메뉴 건너뛰기




Volumn 62, Issue 12, 2010, Pages 1196-1203

Platelets as delivery systems for disease treatments

Author keywords

Gene therapy; Hemostasis; Platelet; Platelet specific promoter

Indexed keywords

BIOLOGICAL MOLECULE; BIOLOGICALLY ACTIVE MOLECULES; BLEEDING DISORDER; BLOOD CELLS; DELIVERY SYSTEMS; DISEASE TREATMENT; GENETIC THERAPY; HEMOPHILIA A; HEMOSTASIS; LOGICAL APPROACHES; PATHOPHYSIOLOGIC;

EID: 78649321428     PISSN: 0169409X     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.addr.2010.06.007     Document Type: Review
Times cited : (48)

References (148)
  • 1
    • 65849360502 scopus 로고    scopus 로고
    • Platelet alpha-granules: basic biology and clinical correlates
    • Blair P., Flaumenhaft R. Platelet alpha-granules: basic biology and clinical correlates. Blood Rev. 2009, 23:177-189.
    • (2009) Blood Rev. , vol.23 , pp. 177-189
    • Blair, P.1    Flaumenhaft, R.2
  • 2
    • 0014313727 scopus 로고
    • Polyploidy and maturation of rat megakaryocytes
    • Odell T.T., Jackson C.W. Polyploidy and maturation of rat megakaryocytes. Blood 1968, 32:102-110.
    • (1968) Blood , vol.32 , pp. 102-110
    • Odell, T.T.1    Jackson, C.W.2
  • 3
    • 0014806353 scopus 로고
    • Megakaryocytopoiesis in rats with special reference to polyploidy
    • Odell T.T., Jackson C.W., Friday T.J. Megakaryocytopoiesis in rats with special reference to polyploidy. Blood 1970, 35:775-782.
    • (1970) Blood , vol.35 , pp. 775-782
    • Odell, T.T.1    Jackson, C.W.2    Friday, T.J.3
  • 4
    • 0002170705 scopus 로고
    • Megakaryocytopoiesis in the rat
    • EBBE S., STOHLMAN F. Megakaryocytopoiesis in the rat. Blood 1965, 26:20-35.
    • (1965) Blood , vol.26 , pp. 20-35
    • Ebbe, S.1    Stohlman, F.2
  • 5
    • 0023583163 scopus 로고
    • Purification of human megakaryocytes by fluorescence-activated cell sorting
    • Tomer A., Harker L.A., Burstein S.A. Purification of human megakaryocytes by fluorescence-activated cell sorting. Blood 1987, 70:1735-1742.
    • (1987) Blood , vol.70 , pp. 1735-1742
    • Tomer, A.1    Harker, L.A.2    Burstein, S.A.3
  • 6
    • 0023888738 scopus 로고
    • Flow cytometric analysis of normal human megakaryocytes
    • Tomer A., Harker L.A., Burstein S.A. Flow cytometric analysis of normal human megakaryocytes. Blood 1988, 71:1244-1252.
    • (1988) Blood , vol.71 , pp. 1244-1252
    • Tomer, A.1    Harker, L.A.2    Burstein, S.A.3
  • 7
    • 0017151562 scopus 로고
    • Ultrastructure of human bone marrow cell maturation
    • Breton-Gorius J., Reyes F. Ultrastructure of human bone marrow cell maturation. Int. Rev. Cytol. 1976, 46:251-321.
    • (1976) Int. Rev. Cytol. , vol.46 , pp. 251-321
    • Breton-Gorius, J.1    Reyes, F.2
  • 8
    • 0016910150 scopus 로고
    • Biology of megakaryocytes
    • EBBE S. Biology of megakaryocytes. Prog. Hemost. Thromb. 1976, 3:211-229.
    • (1976) Prog. Hemost. Thromb. , vol.3 , pp. 211-229
    • Ebbe, S.1
  • 9
    • 0014407409 scopus 로고
    • Membrane surface specialization of blood platelet and megakaryocyte
    • Nakao K., Angrist A.A. Membrane surface specialization of blood platelet and megakaryocyte. Nature 1968, 217:960-961.
    • (1968) Nature , vol.217 , pp. 960-961
    • Nakao, K.1    Angrist, A.A.2
  • 10
    • 0033552631 scopus 로고    scopus 로고
    • Blood platelets are assembled principally at the ends of proplatelet processes produced by differentiated megakaryocytes
    • Italiano J.E., Lecine P., Shivdasani R.A., Hartwig J.H. Blood platelets are assembled principally at the ends of proplatelet processes produced by differentiated megakaryocytes. J. Cell Biol. 1999, 147:1299-1312.
    • (1999) J. Cell Biol. , vol.147 , pp. 1299-1312
    • Italiano, J.E.1    Lecine, P.2    Shivdasani, R.A.3    Hartwig, J.H.4
  • 11
    • 2142699977 scopus 로고    scopus 로고
    • Megakaryocytes and beyond: the birth of platelets
    • Italiano J.E., Shivdasani R.A. Megakaryocytes and beyond: the birth of platelets. J. Thromb. Haemost. 2003, 1:1174-1182.
    • (2003) J. Thromb. Haemost. , vol.1 , pp. 1174-1182
    • Italiano, J.E.1    Shivdasani, R.A.2
  • 12
    • 31044443970 scopus 로고    scopus 로고
    • The biogenesis of platelets from megakaryocyte proplatelets
    • Patel S.R., Hartwig J.H., Italiano J.E. The biogenesis of platelets from megakaryocyte proplatelets. J. Clin. Invest. 2005, 115:3348-3354.
    • (2005) J. Clin. Invest. , vol.115 , pp. 3348-3354
    • Patel, S.R.1    Hartwig, J.H.2    Italiano, J.E.3
  • 13
    • 0021259611 scopus 로고
    • Thrombocytopoiesis-analysis by membrane tracer and freeze-fracture studies on fresh human and cultured mouse megakaryocytes
    • Zucker-Franklin D., Petursson S. Thrombocytopoiesis-analysis by membrane tracer and freeze-fracture studies on fresh human and cultured mouse megakaryocytes. J. Cell Biol. 1984, 99:390-402.
    • (1984) J. Cell Biol. , vol.99 , pp. 390-402
    • Zucker-Franklin, D.1    Petursson, S.2
  • 14
    • 0027818891 scopus 로고
    • Proliferation, migration and platelet release by megakaryocytes in long-term bone marrow culture in collagen gel
    • Mori M., Tsuchiyama J., Okada S. Proliferation, migration and platelet release by megakaryocytes in long-term bone marrow culture in collagen gel. Cell Struct. Funct. 1993, 18:409-417.
    • (1993) Cell Struct. Funct. , vol.18 , pp. 409-417
    • Mori, M.1    Tsuchiyama, J.2    Okada, S.3
  • 15
    • 0023035166 scopus 로고
    • Adhesion, spreading and fragmentation of human megakaryocytes exposed to subendothelial extracellular matrix: a scanning electron microscopy study
    • Caine Y.G., Vlodavsky I., Hersh M., Polliack A., Gurfel D., Or R., Levine R.F., Eldor A. Adhesion, spreading and fragmentation of human megakaryocytes exposed to subendothelial extracellular matrix: a scanning electron microscopy study. Scan. Electron Microsc. 1986, 1087-1094.
