메뉴 건너뛰기




Volumn 10, Issue 1, 1999, Pages 61-76

Bone marrow stromal cell-mediated gene therapy for hemophilia A: In vitro expression of human factor VIII with high biological activity requires the inclusion of the proteolytic site at amino acid 1648

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; PLASMID VECTOR; THROMBIN;

EID: 0032958003     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950019192     Document Type: Article
Times cited : (28)

References (51)
  • 1
    • 0028280446 scopus 로고
    • Design and operation of a recombinant mammalian cell manufacturing process for rFVIII
    • ADAMSON, R. (1994). Design and operation of a recombinant mammalian cell manufacturing process for rFVIII. Ann. Hematol. 68, S9-S14.
    • (1994) Ann. Hematol. , vol.68
    • Adamson, R.1
  • 2
    • 0030000014 scopus 로고    scopus 로고
    • Prophylaxis and continuous infusion for hemophilia: Can we afford it?
    • ALEDORT, L.M., and BOHN, R.L. (1996). Prophylaxis and continuous infusion for hemophilia: Can we afford it? Blood Coagul. Fibrinol. 7(Suppl. 1), S35-S37.
    • (1996) Blood Coagul. Fibrinol. , vol.7 , Issue.1 SUPPL.
    • Aledort, L.M.1    Bohn, R.L.2
  • 4
    • 0030855794 scopus 로고    scopus 로고
    • Second generation, B-domain deleted recombinant factor VIII
    • BERNTORP, E. (1997). Second generation, B-domain deleted recombinant factor VIII. Thromb. Haemost. 78, 256-260.
    • (1997) Thromb. Haemost. , vol.78 , pp. 256-260
    • Berntorp, E.1
  • 5
    • 0025784158 scopus 로고
    • Structural and functional characterization of factor VIII-delta II, a new recombinant factor VIII lacking most of the B-domain
    • BIHOREAU, N., PAOLANTONACCI, P., BARDELLE, C., FONTAINE-AUPART, M.P., KRISHNAN, S., YON, J., and ROMET-LEMONNE, J.L. (1991). Structural and functional characterization of factor VIII-delta II, a new recombinant factor VIII lacking most of the B-domain. Biochem. J. 277, 23-31.
    • (1991) Biochem. J. , vol.277 , pp. 23-31
    • Bihoreau, N.1    Paolantonacci, P.2    Bardelle, C.3    Fontaine-Aupart, M.P.4    Krishnan, S.5    Yon, J.6    Romet-Lemonne, J.L.7
  • 6
    • 0029803652 scopus 로고    scopus 로고
    • Gene therapy to human diseases: Ex vivo and in vivo studies
    • BOULIKAS, T. (1996). Gene therapy to human diseases: Ex vivo and in vivo studies. Int. J. Oncol. 9, 1239-1251. [Review]
    • (1996) Int. J. Oncol. , vol.9 , pp. 1239-1251
    • Boulikas, T.1
  • 7
    • 0003135354 scopus 로고
    • Clinical manifestations and therapy of inherited coagulation factor deficiencies
    • R.W. Colman, J. Hirsh, V.J. Marder, and E.W. Salzman, eds. (Lippincott, Philadelphia, PA)
    • BRETTLER, D.B., and LEVINE, P.H. (1994). Clinical manifestations and therapy of inherited coagulation factor deficiencies. In Hemostasis and Thrombosis. Basic Principles and Clinical Practice, 3rd Ed. R.W. Colman, J. Hirsh, V.J. Marder, and E.W. Salzman, eds. (Lippincott, Philadelphia, PA) pp. 169-183.
    • (1994) Hemostasis and Thrombosis. Basic Principles and Clinical Practice, 3rd Ed. , pp. 169-183
    • Brettler, D.B.1    Levine, P.H.2
  • 10
    • 0028805939 scopus 로고
    • Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A
    • CHUAH, M.K., VANDENDRIESSCHE, T., and MORGAN, R.A. (1995). Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A. Hum. Gene Ther. 6, 1363-1377.
    • (1995) Hum. Gene Ther. , vol.6 , pp. 1363-1377
    • Chuah, M.K.1    Vandendriessche, T.2    Morgan, R.A.3
  • 12
    • 0030797290 scopus 로고    scopus 로고
    • Gene therapy for hemophilia A
    • CONNELLY, S., and KALEKO, M. (1997). Gene therapy for hemophilia A. Thromb. Haemost. 78, 31-36.
