; Gene therapy; Hepatocytes; Primary cells

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; RNA;

EID: 0032796661     PISSN: 13518216     EISSN: None     Source Type: Journal    
DOI: 10.1046/j.1365-2516.1999.00304.x     Document Type: Article
Times cited : (3)

References (41)
  • 1
    • 0031828432 scopus 로고    scopus 로고
    • Haemophilia A gene therapy
    • Connelly S, Kaleko M. Haemophilia A gene therapy. Haemophilila 1998; 4: 380-8.
    • (1998) Haemophilila , vol.4 , pp. 380-388
    • Connelly, S.1    Kaleko, M.2
  • 2
    • 0030797290 scopus 로고    scopus 로고
    • Gene therapy for hemophilia A
    • Connelly S, Kaleko M. Gene therapy for hemophilia A. Thromb Haemost 1997; 78: 31-6.
    • (1997) Thromb Haemost , vol.78 , pp. 31-36
    • Connelly, S.1    Kaleko, M.2
  • 3
    • 0030700693 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Smith TAG. Gene therapy for haemophilia. Exp Opin Invest Drugs 1997; 6: 1685-90.
    • (1997) Exp Opin Invest Drugs , vol.6 , pp. 1685-1690
    • Smith, T.A.G.1
  • 4
    • 0022909287 scopus 로고
    • Construction and characterization of an active factor VIII variant lacking the central one-third of the molecule
    • Eaton DL, Wood WI, Eaton D, et al. Construction and characterization of an active factor VIII variant lacking the central one-third of the molecule. Biochem 1986; 25: 1-2.
    • (1986) Biochem , vol.25 , pp. 1-2
    • Eaton, D.L.1    Wood, W.I.2    Eaton, D.3
  • 6
    • 0027319588 scopus 로고
    • Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
    • Pittman DD, Alderman EM, Tomkinson KN, Wang JH, Giles AR, Kaufman RJ. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 1993; 81: 2925-35.
    • (1993) Blood , vol.81 , pp. 2925-2935
    • Pittman, D.D.1    Alderman, E.M.2    Tomkinson, K.N.3    Wang, J.H.4    Giles, A.R.5    Kaufman, R.J.6
  • 7
    • 0029664338 scopus 로고    scopus 로고
    • Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy
    • Gorziglia MI, Kadan MJ, Yei S, et al. Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy. J Virol 1996; 6: 4173-8.
    • (1996) J Virol , vol.6 , pp. 4173-4178
    • Gorziglia, M.I.1    Kadan, M.J.2    Yei, S.3
  • 9
    • 0028842288 scopus 로고
    • Gene therapy for hemophilia A. Production of therapeutic levels of human factor VIII in vivo in mice
    • Dwarki VJ, Belloni P, Nijjar T, 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: 1023-7.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 1023-1027
    • Dwarki, V.J.1    Belloni, P.2    Nijjar, T.3
  • 10
    • 0027237331 scopus 로고
    • Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production
    • Lynch CM, Israel DI, Kaufman RJ, Miller AD. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther 1993; 4: 259-72.
    • (1993) Hum Gene Ther , vol.4 , pp. 259-272
    • Lynch, C.M.1    Israel, D.I.2    Kaufman, R.J.3    Miller, A.D.4
  • 11
    • 0028805939 scopus 로고
    • Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A
    • Chuah MKL, Vandendriessche T, Morgan RA. Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A. Hum Gene Ther 1995; 6: 1363-77.
    • (1995) Hum Gene Ther , vol.6 , pp. 1363-1377
    • Chuah, M.K.L.1    Vandendriessche, T.2    Morgan, R.A.3
  • 12
    • 0025306056 scopus 로고
    • Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer
    • Hoeben RC, van der Jagt RC, Schoute F, et al. Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer. J Biol Chem 1990; 265: 7318-23.
    • (1990) J Biol Chem , vol.265 , pp. 7318-7323
    • Hoeben, R.C.1    Van Der Jagt, R.C.2    Schoute, F.3
  • 13
    • 0026534020 scopus 로고
    • Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells
    • Hoeben RC, Einerhand MP, Briet E, Van Ormondt H, Valerio D, Van Der Eb AJ. Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 1992; 67: 341-5.