    • (1986) Scan. Electron Microsc. , pp. 1087-1094
    • Caine, Y.G.1    Vlodavsky, I.2    Hersh, M.3    Polliack, A.4    Gurfel, D.5    Or, R.6    Levine, R.F.7    Eldor, A.8
  • 16
    • 0028964228 scopus 로고
    • The response of cord blood megakaryocyte progenitors to IL-3, IL-6 and aplastic canine serum varies with gestational age
    • Deutsch V.R., Olson T.A., Nagler A., Slavin S., Levine R.F., Eldor A. The response of cord blood megakaryocyte progenitors to IL-3, IL-6 and aplastic canine serum varies with gestational age. Br. J. Haematol. 1995, 89:8-16.
    • (1995) Br. J. Haematol. , vol.89 , pp. 8-16
    • Deutsch, V.R.1    Olson, T.A.2    Nagler, A.3    Slavin, S.4    Levine, R.F.5    Eldor, A.6
  • 17
    • 60049099167 scopus 로고    scopus 로고
    • Platelet production by megakaryocytes: protoplatelet theory justifies cytoplasmic fragmentation model
    • Kosaki G. Platelet production by megakaryocytes: protoplatelet theory justifies cytoplasmic fragmentation model. Int. J. Hematol. 2008, 88:255-267.
    • (2008) Int. J. Hematol. , vol.88 , pp. 255-267
    • Kosaki, G.1
  • 18
    • 20344393378 scopus 로고    scopus 로고
    • In vivo platelet production from mature megakaryocytes: does platelet release occur via proplatelets?
    • Kosaki G. In vivo platelet production from mature megakaryocytes: does platelet release occur via proplatelets?. Int. J. Hematol. 2005, 81:208-219.
    • (2005) Int. J. Hematol. , vol.81 , pp. 208-219
    • Kosaki, G.1
  • 19
    • 0014863254 scopus 로고
    • The submembranous fibrils of human blood platelets
    • Zucker-Franklin D. The submembranous fibrils of human blood platelets. J. Cell Biol. 1970, 47:293-299.
    • (1970) J. Cell Biol. , vol.47 , pp. 293-299
    • Zucker-Franklin, D.1
  • 20
    • 0028928884 scopus 로고
    • Platelets generated in vitro from proplatelet-displaying human megakaryocytes are functional
    • Choi E.S., Nichol J.L., Hokom M.M., Hornkohl A.C., Hunt P. Platelets generated in vitro from proplatelet-displaying human megakaryocytes are functional. Blood 1995, 85:402-413.
    • (1995) Blood , vol.85 , pp. 402-413
    • Choi, E.S.1    Nichol, J.L.2    Hokom, M.M.3    Hornkohl, A.C.4    Hunt, P.5
  • 21
    • 0031868521 scopus 로고    scopus 로고
    • Megakaryocyte differentiation events
    • Long M.W. Megakaryocyte differentiation events. Semin. Hematol. 1998, 35:192-199.
    • (1998) Semin. Hematol. , vol.35 , pp. 192-199
    • Long, M.W.1
  • 23
    • 0024530858 scopus 로고
    • Mechanisms of platelet production
    • Stenberg P.E., Levin J. Mechanisms of platelet production. Blood Cells 1989, 15:23-47.
    • (1989) Blood Cells , vol.15 , pp. 23-47
    • Stenberg, P.E.1    Levin, J.2
  • 24
    • 31044439372 scopus 로고    scopus 로고
    • The molecular mechanisms that control thrombopoiesis
    • Kaushansky K. The molecular mechanisms that control thrombopoiesis. J. Clin. Invest. 2005, 115:3339-3347.
    • (2005) J. Clin. Invest. , vol.115 , pp. 3339-3347
    • Kaushansky, K.1
  • 25
    • 33746508365 scopus 로고    scopus 로고
    • Megakaryocyte development and platelet production
    • Deutsch V.R., Tomer A. Megakaryocyte development and platelet production. Br. J. Haematol. 2006, 134:453-466.
    • (2006) Br. J. Haematol. , vol.134 , pp. 453-466
    • Deutsch, V.R.1    Tomer, A.2
  • 26
    • 67849111576 scopus 로고    scopus 로고
    • The evolution of platelet-directed pharmacotherapy
    • Becker R.C., Smyth S. The evolution of platelet-directed pharmacotherapy. J. Thromb. Haemost. 2009, 7(Suppl 1):266-271.
    • (2009) J. Thromb. Haemost. , vol.7 , Issue.SUPPL 1 , pp. 266-271
    • Becker, R.C.1    Smyth, S.2
  • 27
    • 0016744456 scopus 로고
    • Megakaryocyte quantifications in relation to thrombokinetics in primary thrombocythaemia and allied diseases
    • Branehog I., Ridell B., Swolin B., Weinfeld A. Megakaryocyte quantifications in relation to thrombokinetics in primary thrombocythaemia and allied diseases. Scand. J. Haematol. 1975, 15:321-332.
    • (1975) Scand. J. Haematol. , vol.15 , pp. 321-332
    • Branehog, I.1    Ridell, B.2    Swolin, B.3    Weinfeld, A.4
  • 28
    • 0016707740 scopus 로고
    • Cytochemical localization of lysosomal enzymes in rat megakaryocytes and platelets
    • Bentfeld M.E., Bainton D.F. Cytochemical localization of lysosomal enzymes in rat megakaryocytes and platelets. J. Clin. Invest. 1975, 56:1635-1649.
    • (1975) J. Clin. Invest. , vol.56 , pp. 1635-1649
    • Bentfeld, M.E.1    Bainton, D.F.2
  • 29
    • 0021277751 scopus 로고
    • Heterogeneity of human whole blood platelet subpopulations. III. Density-dependent differences in subcellular constituents
    • Corash L., Costa J.L., Shafer B., Donlon J.A., Murphy D. Heterogeneity of human whole blood platelet subpopulations. III. Density-dependent differences in subcellular constituents. Blood 1984, 64:185-193.
    • (1984) Blood , vol.64 , pp. 185-193
    • Corash, L.1    Costa, J.L.2    Shafer, B.3    Donlon, J.A.4    Murphy, D.5
  • 30
    • 0021024783 scopus 로고
    • Evidence that platelet alpha-granules are a major determinant of platelet density: studies in storage pool deficiency
    • Vicic W.J., Weiss H.J. Evidence that platelet alpha-granules are a major determinant of platelet density: studies in storage pool deficiency. Thromb. Haemost. 1983, 50:878-880.
    • (1983) Thromb. Haemost. , vol.50 , pp. 878-880
    • Vicic, W.J.1    Weiss, H.J.2
  • 31
  • 32
    • 7244258902 scopus 로고    scopus 로고
    • Human marrow megakaryocyte differentiation: multiparameter correlative analysis identifies von Willebrand factor as a sensitive and distinctive marker for early (2N and 4N) megakaryocytes
    • Tomer A. Human marrow megakaryocyte differentiation: multiparameter correlative analysis identifies von Willebrand factor as a sensitive and distinctive marker for early (2N and 4N) megakaryocytes. Blood 2004, 104:2722-2727.
    • (2004) Blood , vol.104 , pp. 2722-2727
    • Tomer, A.1
  • 33
    • 0036851876 scopus 로고    scopus 로고
    • Platelets in atherothrombosis
    • Ruggeri Z.M. Platelets in atherothrombosis. Nat. Med. 2002, 8:1227-1234.
    • (2002) Nat. Med. , vol.8 , pp. 1227-1234
    • Ruggeri, Z.M.1
  • 34
    • 0022541463 scopus 로고
    • Identification of platelet glycoprotein IIb/IIIa as the major binding site for released platelet-von Willebrand factor
    • Parker R.I., Gralnick H.R. Identification of platelet glycoprotein IIb/IIIa as the major binding site for released platelet-von Willebrand factor. Blood 1986, 68:732-736.