    • (1997) Thromb. Haemost. , vol.78 , pp. 31-36
    • Connelly, S.1    Kaleko, M.2
  • 13
    • 0031435917 scopus 로고    scopus 로고
    • On the mechanism of DNA transfection: Efficient gene transfer without viruses
    • COONROD, A., LI, F.-Q., and HORWITZ, M. (1997). On the mechanism of DNA transfection: Efficient gene transfer without viruses. Gene Ther. 4, 1313-1321.
    • (1997) Gene Ther. , vol.4 , pp. 1313-1321
    • Coonrod, A.1    Li, F.-Q.2    Horwitz, M.3
  • 14
    • 0027538181 scopus 로고
    • Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity
    • DRANOFF, G., JAFFEE, E., LAZENBY, A., GOLUMBEK, P., LEVITSKY, H., BROSE, K., JACKSON, V., HAMADA, H., PARDOLL, D., and MULLIGAN, R.C. (1993). Vaccination with irradiated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulates potent, specific, and long-lasting anti-tumor immunity. Proc. Natl. Acad. Sci. U.S.A. 90, 3539-3543.
    • (1993) Proc. Natl. Acad. Sci. U.S.A. , vol.90 , pp. 3539-3543
    • Dranoff, G.1    Jaffee, E.2    Lazenby, A.3    Golumbek, P.4    Levitsky, H.5    Brose, K.6    Jackson, V.7    Hamada, H.8    Pardoll, D.9    Mulligan, R.C.10
  • 16
    • 0022454539 scopus 로고
    • Proteolytic processing of human factor VIII. Correlation of specific cleavages by thrombin, factor Xa, and activated protein C with activation and inactivation of factor VIII coagulant activity
    • EATON, D., RODRIGUEZ, H., and VEHAR, G.A. (1986a). Proteolytic processing of human factor VIII. Correlation of specific cleavages by thrombin, factor Xa, and activated protein C with activation and inactivation of factor VIII coagulant activity. Biochemistry 25, 505-512.
    • (1986) Biochemistry , vol.25 , pp. 505-512
    • Eaton, D.1    Rodriguez, H.2    Vehar, G.A.3
  • 19
    • 0029119078 scopus 로고
    • State and prospects of gene therapy for the hemophilias
    • FALLAUX, F.J., HOEBEN, R.C., and BRIET, E. (1995). State and prospects of gene therapy for the hemophilias. Thromb. Haemost. 74, 266-273.
    • (1995) Thromb. Haemost. , vol.74 , pp. 266-273
    • Fallaux, F.J.1    Hoeben, R.C.2    Briet, E.3
  • 20
    • 0000865045 scopus 로고    scopus 로고
    • Methods for the use of stromal cells for therapeutic gene therapy
    • P. Robbins, ed. (Humana Press, Totowa, NJ)
    • GREENBERGER, J.S. (1996). Methods for the use of stromal cells for therapeutic gene therapy. In Methods in Molecular Medicine, Gene Therapy Protocols. P. Robbins, ed. (Humana Press, Totowa, NJ) pp. 375-390.
    • (1996) Methods in Molecular Medicine, Gene Therapy Protocols , pp. 375-390
    • Greenberger, J.S.1
  • 21
    • 0028136218 scopus 로고
    • The immediate downstream codon strongly influences the efficiency of utilization of eukaryotic translation initiation codons
    • GRÜNERT, S., and JACKSON, R.J. (1994). The immediate downstream codon strongly influences the efficiency of utilization of eukaryotic translation initiation codons. EMBO J. 13, 3618-3630.
    • (1994) EMBO J. , vol.13 , pp. 3618-3630
    • Grünert, S.1    Jackson, R.J.2
  • 22
    • 0013599178 scopus 로고    scopus 로고
    • Xenotransplantation of electrotransfected marrow stromal cells express human factor IX protein in SCID mice: A pre-clinical model for gene therapy
    • GUINN, B., WANG, X.-H., LARAYA, P., and KEATING, A. (1996). Xenotransplantation of electrotransfected marrow stromal cells express human factor IX protein in SCID mice: A pre-clinical model for gene therapy. Blood 88(10: Suppl. 1), 3921.
    • (1996) Blood , vol.88 , Issue.10 SUPPL. 1 , pp. 3921
    • Guinn, B.1    Wang, X.-H.2    Laraya, P.3    Keating, A.4
  • 24
    • 0029059149 scopus 로고
    • Why do so many haemophilia A patients develop an inhibitor?