    • (1992) Thromb Haemost , vol.67 , pp. 341-345
    • Hoeben, R.C.1    Einerhand, M.P.2    Briet, E.3    Van Ormondt, H.4    Valerio, D.5    Van Der Eb, A.J.6
  • 14
    • 0027283419 scopus 로고
    • Toward gene therapy for hemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice
    • Hoeben RC, Fallaux FJ, Van Tilburg NH, 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: 179-86.
    • (1993) Hum Gene Ther , vol.4 , pp. 179-186
    • Hoeben, R.C.1    Fallaux, F.J.2    Van Tilburg, N.H.3
  • 15
    • 0025279872 scopus 로고
    • Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection
    • Miller DG, Adam MA, Miller AD. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 1990; 10: 4239-42.
    • (1990) Mol Cell Biol , vol.10 , pp. 4239-4242
    • Miller, D.G.1    Adam, M.A.2    Miller, A.D.3
  • 16
    • 0026762462 scopus 로고
    • Biological regulation of factor VIII activity
    • Kaufman RJ. Biological regulation of factor VIII activity. Annu Rev Med 1992; 43: 325-39.
    • (1992) Annu Rev Med , vol.43 , pp. 325-339
    • Kaufman, R.J.1
  • 18
    • 0027521773 scopus 로고
    • Adeno-virus-mediated expression of therapeutic plasma levels of human factor IX in mice
    • Smith TAG, Mehaffey MG, Kayda DB et al. Adeno-virus-mediated expression of therapeutic plasma levels of human factor IX in mice. Nat Genet 1993; 5: 397-402.
    • (1993) Nat Genet , vol.5 , pp. 397-402
    • Smith, T.A.G.1    Mehaffey, M.G.2    Kayda, D.B.3
  • 19
    • 0028991820 scopus 로고
    • In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice
    • Connelly S, Smith TAG, Dhir G, et al. In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice. Hum Gene Ther 1995; 6: 185-93.
    • (1995) Hum Gene Ther , vol.6 , pp. 185-193
    • Connelly, S.1    Smith, T.A.G.2    Dhir, G.3
  • 20
    • 0029943193 scopus 로고    scopus 로고
    • Sustained expression of therapeutic levels of human factor VIII in mice
    • Connelly S, Gardner JM, Lyons RM, McClelland A, Kaleko M. Sustained expression of therapeutic levels of human factor VIII in mice. Blood 1996; 87: 4671-7.
    • (1996) Blood , vol.87 , pp. 4671-4677
    • Connelly, S.1    Gardner, J.M.2    Lyons, R.M.3    McClelland, A.4    Kaleko, M.5
  • 21
    • 0030069229 scopus 로고    scopus 로고
    • High level tissue-specific expression of functional human FVIII in mice
    • Connelly S, Gardner JM, McClelland A, Kaleko M. High level tissue-specific expression of functional human FVIII in mice. Hum Gene Ther 1996; 7: 183-95.
    • (1996) Hum Gene Ther , vol.7 , pp. 183-195
    • Connelly, S.1    Gardner, J.M.2    McClelland, A.3    Kaleko, M.4
  • 22
    • 0029850534 scopus 로고    scopus 로고
    • Complete short-term correction of canine hemophilia A by in vivo gene therapy
    • Connelly S, Mount J, Mauser A, et al. Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood 1996; 88: 3846-53.
    • (1996) Blood , vol.88 , pp. 3846-3853
    • Connelly, S.1    Mount, J.2    Mauser, A.3
  • 23
    • 0032080179 scopus 로고    scopus 로고
    • Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy
    • Connelly S, Andrews J, Gallo AM, et al. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood 1998; 91: 3273-81.
    • (1998) Blood , vol.91 , pp. 3273-3281
    • Connelly, S.1    Andrews, J.2    Gallo, A.M.3
  • 24
    • 0021715168 scopus 로고
    • Expression of active human factor VIII from recombinant DNA cloned
    • Wood WI, Capon DJ, Simonsen CC, et al. Expression of active human factor VIII from recombinant DNA cloned. Nature 1984; 312: 330-7.