    • (1986) Blood , vol.68 , pp. 732-736
    • Parker, R.I.1    Gralnick, H.R.2
  • 35
    • 0025033429 scopus 로고
    • Use of homologous platelet factors in achieving total healing of recalcitrant skin ulcers
    • Atri S.C., Misra J., Bisht D., Misra K. Use of homologous platelet factors in achieving total healing of recalcitrant skin ulcers. Surgery 1990, 108:508-512.
    • (1990) Surgery , vol.108 , pp. 508-512
    • Atri, S.C.1    Misra, J.2    Bisht, D.3    Misra, K.4
  • 36
    • 31044436262 scopus 로고    scopus 로고
    • Platelets in inflammation and atherogenesis
    • Gawaz M., Langer H., May A.E. Platelets in inflammation and atherogenesis. J. Clin. Invest. 2005, 115:3378-3384.
    • (2005) J. Clin. Invest. , vol.115 , pp. 3378-3384
    • Gawaz, M.1    Langer, H.2    May, A.E.3
  • 37
    • 16444364523 scopus 로고    scopus 로고
    • Platelets and chemokines in atherosclerosis: partners in crime
    • Weber C. Platelets and chemokines in atherosclerosis: partners in crime. Circ. Res. 2005, 96:612-616.
    • (2005) Circ. Res. , vol.96 , pp. 612-616
    • Weber, C.1
  • 38
    • 1642475081 scopus 로고    scopus 로고
    • Autologous platelets as a source of proteins for healing and tissue regeneration
    • Anitua E., Andia I., Ardanza B., Nurden P., Nurden A.T. Autologous platelets as a source of proteins for healing and tissue regeneration. Thromb. Haemost. 2004, 91:4-15.
    • (2004) Thromb. Haemost. , vol.91 , pp. 4-15
    • Anitua, E.1    Andia, I.2    Ardanza, B.3    Nurden, P.4    Nurden, A.T.5
  • 39
    • 0036396275 scopus 로고    scopus 로고
    • Role of blood platelets in infection and inflammation
    • Klinger M.H., Jelkmann W. Role of blood platelets in infection and inflammation. J. Interferon Cytokine Res. 2002, 22:913-922.
    • (2002) J. Interferon Cytokine Res. , vol.22 , pp. 913-922
    • Klinger, M.H.1    Jelkmann, W.2
  • 40
    • 35148876896 scopus 로고    scopus 로고
    • Silencing of a targeted protein in in vivo platelets using a lentiviral vector delivering short hairpin RNA sequence
    • Ohmori T., Kashiwakura Y., Ishiwata A., Madoiwa S., Mimuro J., Sakata Y. Silencing of a targeted protein in in vivo platelets using a lentiviral vector delivering short hairpin RNA sequence. Arterioscler. Thromb. Vasc. Biol. 2007, 27:2266-2272.
    • (2007) Arterioscler. Thromb. Vasc. Biol. , vol.27 , pp. 2266-2272
    • Ohmori, T.1    Kashiwakura, Y.2    Ishiwata, A.3    Madoiwa, S.4    Mimuro, J.5    Sakata, Y.6
  • 41
    • 0030951133 scopus 로고    scopus 로고
    • Viral vectors for gene therapy of hematopoietic cells
    • Medin J.A., Karlsson S. Viral vectors for gene therapy of hematopoietic cells. Immunotechnology 1997, 3:3-19.
    • (1997) Immunotechnology , vol.3 , pp. 3-19
    • Medin, J.A.1    Karlsson, S.2
  • 42
    • 0030899477 scopus 로고    scopus 로고
    • Selection of retrovirally transduced cells to enhance the efficiency of gene therapy
    • Medin J.A., Karlsson S. Selection of retrovirally transduced cells to enhance the efficiency of gene therapy. Proc. Assoc. Am. Physicians 1997, 109:111-119.
    • (1997) Proc. Assoc. Am. Physicians , vol.109 , pp. 111-119
    • Medin, J.A.1    Karlsson, S.2
  • 43
    • 0035713273 scopus 로고    scopus 로고
    • Designing gene therapy vectors: avoiding immune responses by using tissue-specific promoters
    • Weeratna R.D., Wu T., Efler S.M., Zhang L., Davis H.L. Designing gene therapy vectors: avoiding immune responses by using tissue-specific promoters. Gene Ther. 2001, 8:1872-1878.
    • (2001) Gene Ther. , vol.8 , pp. 1872-1878
    • Weeratna, R.D.1    Wu, T.2    Efler, S.M.3    Zhang, L.4    Davis, H.L.5
  • 44
    • 0029053750 scopus 로고
    • Suppression of erythro-megakaryocytopoiesis and the induction of reversible thrombocytopenia in mice transgenic for the thymidine kinase gene targeted by the platelet glycoprotein alpha IIb promoter
    • Tronik-Le R.D., Roullot V., Schweitzer A., Berthier R., Marguerie G. Suppression of erythro-megakaryocytopoiesis and the induction of reversible thrombocytopenia in mice transgenic for the thymidine kinase gene targeted by the platelet glycoprotein alpha IIb promoter. J. Exp. Med. 1995, 181:2141-2151.
    • (1995) J. Exp. Med. , vol.181 , pp. 2141-2151
    • Tronik-Le, R.D.1    Roullot, V.2    Schweitzer, A.3    Berthier, R.4    Marguerie, G.5
  • 45
    • 3543139967 scopus 로고    scopus 로고
    • Ultrastructural analysis of bone marrow hematopoiesis in mice transgenic for the thymidine kinase gene driven by the alpha IIb promoter
    • Poujol C., Tronik-Le R.D., Tropel P., Roullot V., Nurden A., Marguerie G., Nurden P. Ultrastructural analysis of bone marrow hematopoiesis in mice transgenic for the thymidine kinase gene driven by the alpha IIb promoter. Blood 1998, 92:2012-2023.
    • (1998) Blood , vol.92 , pp. 2012-2023
    • Poujol, C.1    Tronik-Le, R.D.2    Tropel, P.3    Roullot, V.4    Nurden, A.5    Marguerie, G.6    Nurden, P.7
  • 46
    • 0030879352 scopus 로고    scopus 로고
    • A 2.7-kb portion of the 5? flanking region of the murine glycoprotein alphaIIb gene is transcriptionally active in primitive hematopoietic progenitor cells
    • Tropel P., Roullot V., Vernet M., Poujol C., Pointu H., Nurden P., Marguerie G., Tronik-Le R.D. A 2.7-kb portion of the 5? flanking region of the murine glycoprotein alphaIIb gene is transcriptionally active in primitive hematopoietic progenitor cells. Blood 1997, 90:2995-3004.
    • (1997) Blood , vol.90 , pp. 2995-3004
    • Tropel, P.1    Roullot, V.2    Vernet, M.3    Poujol, C.4    Pointu, H.5    Nurden, P.6    Marguerie, G.7    Tronik-Le, R.D.8
  • 47
    • 0021792005 scopus 로고
    • Cell-lineage antigens of the stem cell-megakaryocyte-platelet lineage are associated with the platelet IIb-IIIa glycoprotein complex
    • Berridge M.V., Ralph S.J., Tan A.S. Cell-lineage antigens of the stem cell-megakaryocyte-platelet lineage are associated with the platelet IIb-IIIa glycoprotein complex. Blood 1985, 66:76-85.