    • HOYER, L.W. (1995). Why do so many haemophilia A patients develop an inhibitor? Br. J. Haematol. 90, 498-501.
    • (1995) Br. J. Haematol. , vol.90 , pp. 498-501
    • Hoyer, L.W.1
  • 26
    • 0029101389 scopus 로고
    • Homing and immunogenicity of murine stromal cells transfected with xenogeneic MHC class II genes
    • HUSS, R., SMITH, P.O., MYERSON, D.H., and DEEG, H.J. (1995). Homing and immunogenicity of murine stromal cells transfected with xenogeneic MHC class II genes. Cell Transplant. 4, 483-491.
    • (1995) Cell Transplant. , vol.4 , pp. 483-491
    • Huss, R.1    Smith, P.O.2    Myerson, D.H.3    Deeg, H.J.4
  • 27
    • 0025257348 scopus 로고
    • Retroviral-mediated transfer and amplification of a functional human factor VIII gene
    • ISRAEL, D.I., and KAUFMAN, R.J. (1990). Retroviral-mediated transfer and amplification of a functional human factor VIII gene. Blood 75, 1074-1080.
    • (1990) Blood , vol.75 , pp. 1074-1080
    • Israel, D.I.1    Kaufman, R.J.2
  • 28
    • 0026762462 scopus 로고
    • Biological regulation of factor VIII activity
    • KAUFMAN, R.J. (1992). Biological regulation of factor VIII activity. Annu. Rev. Med. 43, 325-339.
    • (1992) Annu. Rev. Med. , vol.43 , pp. 325-339
    • Kaufman, R.J.1
  • 29
    • 0023918719 scopus 로고
    • Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells
    • KAUFMAN, R.J., WASLEY, L.C., and DORNER, A.J. (1988). Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J. Biol. Chem. 263, 6352-6362.
    • (1988) J. Biol. Chem. , vol.263 , pp. 6352-6362
    • Kaufman, R.J.1    Wasley, L.C.2    Dorner, A.J.3
  • 30
    • 0024546509 scopus 로고
    • The scanning model for translation: An update
    • KOZAK, M. (1989). The scanning model for translation: An update. J. Cell Biol. 108, 229-241.
    • (1989) J. Cell Biol. , vol.108 , pp. 229-241
    • Kozak, M.1
  • 31
    • 0029965285 scopus 로고    scopus 로고
    • Increased levels of spliced RNA account for augmented expression from the MFG retroviral vector in hematopoietic cells
    • KRALL, W.J., SKELTON, D.C., YU, X.J., RIVIERE, I., LEHN, P., MULLIGAN, R.C., and KOHN, D.B. (1996). Increased levels of spliced RNA account for augmented expression from the MFG retroviral vector in hematopoietic cells. Gene Ther. 3, 37-48.
    • (1996) Gene Ther. , vol.3 , pp. 37-48
    • Krall, W.J.1    Skelton, D.C.2    Yu, X.J.3    Riviere, I.4    Lehn, P.5    Mulligan, R.C.6    Kohn, D.B.7
  • 33
    • 0029133142 scopus 로고
    • Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization
    • LIND, P., LARSSON, K., SPIRA, J., SYDOW-BACKMAN, M., ALMSTEDT, A., GRAY, E., and SANDBERG, H. (1995). Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization. Eur. J. Biochem. 232, 19-27.
    • (1995) Eur. J. Biochem. , vol.232 , pp. 19-27
    • Lind, P.1    Larsson, K.2    Spira, J.3    Sydow-Backman, M.4    Almstedt, A.5    Gray, E.6    Sandberg, H.7
  • 34
    • 0027237331 scopus 로고
    • Sequences in the coding region of cloning factor VIII act as dominant inhibitors of RNA accumulation and protein production
    • LYNCH, C.M., ISRAEL, D.I., KAUFMAN, R.J., and MILLER, A.D. (1993). Sequences in the coding region of cloning factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum. Gene Ther. 4, 259-272.
    • (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
  • 35
    • 0023871422 scopus 로고
    • A safe packaging line for gene transfer: Separating viral genes on two different plasmids
    • MARKOWITZ, D., GOFF, S., and BANK, A. (1988a). A safe packaging line for gene transfer: Separating viral genes on two different plasmids. J. Virol. 62, 1120-1124.
    • (1988) J. Virol. , vol.62 , pp. 1120-1124
    • Markowitz, D.1    Goff, S.2    Bank, A.3
  • 36
    • 0024239024 scopus 로고
    • Construction and use of a safe and efficient amphotropic packaging cell line
    • MARKOWITZ, D., GOFF, S., and BANK, A. (1988b). Construction and use of a safe and efficient amphotropic packaging cell line. Virology 167, 400-406.