    • (1984) Nature , vol.312 , pp. 330-337
    • Wood, W.I.1    Capon, D.J.2    Simonsen, C.C.3
  • 25
    • 0023266138 scopus 로고
    • Cell-specific enhancer activity in a far upstream region of the human alphafetoprotein gene
    • Watanabe K, Saito A, Tamaoki T. Cell-specific enhancer activity in a far upstream region of the human alphafetoprotein gene. J Biol Chem 1987; 262: 4812-8.
    • (1987) J Biol Chem , vol.262 , pp. 4812-4818
    • Watanabe, K.1    Saito, A.2    Tamaoki, T.3
  • 26
    • 0029836780 scopus 로고    scopus 로고
    • Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy
    • Mittereder N, March KL, Trapnell BC. Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy. J Virol 1996; 70: 7498-509.
    • (1996) J Virol , vol.70 , pp. 7498-7509
    • Mittereder, N.1    March, K.L.2    Trapnell, B.C.3
  • 28
    • 0021715169 scopus 로고
    • Molecular cloning of a cDNA encoding antihemophiliac factor
    • Toole JJ, Knopf JL, Wozney JM, et al. Molecular cloning of a cDNA encoding antihemophiliac factor. Nature 1984; 312: 342-6.
    • (1984) Nature , vol.312 , pp. 342-346
    • Toole, J.J.1    Knopf, J.L.2    Wozney, J.M.3
  • 29
    • 0030777488 scopus 로고    scopus 로고
    • Adenoviral vector transduction of quiescent primary human hepatocytes
    • Yang L, Skotzko M, Stain S, et al. Adenoviral vector transduction of quiescent primary human hepatocytes. Int J Ped Hemotol/Oncol 1997; 4: 307-15.
    • (1997) Int J Ped Hemotol/Oncol , vol.4 , pp. 307-315
    • Yang, L.1    Skotzko, M.2    Stain, S.3
  • 30
    • 0021659730 scopus 로고
    • Transcriptional and post-transcriptional control of specific messenger RNAs in adult and embryonic liver
    • Powell DJ, Friedman JM, Oulette AJ, Krauter KS, Darnell JEJ. Transcriptional and post-transcriptional control of specific messenger RNAs in adult and embryonic liver. J Mol Biol 1984; 179: 21-35.
    • (1984) J Mol Biol , vol.179 , pp. 21-35
    • Powell, D.J.1    Friedman, J.M.2    Oulette, A.J.3    Krauter, K.S.4    Darnell, J.E.J.5
  • 31
    • 0019332750 scopus 로고
    • Human hepatocellular carcinoma cell lines secrete the major plasma proteins and hepatitis B surface antigen
    • Knowles BB, Howe CC, Aden DP. Human hepatocellular carcinoma cell lines secrete the major plasma proteins and hepatitis B surface antigen. Sci 1980; 209: 497-9.
    • (1980) Sci , vol.209 , pp. 497-499
    • Knowles, B.B.1    Howe, C.C.2    Aden, D.P.3
  • 32
    • 0018747680 scopus 로고
    • Changes in expression of albumin and alphafetoprotein genes during rat liver development and neoplasia
    • Sala-Trepat JM, Dever J, Sargent TD, Thomas K, Sell S, Bonner J. Changes in expression of albumin and alphafetoprotein genes during rat liver development and neoplasia. Biochem 1979; 18: 2167-78.
    • (1979) Biochem , vol.18 , pp. 2167-2178
    • Sala-Trepat, J.M.1    Dever, J.2    Sargent, T.D.3    Thomas, K.4    Sell, S.5    Bonner, J.6
  • 33
    • 0016137449 scopus 로고
    • Alpha-fetoprotein as a maker of embryo-specific differentiations in normal and tumor tissues
    • Abelev GI. Alpha-fetoprotein as a maker of embryo-specific differentiations in normal and tumor tissues. Transplant Rev 1974; 20: 3-37.