    • (1985) Blood , vol.66 , pp. 76-85
    • Berridge, M.V.1    Ralph, S.J.2    Tan, A.S.3
  • 48
    • 27144537145 scopus 로고    scopus 로고
    • Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia
    • Fang J., Hodivala-Dilke K., Johnson B.D., DU L.M., Hynes R.O., White G.C., Wilcox D.A. Therapeutic expression of the platelet-specific integrin, alphaIIbbeta3, in a murine model for Glanzmann thrombasthenia. Blood 2005, 106:2671-2679.
    • (2005) Blood , vol.106 , pp. 2671-2679
    • Fang, J.1    Hodivala-Dilke, K.2    Johnson, B.D.3    Du, L.M.4    Hynes, R.O.5    White, G.C.6    Wilcox, D.A.7
  • 50
    • 13244262627 scopus 로고    scopus 로고
    • Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIX
    • Shi Q., Wilcox D.A., Morateck P.A., Fahs S.A., Kenny D., Montgomery R.R. Targeting platelet GPIbalpha transgene expression to human megakaryocytes and forming a complete complex with endogenous GPIbbeta and GPIX. J. Thromb. Haemost. 2004, 2:1989-1997.
    • (2004) J. Thromb. Haemost. , vol.2 , pp. 1989-1997
    • Shi, Q.1    Wilcox, D.A.2    Morateck, P.A.3    Fahs, S.A.4    Kenny, D.5    Montgomery, R.R.6
  • 52
    • 0033578285 scopus 로고    scopus 로고
    • Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells
    • Wilcox D.A., Olsen J.C., Ishizawa L., Griffith M., White G.C. Integrin alphaIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus-transduced human hematopoietic cells. Proc. Natl Acad. Sci. USA 1999, 96:9654-9659.
    • (1999) Proc. Natl Acad. Sci. USA , vol.96 , pp. 9654-9659
    • Wilcox, D.A.1    Olsen, J.C.2    Ishizawa, L.3    Griffith, M.4    White, G.C.5
  • 53
    • 0034660309 scopus 로고    scopus 로고
    • Megakaryocyte-targeted synthesis of the integrin beta(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia
    • Wilcox D.A., Olsen J.C., Ishizawa L., Bray P.F., French D.L., Steeber D.A., Bell W.R., Griffith M., White G.C. Megakaryocyte-targeted synthesis of the integrin beta(3)-subunit results in the phenotypic correction of Glanzmann thrombasthenia. Blood 2000, 95:3645-3651.
    • (2000) Blood , vol.95 , pp. 3645-3651
    • Wilcox, D.A.1    Olsen, J.C.2    Ishizawa, L.3    Bray, P.F.4    French, D.L.5    Steeber, D.A.6    Bell, W.R.7    Griffith, M.8    White, G.C.9
  • 57
    • 33845648448 scopus 로고    scopus 로고
    • Efficient expression of a transgene in platelets using simian immunodeficiency virus-based vector harboring glycoprotein Ibalpha promoter: in vivo model for platelet-targeting gene therapy
    • Ohmori T., Mimuro J., Takano K., Madoiwa S., Kashiwakura Y., Ishiwata A., Niimura M., Mitomo K., Tabata T., Hasegawa M., Ozawa K., Sakata Y. Efficient expression of a transgene in platelets using simian immunodeficiency virus-based vector harboring glycoprotein Ibalpha promoter: in vivo model for platelet-targeting gene therapy. FASEB J. 2006, 20:1522-1524.
    • (2006) FASEB J. , vol.20 , pp. 1522-1524
    • Ohmori, T.1    Mimuro, J.2    Takano, K.3    Madoiwa, S.4    Kashiwakura, Y.5    Ishiwata, A.6    Niimura, M.7    Mitomo, K.8    Tabata, T.9    Hasegawa, M.10    Ozawa, K.11    Sakata, Y.12
  • 60
    • 57349147041 scopus 로고    scopus 로고
    • Altered bioavailability of platelet-derived factor VIII during thrombocytosis reverses phenotypic efficacy in haemophilic mice
    • Damon A.L., Scudder L.E., Gnatenko D.V., Sitaraman V., Hearing P., Jesty J., Bahou W.F. Altered bioavailability of platelet-derived factor VIII during thrombocytosis reverses phenotypic efficacy in haemophilic mice. Thromb. Haemost. 2008, 100:1111-1122.
    • (2008) Thromb. Haemost. , vol.100 , pp. 1111-1122
    • Damon, A.L.1    Scudder, L.E.2    Gnatenko, D.V.3    Sitaraman, V.4    Hearing, P.5    Jesty, J.6    Bahou, W.F.7
  • 61
    • 0029944191 scopus 로고    scopus 로고
    • Analysis of the thrombopoietin receptor (MPL) promoter implicates GATA and Ets proteins in the coregulation of megakaryocyte-specific genes
    • Deveaux S., Filipe A., Lemarchandel V., Ghysdael J., Romeo P.H., Mignotte V. Analysis of the thrombopoietin receptor (MPL) promoter implicates GATA and Ets proteins in the coregulation of megakaryocyte-specific genes. Blood 1996, 87:4678-4685.
    • (1996) Blood , vol.87 , pp. 4678-4685
    • Deveaux, S.1    Filipe, A.2    Lemarchandel, V.3    Ghysdael, J.4    Romeo, P.H.5    Mignotte, V.6
  • 62
    • 0037071389 scopus 로고    scopus 로고
    • The molecular and cellular biology of thrombopoietin: the primary regulator of platelet production
    • Kaushansky K., Drachman J.G. The molecular and cellular biology of thrombopoietin: the primary regulator of platelet production. Oncogene 2002, 21:3359-3367.
    • (2002) Oncogene , vol.21 , pp. 3359-3367
    • Kaushansky, K.1    Drachman, J.G.2
  • 63
    • 0029763215 scopus 로고    scopus 로고
    • Analysis of GPIIb/IIIa receptor number by quantification of 7E3 binding to human platelets
    • Wagner C.L., Mascelli M.A., Neblock D.S., Weisman H.F., Coller B.S., Jordan R.E. Analysis of GPIIb/IIIa receptor number by quantification of 7E3 binding to human platelets. Blood 1996, 88:907-914.
    • (1996) Blood , vol.88 , pp. 907-914
    • Wagner, C.L.1    Mascelli, M.A.2    Neblock, D.S.3    Weisman, H.F.4    Coller, B.S.5    Jordan, R.E.6
  • 64
    • 0029818864 scopus 로고    scopus 로고
    • The tissue-specific transcriptional regulation of the megakaryocytic glycoprotein IIb gene is controlled by interactions between a repressor and positive cis-acting elements
    • Prandini M.H., Martin F., Thevenon D., Uzan G. The tissue-specific transcriptional regulation of the megakaryocytic glycoprotein IIb gene is controlled by interactions between a repressor and positive cis-acting elements. Blood 1996, 88:2062-2070.
    • (1996) Blood , vol.88 , pp. 2062-2070
    • Prandini, M.H.1    Martin, F.2    Thevenon, D.3    Uzan, G.4
  • 65
    • 0029924148 scopus 로고    scopus 로고
    • Fetal bone marrow CD34+CD41+ cells are enriched for multipotent hematopoietic progenitors, but not for pluripotent stem cells
    • Murray L.J., Mandich D., Bruno E., DiGiusto R.K., Fu W.C., Sutherland D.R., Hoffman R., Tsukamoto A. Fetal bone marrow CD34+CD41+ cells are enriched for multipotent hematopoietic progenitors, but not for pluripotent stem cells. Exp. Hematol. 1996, 24:236-245.