    • (1988) Virology , vol.167 , pp. 400-406
    • Markowitz, D.1    Goff, S.2    Bank, A.3
  • 38
    • 0028325345 scopus 로고
    • Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: Analysis of gene transduction of long-lived progenitors
    • NOLTA, J.A., HANLEY, M.B., and KOHN, D.B. (1994). Sustained human hematopoiesis in immunodeficient mice by cotransplantation of marrow stroma expressing human interleukin-3: Analysis of gene transduction of long-lived progenitors. Blood 83, 3041-3051.
    • (1994) Blood , vol.83 , pp. 3041-3051
    • Nolta, J.A.1    Hanley, M.B.2    Kohn, D.B.3
  • 41
    • 1842408993 scopus 로고
    • Proteolytic requirements for thrombin activation of anti-hemophilic factor (factor VIII)
    • PITTMAN, D.D., and KAUFMAN, R.J. (1988). Proteolytic requirements for thrombin activation of anti-hemophilic factor (factor VIII). Proc. Natl. Acad. Sci. U.S.A. 85, 2429-2433.
    • (1988) Proc. Natl. Acad. Sci. U.S.A. , vol.85 , pp. 2429-2433
    • Pittman, D.D.1    Kaufman, R.J.2
  • 42
    • 0027319588 scopus 로고
    • Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII
    • PITTMAN, D.D., ALDERMAN, E.M., TOMKINSON, K.N., WANG, J.H., GILES, A.R., and KAUFMAN, R.J. (1993). Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 81, 2925-2935.
    • (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
  • 43
    • 0030889354 scopus 로고    scopus 로고
    • Marrow stromal cells as stem cells for non-hematopoietic tissues
    • PROCKOP, D.J. (1997). Marrow stromal cells as stem cells for non-hematopoietic tissues. Science 276, 71-74.
    • (1997) Science , vol.276 , pp. 71-74
    • Prockop, D.J.1
  • 44
    • 0025829429 scopus 로고
    • Hemophilia treatment in historical perspective: A review of medical and social developments
    • ROSENDAAL, F.R., SMIT, C., and BRIET, E. (1991). Hemophilia treatment in historical perspective: A review of medical and social developments. Ann. Hematol. 62, 5-15.
    • (1991) Ann. Hematol. , vol.62 , pp. 5-15
    • Rosendaal, F.R.1    Smit, C.2    Briet, E.3
  • 45
    • 0023491379 scopus 로고
    • Stable expression of recombinant factor VIII molecules using a bovine papillomavirus vector
    • SARVER, N., RICCA, G.A., LINK, J., NATHAN, M.H., NEWMAN, J., and DROHAN, W.N. (1987). Stable expression of recombinant factor VIII molecules using a bovine papillomavirus vector. DNA 6, 553-564.
    • (1987) DNA , vol.6 , pp. 553-564
    • Sarver, N.1    Ricca, G.A.2    Link, J.3    Nathan, M.H.4    Newman, J.5    Drohan, W.N.6
  • 46
    • 0029144825 scopus 로고
    • Progress towards gene therapy for the hemophilias
    • THOMPSON, A.R. (1995). Progress towards gene therapy for the hemophilias. Thromb. Haemost. 74, 45-51.
    • (1995) Thromb. Haemost. , vol.74 , pp. 45-51
    • Thompson, A.R.1
  • 48
    • 0022760260 scopus 로고
    • A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity
    • TOOLE, J.J., PITTMAN, D.D., ORR, E.C., MURTHA, P., WASLEY, L.C., and KAUFMAN, R.J. (1986). A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc. Natl. Acad. Sci. U.S.A. 83, 5939-5942.
    • (1986) Proc. Natl. Acad. Sci. U.S.A. , vol.83 , pp. 5939-5942
    • Toole, J.J.1    Pittman, D.D.2    Orr, E.C.3    Murtha, P.4    Wasley, L.C.5    Kaufman, R.J.6
  • 51
    • 0031300389 scopus 로고    scopus 로고
    • Gene therapy for the hemophilias
    • WALTER, J., and HIGH, K.A. (1997). Gene therapy for the hemophilias. Adv. Vet. Med. 40, 119-134.
    • (1997) Adv. Vet. Med. , vol.40 , pp. 119-134
    • Walter, J.1    High, K.A.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.