    • (1974) Transplant Rev , vol.20 , pp. 3-37
    • Abelev, G.I.1
  • 34
    • 0017180441 scopus 로고
    • Expression of an oncodevelopmental gene product (alpha-fetoprotein) during fetal development and adult oncogenesis
    • Sell S, Becker FF, Leffert HL, Watabe L. Expression of an oncodevelopmental gene product (alpha-fetoprotein) during fetal development and adult oncogenesis. Cancer Res 1976; 36: 4239-49.
    • (1976) Cancer Res , vol.36 , pp. 4239-4249
    • Sell, S.1    Becker, F.F.2    Leffert, H.L.3    Watabe, L.4
  • 35
    • 0026053032 scopus 로고
    • Interleukin 6 enhances a cellular activity that functionally substitutes for E1A protein in transactivation
    • Spergel JM, Chen-Kiang S. Interleukin 6 enhances a cellular activity that functionally substitutes for E1A protein in transactivation. Proc Natl Acad Sci USA 1991; 88: 6472-6.
    • (1991) Proc Natl Acad Sci USA , vol.88 , pp. 6472-6476
    • Spergel, J.M.1    Chen-Kiang, S.2
  • 36
    • 0026308289 scopus 로고
    • Hepatocellular transplantation in acute hepatic function and targeting genetic markers to hepatic cells
    • Ledley FD. Hepatocellular transplantation in acute hepatic function and targeting genetic markers to hepatic cells. Hum Gene Ther 1991; 2: 331-58.
    • (1991) Hum Gene Ther , vol.2 , pp. 331-358
    • Ledley, F.D.1
  • 37
    • 0023771077 scopus 로고
    • A new recombinant procoagulant protein derived from the cDNA encoding human factor VIII
    • Meulien P, Faure T, Mischler F, et al. A new recombinant procoagulant protein derived from the cDNA encoding human factor VIII. Protein Eng 1988; 2: 301-6.
    • (1988) Protein Eng , vol.2 , pp. 301-306
    • Meulien, P.1    Faure, T.2    Mischler, F.3
  • 38
    • 0023597393 scopus 로고
    • The relationship of N-linked glycosylation and heavy chain-binding protein association with the secretion of glycoproteins
    • Dorner AJ, Bole DG, Kaufman RJ. The relationship of N-linked glycosylation and heavy chain-binding protein association with the secretion of glycoproteins. J Cell Biol 1987; 105: 2665-74.
    • (1987) J Cell Biol , vol.105 , pp. 2665-2674
    • Dorner, A.J.1    Bole, D.G.2    Kaufman, R.J.3
  • 39
    • 0032007271 scopus 로고    scopus 로고
    • Intracellular trafficking of factor VIII to von Willebrand factor storage granules
    • Rosenberg JB, Foster PA, Kaufman RJ, et al. Intracellular trafficking of factor VIII to von Willebrand factor storage granules. J Clin Invest 1998; 101: 613-24.
    • (1998) J Clin Invest , vol.101 , pp. 613-624
    • Rosenberg, J.B.1    Foster, P.A.2    Kaufman, R.J.3
  • 40
    • 0024393778 scopus 로고
    • Peptide binding and release by proteins implicated as catalysts of protein assembly
    • Flynn GC, Chappell TG, Rothman JE. Peptide binding and release by proteins implicated as catalysts of protein assembly. Sci 1989; 245: 385-90.
    • (1989) Sci , vol.245 , pp. 385-390
    • Flynn, G.C.1    Chappell, T.G.2    Rothman, J.E.3
  • 41
    • 0030875708 scopus 로고    scopus 로고
    • The use of cultured hepatocytes to investigate the mechanisms of drug hepatotoxicity
    • Castell JV, Gomez-Lechon MJ, Ponsoda X, Bort R. The use of cultured hepatocytes to investigate the mechanisms of drug hepatotoxicity. Cell Biol Toxicol 1997; 13: 331-8
    • (1997) Cell Biol Toxicol , vol.13 , pp. 331-338
    • Castell, J.V.1    Gomez-Lechon, M.J.2    Ponsoda, X.3    Bort, R.4


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.