    • (1996) Exp. Hematol. , vol.24 , pp. 236-245
    • Murray, L.J.1    Mandich, D.2    Bruno, E.3    DiGiusto, R.K.4    Fu, W.C.5    Sutherland, D.R.6    Hoffman, R.7    Tsukamoto, A.8
  • 66
    • 15244352092 scopus 로고    scopus 로고
    • GPIIb (CD41) integrin is expressed on mast cells and influences their adhesion properties
    • Berlanga O., Emambokus N., Frampton J. GPIIb (CD41) integrin is expressed on mast cells and influences their adhesion properties. Exp. Hematol. 2005, 33:403-412.
    • (2005) Exp. Hematol. , vol.33 , pp. 403-412
    • Berlanga, O.1    Emambokus, N.2    Frampton, J.3
  • 68
    • 0023840727 scopus 로고
    • Cultured human umbilical vein endothelial cells contain a membrane glycoprotein immunologically related to platelet glycoprotein Ib
    • Sprandio J.D., Shapiro S.S., Thiagarajan P., McCord S. Cultured human umbilical vein endothelial cells contain a membrane glycoprotein immunologically related to platelet glycoprotein Ib. Blood 1988, 71:234-237.
    • (1988) Blood , vol.71 , pp. 234-237
    • Sprandio, J.D.1    Shapiro, S.S.2    Thiagarajan, P.3    McCord, S.4
  • 69
    • 0025226575 scopus 로고
    • Cytokine-enhanced expression of glycoprotein Ib alpha in human endothelium
    • Konkle B.A., Shapiro S.S., Asch A.S., Nachman R.L. Cytokine-enhanced expression of glycoprotein Ib alpha in human endothelium. J. Biol. Chem. 1990, 265:19833-19838.
    • (1990) J. Biol. Chem. , vol.265 , pp. 19833-19838
    • Konkle, B.A.1    Shapiro, S.S.2    Asch, A.S.3    Nachman, R.L.4
  • 70
    • 0030750924 scopus 로고    scopus 로고
    • Human endothelial cells in culture and in vivo express on their surface all four components of the glycoprotein Ib/IX/V complex
    • Wu G., Essex D.W., Meloni F.J., Takafuta T., Fujimura K., Konkle B.A., Shapiro S.S. Human endothelial cells in culture and in vivo express on their surface all four components of the glycoprotein Ib/IX/V complex. Blood 1997, 90:2660-2669.
    • (1997) Blood , vol.90 , pp. 2660-2669
    • Wu, G.1    Essex, D.W.2    Meloni, F.J.3    Takafuta, T.4    Fujimura, K.5    Konkle, B.A.6    Shapiro, S.S.7
  • 71
    • 0033505511 scopus 로고    scopus 로고
    • Von Willebrand factor receptor GPIb alpha is expressed by human factor XIIIa-positive dermal dendrocytes and is upregulated by mast cell degranulation
    • Monteiro M.R., Shapiro S.S., Takafuta T., Menezes D.W., Murphy G.F. Von Willebrand factor receptor GPIb alpha is expressed by human factor XIIIa-positive dermal dendrocytes and is upregulated by mast cell degranulation. J. Invest. Dermatol. 1999, 113:272-276.
    • (1999) J. Invest. Dermatol. , vol.113 , pp. 272-276
    • Monteiro, M.R.1    Shapiro, S.S.2    Takafuta, T.3    Menezes, D.W.4    Murphy, G.F.5
  • 72
    • 0031586584 scopus 로고    scopus 로고
    • A GPIb alpha-related protein is expressed by fresh human breast carcinoma tissue and is regulated by a PKC-sensitive mechanism
    • Oleksowicz L., Dutcher J.P., DeLeon-Fernandez M., Etkind P. A GPIb alpha-related protein is expressed by fresh human breast carcinoma tissue and is regulated by a PKC-sensitive mechanism. Exp. Cell Res. 1997, 237:110-117.
    • (1997) Exp. Cell Res. , vol.237 , pp. 110-117
    • Oleksowicz, L.1    Dutcher, J.P.2    DeLeon-Fernandez, M.3    Etkind, P.4
  • 73
    • 0035261381 scopus 로고    scopus 로고
    • Genomic strategies to identify mammalian regulatory sequences
    • Pennacchio L.A., Rubin E.M. Genomic strategies to identify mammalian regulatory sequences. Nat. Rev. Genet. 2001, 2:100-109.
    • (2001) Nat. Rev. Genet. , vol.2 , pp. 100-109
    • Pennacchio, L.A.1    Rubin, E.M.2
  • 74
    • 0028055281 scopus 로고
    • Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus
    • Lewis P.F., Emerman M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J. Virol. 1994, 68:510-516.
    • (1994) J. Virol. , vol.68 , pp. 510-516
    • Lewis, P.F.1    Emerman, M.2
  • 75
    • 0025279872 scopus 로고
    • Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection
    • Miller D.G., Adam M.A., Miller A.D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell. Biol. 1990, 10:4239-4242.
    • (1990) Mol. Cell. Biol. , vol.10 , pp. 4239-4242
    • Miller, D.G.1    Adam, M.A.2    Miller, A.D.3
  • 77
    • 0027199982 scopus 로고
    • Association of integrase, matrix, and reverse transcriptase antigens of human immunodeficiency virus type 1 with viral nucleic acids following acute infection
    • Bukrinsky M.I., Sharova N., McDonald T.L., Pushkarskaya T., Tarpley W.G., Stevenson M. Association of integrase, matrix, and reverse transcriptase antigens of human immunodeficiency virus type 1 with viral nucleic acids following acute infection. Proc. Natl Acad. Sci. USA 1993, 90:6125-6129.
    • (1993) Proc. Natl Acad. Sci. USA , vol.90 , pp. 6125-6129
    • Bukrinsky, M.I.1    Sharova, N.2    McDonald, T.L.3    Pushkarskaya, T.4    Tarpley, W.G.5    Stevenson, M.6
  • 79
    • 33745225238 scopus 로고    scopus 로고
    • K. C. von, A. Fischer, and B. Kerem, Fragile sites are preferential targets for integrations of MLV vectors in gene therapy
    • Bester A.C., Schwartz M., Schmidt M., Garrigue A., Hacein-Bey-Abina S., Cavazzana-Calvo M., Ben-Porat N. K. C. von, A. Fischer, and B. Kerem, Fragile sites are preferential targets for integrations of MLV vectors in gene therapy. Gene Ther. 2006, 13:1057-1059.
    • (2006) Gene Ther. , vol.13 , pp. 1057-1059
    • Bester, A.C.1    Schwartz, M.2    Schmidt, M.3    Garrigue, A.4    Hacein-Bey-Abina, S.5    Cavazzana-Calvo, M.6    Ben-Porat, N.7
  • 80
    • 15244346492 scopus 로고    scopus 로고
    • Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells
    • De P.M., Montini E., Santoni de Sio F.R., Benedicenti F., Gentile A., Medico E., Naldini L. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 2005, 105:2307-2315.
    • (2005) Blood , vol.105 , pp. 2307-2315
    • De, P.M.1    Montini, E.2    Santoni de Sio, F.R.3    Benedicenti, F.4    Gentile, A.5    Medico, E.6    Naldini, L.7
  • 81
    • 36049014397 scopus 로고    scopus 로고
    • A roadmap to safe, efficient, and stable lentivirus-mediated gene therapy with hematopoietic cell transplantation
    • Neschadim A., McCart J.A., Keating A., Medin J.A. A roadmap to safe, efficient, and stable lentivirus-mediated gene therapy with hematopoietic cell transplantation. Biol. Blood Marrow Transplant. 2007, 13:1407-1416.
    • (2007) Biol. Blood Marrow Transplant. , vol.13 , pp. 1407-1416
    • Neschadim, A.1    McCart, J.A.2    Keating, A.3    Medin, J.A.4
  • 82
    • 84988366841 scopus 로고    scopus 로고
    • Intravenous Immunoglobulin (IVIG) Diminishes Immune-Mediated Clearance of Platelets Expressing an Integrin {alpha}IIb{beta}3 Transgene Product That Restores Hemostasis in a Canine Model for Glanzmann Thrombasthenia
    • Fang J., Jensen E.S., Du L.M., Boudreaux M.K., Wilcox D.A. Intravenous Immunoglobulin (IVIG) Diminishes Immune-Mediated Clearance of Platelets Expressing an Integrin {alpha}IIb{beta}3 Transgene Product That Restores Hemostasis in a Canine Model for Glanzmann Thrombasthenia. ASH Annual Meeting Abstracts 2006, 108:3263.
    • (2006) ASH Annual Meeting Abstracts , vol.108 , pp. 3263
    • Fang, J.1    Jensen, E.S.2    Du, L.M.3    Boudreaux, M.K.4    Wilcox, D.A.5
  • 83
    • 84957409544 scopus 로고
    • Sur une nouvelle variet e de dystrophie thrombocytarie-hemorragipare congenitale.
    • BERNARD J., SOULIER J.P. Sur une nouvelle variet e de dystrophie thrombocytarie-hemorragipare congenitale. Sem. Hôp. 1948, 24:3217-3223.
    • (1948) Sem. Hôp. , vol.24 , pp. 3217-3223
    • Bernard, J.1    Soulier, J.P.2
  • 84
    • 0035986783 scopus 로고    scopus 로고
    • Inherited thrombocytopenias: from genes to therapy
    • Balduini C.L., Iolascon A., Savoia A. Inherited thrombocytopenias: from genes to therapy. Haematologica 2002, 87:860-880.
    • (2002) Haematologica , vol.87 , pp. 860-880
    • Balduini, C.L.1    Iolascon, A.2    Savoia, A.3
  • 85
    • 0034646267 scopus 로고    scopus 로고
    • Generation and rescue of a murine model of platelet dysfunction: the Bernard-Soulier syndrome
    • Ware J., Russell S., Ruggeri Z.M. Generation and rescue of a murine model of platelet dysfunction: the Bernard-Soulier syndrome. Proc. Natl Acad. Sci. USA 2000, 97:2803-2808.
    • (2000) Proc. Natl Acad. Sci. USA , vol.97 , pp. 2803-2808
    • Ware, J.1    Russell, S.2    Ruggeri, Z.M.3
  • 86
    • 4544229639 scopus 로고    scopus 로고
    • Recombinant factor VIIa: review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors
    • Abshire T., Kenet G. Recombinant factor VIIa: review of efficacy, dosing regimens and safety in patients with congenital and acquired factor VIII or IX inhibitors. J. Thromb. Haemost. 2004, 2:899-909.
    • (2004) J. Thromb. Haemost. , vol.2 , pp. 899-909
    • Abshire, T.1    Kenet, G.2
  • 87
    • 6644228391 scopus 로고    scopus 로고
    • Safety and efficacy of a new recombinant FVIII formulated with sucrose (rFVIII-FS) in patients with haemophilia A: a long-term, multicentre clinical study in Japan
    • Yoshioka A., Shima M., Fukutake K., Takamatsu J., Shirahata A. Safety and efficacy of a new recombinant FVIII formulated with sucrose (rFVIII-FS) in patients with haemophilia A: a long-term, multicentre clinical study in Japan. Haemophilia 2001, 7:242-249.
    • (2001) Haemophilia , vol.7 , pp. 242-249
    • Yoshioka, A.1    Shima, M.2    Fukutake, K.3    Takamatsu, J.4    Shirahata, A.5
  • 88
    • 15344351616 scopus 로고    scopus 로고
    • Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: results of an international clinical investigation
    • Kreuz W., Gill J.C., Rothschild C., Manco-Johnson M.J., Lusher J.M., Kellermann E., Gorina E., Larson P.J. Full-length sucrose-formulated recombinant factor VIII for treatment of previously untreated or minimally treated young children with severe haemophilia A: results of an international clinical investigation. Thromb. Haemost. 2005, 93:457-467.
    • (2005) Thromb. Haemost. , vol.93 , pp. 457-467
    • Kreuz, W.1    Gill, J.C.2    Rothschild, C.3    Manco-Johnson, M.J.4    Lusher, J.M.5    Kellermann, E.6    Gorina, E.7    Larson, P.J.8
  • 91
    • 41149085573 scopus 로고    scopus 로고
    • Economic evaluation of major knee surgery with recombinant activated factor VII in hemophilia patients with high titer inhibitors and advanced knee arthropathy: exploratory results via literature-based modeling
    • Ballal R.D., Botteman M.F., Foley I., Stephens J.M., Wilke C.T., Joshi A.V. Economic evaluation of major knee surgery with recombinant activated factor VII in hemophilia patients with high titer inhibitors and advanced knee arthropathy: exploratory results via literature-based modeling. Curr. Med. Res. Opin. 2008, 24:753-768.
    • (2008) Curr. Med. Res. Opin. , vol.24 , pp. 753-768
    • Ballal, R.D.1    Botteman, M.F.2    Foley, I.3    Stephens, J.M.4    Wilke, C.T.5    Joshi, A.V.6
  • 94
    • 9144232123 scopus 로고    scopus 로고
    • Inhibitors in congenital coagulation disorders
    • Key N.S. Inhibitors in congenital coagulation disorders. Br. J. Haematol. 2004, 127:379-391.
    • (2004) Br. J. Haematol. , vol.127 , pp. 379-391
    • Key, N.S.1
  • 96
    • 0033936079 scopus 로고    scopus 로고
    • Properties of anti-factor VIII inhibitor antibodies in hemophilia A patients
    • Scandella D.H. Properties of anti-factor VIII inhibitor antibodies in hemophilia A patients. Semin. Thromb. Hemost. 2000, 26:137-142.
    • (2000) Semin. Thromb. Hemost. , vol.26 , pp. 137-142
    • Scandella, D.H.1
  • 97
    • 0028334619 scopus 로고
    • A practical guide to the evaluation and treatment of hemophilia
    • Furie B., Limentani S.A., Rosenfield C.G. A practical guide to the evaluation and treatment of hemophilia. Blood 1994, 84:3-9.
    • (1994) Blood , vol.84 , pp. 3-9
    • Furie, B.1    Limentani, S.A.2    Rosenfield, C.G.3
  • 98
    • 78649328862 scopus 로고    scopus 로고
    • Endothelial and platelet FVIII/VWF expression divergence in clinical effect in murine models of hemophilia a with and without FVIII inhibitory antibodies
    • Shi Q., Fahs S.A., Wilcox D.A., Weiler H., Haberichter S.L., Montgomery R.R. Endothelial and platelet FVIII/VWF expression divergence in clinical effect in murine models of hemophilia a with and without FVIII inhibitory antibodies. Blood 2006, 108:938a.
    • (2006) Blood , vol.108
    • Shi, Q.1    Fahs, S.A.2    Wilcox, D.A.3    Weiler, H.4    Haberichter, S.L.5    Montgomery, R.R.6
  • 100
    • 0025748557 scopus 로고
    • The role of von Willebrand factor multimers and propeptide cleavage in binding and stabilization of factor VIII
    • Wise R.J., Dorner A.J., Krane M., Pittman D.D., Kaufman R.J. The role of von Willebrand factor multimers and propeptide cleavage in binding and stabilization of factor VIII. J. Biol. Chem. 1991, 266:21948-21955.
    • (1991) J. Biol. Chem. , vol.266 , pp. 21948-21955
    • Wise, R.J.1    Dorner, A.J.2    Krane, M.3    Pittman, D.D.4    Kaufman, R.J.5
  • 101
    • 0032696798 scopus 로고    scopus 로고
    • Regulation of factor VIII expression and activity by von Willebrand factor
    • Kaufman R.J., Pipe S.W. Regulation of factor VIII expression and activity by von Willebrand factor. Thromb. Haemost. 1999, 82:201-208.
    • (1999) Thromb. Haemost. , vol.82 , pp. 201-208
    • Kaufman, R.J.1    Pipe, S.W.2
  • 102
    • 0015923364 scopus 로고
    • Von Willebrand factor: dissociation from antihemophilic factor procoagulant activity
    • Weiss H.J., Hoyer I.W. Von Willebrand factor: dissociation from antihemophilic factor procoagulant activity. Science 1973, 182:1149-1151.
    • (1973) Science , vol.182 , pp. 1149-1151
    • Weiss, H.J.1    Hoyer, I.W.2
  • 103
    • 0035822005 scopus 로고    scopus 로고
    • Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A
    • Roth D.A., Tawa N.E., O'Brien J.M., Treco D.A., Selden R.F. 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    Tawa, N.E.2    O'Brien, J.M.3    Treco, D.A.4    Selden, R.F.5
  • 106
    • 0025243601 scopus 로고
    • Cell biology of von Willebrand factor
    • Wagner D.D. Cell biology of von Willebrand factor. Annu. Rev. Cell Biol. 1990, 6:217-246.
    • (1990) Annu. Rev. Cell Biol. , vol.6 , pp. 217-246
    • Wagner, D.D.1
  • 107
    • 0017658776 scopus 로고
    • Synthesis of factor VIII antigen by cultured guinea pig megakaryocytes
    • Nachman R., Levine R., Jaffe E.A. Synthesis of factor VIII antigen by cultured guinea pig megakaryocytes. J. Clin. Invest. 1977, 60:914-921.
    • (1977) J. Clin. Invest. , vol.60 , pp. 914-921
    • Nachman, R.1    Levine, R.2    Jaffe, E.A.3
  • 108
    • 0015905229 scopus 로고
    • Synthesis of antihemophilic factor antigen by cultured human endothelial cells
    • Jaffe E.A., Hoyer L.W., Nachman R.L. Synthesis of antihemophilic factor antigen by cultured human endothelial cells. J. Clin. Invest. 1973, 52:2757-2764.
    • (1973) J. Clin. Invest. , vol.52 , pp. 2757-2764
    • Jaffe, E.A.1    Hoyer, L.W.2    Nachman, R.L.3
  • 109
    • 0027259388 scopus 로고
    • The Weibel-Palade body: the storage granule for von Willebrand factor and P-selectin
    • Wagner D.D. The Weibel-Palade body: the storage granule for von Willebrand factor and P-selectin. Thromb. Haemost. 1993, 70:105-110.
    • (1993) Thromb. Haemost. , vol.70 , pp. 105-110
    • Wagner, D.D.1
  • 110
    • 17844372996 scopus 로고    scopus 로고
    • Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy
    • Xu L., Nichols T.C., Sarkar R., McCorquodale S., Bellinger D.A., Ponder K.P. Absence of a desmopressin response after therapeutic expression of factor VIII in hemophilia A dogs with liver-directed neonatal gene therapy. Proc. Natl Acad. Sci. USA 2005, 102:6080-6085.
    • (2005) Proc. Natl Acad. Sci. USA , vol.102 , pp. 6080-6085
    • Xu, L.1    Nichols, T.C.2    Sarkar, R.3    McCorquodale, S.4    Bellinger, D.A.5    Ponder, K.P.6
  • 111
    • 0037182848 scopus 로고    scopus 로고
    • Treatment of a patient with hemophilia A and hepatitis C virus-related cirrhosis by living-related liver transplantation from an obligate carrier donor
    • Horita K., Matsunami H., Shimizu Y., Shimizu A., Kurimoto M., Suzuki K., Tsukadaira T., Arai M. Treatment of a patient with hemophilia A and hepatitis C virus-related cirrhosis by living-related liver transplantation from an obligate carrier donor. Transplantation 2002, 73:1909-1912.
    • (2002) Transplantation , vol.73 , pp. 1909-1912
    • Horita, K.1    Matsunami, H.2    Shimizu, Y.3    Shimizu, A.4    Kurimoto, M.5    Suzuki, K.6    Tsukadaira, T.7    Arai, M.8
  • 112
    • 28444498815 scopus 로고    scopus 로고
    • Lack of desmopressin (DDAVP) response in men with hemophilia A following liver transplantation
    • Lamont P.A., Ragni M.V. Lack of desmopressin (DDAVP) response in men with hemophilia A following liver transplantation. J. Thromb. Haemost. 2005, 3:2259-2263.
    • (2005) J. Thromb. Haemost. , vol.3 , pp. 2259-2263
    • Lamont, P.A.1    Ragni, M.V.2
  • 113
    • 0033538448 scopus 로고    scopus 로고
    • Expression of factor VIII by murine liver sinusoidal endothelial cells
    • Do H., Healey J.F., Waller E.K., Lollar P. Expression of factor VIII by murine liver sinusoidal endothelial cells. J. Biol. Chem. 1999, 274:19587-19592.
    • (1999) J. Biol. Chem. , vol.274 , pp. 19587-19592
    • Do, H.1    Healey, J.F.2    Waller, E.K.3    Lollar, P.4
  • 114
    • 0034455182 scopus 로고    scopus 로고
    • Interactions between von Willebrand factor and Factor VIII: where did they first meet
    • Montgomery R.R., Gill J.C. Interactions between von Willebrand factor and Factor VIII: where did they first meet. J. Pediatr. Hematol. Oncol. 2000, 22:269-275.
    • (2000) J. Pediatr. Hematol. Oncol. , vol.22 , pp. 269-275
    • Montgomery, R.R.1    Gill, J.C.2
  • 115
    • 77954729893 scopus 로고    scopus 로고
    • Activation of human endothelial cells from specific vascular beds induces the release of a FVIII storage pool
    • Shahani T., Lavend'homme R., Luttun A., Saint-Remy J.M., Peerlinck K., Jacquemin M. Activation of human endothelial cells from specific vascular beds induces the release of a FVIII storage pool. Blood 2010, 115:4902-4909.
    • (2010) Blood , vol.115 , pp. 4902-4909
    • Shahani, T.1    Lavend'homme, R.2    Luttun, A.3    Saint-Remy, J.M.4    Peerlinck, K.5    Jacquemin, M.6
  • 117
    • 0023918719 scopus 로고
    • Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells
    • Kaufman R.J., Wasley L.C., Dorner A.J. Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J. Biol. Chem. 1988, 263:6352-6362.
    • (1988) J. Biol. Chem. , vol.263 , pp. 6352-6362
    • Kaufman, R.J.1    Wasley, L.C.2    Dorner, A.J.3
  • 118
    • 0024597022 scopus 로고
    • Effect of von Willebrand factor coexpression on the synthesis and secretion of factor VIII in Chinese hamster ovary cells
    • Kaufman R.J., Wasley L.C., Davies M.V., Wise R.J., Israel D.I., Dorner A.J. Effect of von Willebrand factor coexpression on the synthesis and secretion of factor VIII in Chinese hamster ovary cells. Mol. Cell. Biol. 1989, 9:1233-1242.
    • (1989) Mol. Cell. Biol. , vol.9 , pp. 1233-1242
    • Kaufman, R.J.1    Wasley, L.C.2    Davies, M.V.3    Wise, R.J.4    Israel, D.I.5    Dorner, A.J.6
  • 119
    • 53449090226 scopus 로고    scopus 로고
    • Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity
    • Shi Q., Fahs S.A., Wilcox D.A., Kuether E.L., Morateck P.A., Mareno N., Weiler H., Montgomery R.R. Syngeneic transplantation of hematopoietic stem cells that are genetically modified to express factor VIII in platelets restores hemostasis to hemophilia A mice with preexisting FVIII immunity. Blood 2008, 112:2713-2721.
    • (2008) Blood , vol.112 , pp. 2713-2721
    • Shi, Q.1    Fahs, S.A.2    Wilcox, D.A.3    Kuether, E.L.4    Morateck, P.A.5    Mareno, N.6    Weiler, H.7    Montgomery, R.R.8
  • 120
    • 20444417088 scopus 로고    scopus 로고
    • Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: lessons from platelet-expressed factor VIII
    • Yarovoi H., Nurden A.T., Montgomery R.R., Nurden P., Poncz M. Intracellular interaction of von Willebrand factor and factor VIII depends on cellular context: lessons from platelet-expressed factor VIII. Blood 2005, 105:4674-4676.
    • (2005) Blood , vol.105 , pp. 4674-4676
    • Yarovoi, H.1    Nurden, A.T.2    Montgomery, R.R.3    Nurden, P.4    Poncz, M.5
  • 121
    • 45549109031 scopus 로고    scopus 로고
    • Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors
    • Gewirtz J., Thornton M.A., Rauova L., Poncz M. Platelet-delivered factor VIII provides limited resistance to anti-factor VIII inhibitors. J. Thromb. Haemost. 2008, 6:1160-1166.
    • (2008) J. Thromb. Haemost. , vol.6 , pp. 1160-1166
    • Gewirtz, J.1    Thornton, M.A.2    Rauova, L.3    Poncz, M.4
  • 122
    • 51649110213 scopus 로고    scopus 로고
    • Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots
    • Neyman M., Gewirtz J., Poncz M. Analysis of the spatial and temporal characteristics of platelet-delivered factor VIII-based clots. Blood 2008, 112:1101-1108.
    • (2008) Blood , vol.112 , pp. 1101-1108
    • Neyman, M.1    Gewirtz, J.2    Poncz, M.3
  • 123
    • 78649333324 scopus 로고    scopus 로고
    • Sustained Phenotypic Correction of Murine Hemophilia A with Pre-Existing Anti-FVIII Immunity Using Lentivirus-Mediated Platelet-Specific FVIII Gene Transfer
    • Shi Q., Kuether E.L., Cooley B.C., Fahs S.A., Schroeder J.A., Wilcox D.A., Montgomery R.R. Sustained Phenotypic Correction of Murine Hemophilia A with Pre-Existing Anti-FVIII Immunity Using Lentivirus-Mediated Platelet-Specific FVIII Gene Transfer. ASH Annual Meeting Abstracts 2009, 114:29.
    • (2009) ASH Annual Meeting Abstracts , vol.114 , pp. 29
    • Shi, Q.1    Kuether, E.L.2    Cooley, B.C.3    Fahs, S.A.4    Schroeder, J.A.5    Wilcox, D.A.6    Montgomery, R.R.7
  • 125
    • 0031727621 scopus 로고    scopus 로고
    • Development of anaphylactic shock in haemophilia B patients with inhibitors
    • Warrier I., Lusher J.M. Development of anaphylactic shock in haemophilia B patients with inhibitors. Blood Coagul. Fibrinolysis 1998, 9(Suppl 1):S125-S128.
    • (1998) Blood Coagul. Fibrinolysis , vol.9 , Issue.SUPPL 1
    • Warrier, I.1    Lusher, J.M.2
  • 126
    • 0037328109 scopus 로고    scopus 로고
    • Treatment of the bleeding inhibitor patient
    • Astermark J. Treatment of the bleeding inhibitor patient. Semin. Thromb. Hemost. 2003, 29:77-86.
    • (2003) Semin. Thromb. Hemost. , vol.29 , pp. 77-86
    • Astermark, J.1
  • 127
    • 0033935277 scopus 로고    scopus 로고
    • Inhibitor antibodies to factor VIII and factor IX: management
    • Lusher J.M. Inhibitor antibodies to factor VIII and factor IX: management. Semin. Thromb. Hemost. 2000, 26:179-188.
    • (2000) Semin. Thromb. Hemost. , vol.26 , pp. 179-188
    • Lusher, J.M.1
  • 129
    • 0032748593 scopus 로고    scopus 로고
    • Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy
    • Herzog R.W., High K.A. Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy. Thromb. Haemost. 1999, 82:540-546.
    • (1999) Thromb. Haemost. , vol.82 , pp. 540-546
    • Herzog, R.W.1    High, K.A.2
  • 131
    • 7444269023 scopus 로고    scopus 로고
    • AAV vectors for hemophilia B gene therapy Mt. Sinai
    • Chao H., Walsh C.E. AAV vectors for hemophilia B gene therapy Mt. Sinai. J. Med. 2004, 71:305-313.
    • (2004) J. Med. , vol.71 , pp. 305-313
    • Chao, H.1    Walsh, C.E.2
  • 133
    • 44049090692 scopus 로고    scopus 로고
    • AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects
    • Hasbrouck N.C., High K.A. AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects. Gene Ther. 2008, 15:870-875.
    • (2008) Gene Ther. , vol.15 , pp. 870-875
    • Hasbrouck, N.C.1    High, K.A.2
  • 134
    • 54849162200 scopus 로고    scopus 로고
    • Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice
    • Chang A.H., Stephan M.T., Lisowski L., Sadelain M. Erythroid-specific human factor IX delivery from in vivo selected hematopoietic stem cells following nonmyeloablative conditioning in hemophilia B mice. Mol. Ther. 2008, 16:1745-1752.
    • (2008) Mol. Ther. , vol.16 , pp. 1745-1752
    • Chang, A.H.1    Stephan, M.T.2    Lisowski, L.3    Sadelain, M.4
  • 136
    • 3042815635 scopus 로고    scopus 로고
    • In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice
    • Xu L., O'Malley T., Sands M.S., Wang B., Meyerrose T., Haskins M.E., Ponder K.P. In vivo transduction of hematopoietic stem cells after neonatal intravenous injection of an amphotropic retroviral vector in mice. Mol. Ther. 2004, 10:37-44.
    • (2004) Mol. Ther. , vol.10 , pp. 37-44
    • Xu, L.1    O'Malley, T.2    Sands, M.S.3    Wang, B.4    Meyerrose, T.5    Haskins, M.E.6    Ponder, K.P.7
  • 139
    • 38949118647 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Murphy S.L., High K.A. Gene therapy for haemophilia. Br. J. Haematol. 2008, 140:479-487.
    • (2008) Br. J. Haematol. , vol.140 , pp. 479-487
    • Murphy, S.L.1    High, K.A.2
  • 140
    • 44049098137 scopus 로고    scopus 로고
    • Update on progress and hurdles in novel genetic therapies for hemophilia
    • High K.A. Update on progress and hurdles in novel genetic therapies for hemophilia. Hematology Am. Soc. Hematol. Educ. Program 2007, 2007:466-472.
    • (2007) Hematology Am. Soc. Hematol. Educ. Program , vol.2007 , pp. 466-472
    • High, K.A.1
  • 141
    • 33746733616 scopus 로고    scopus 로고
    • Gene therapy for hemophilia
    • Ponder K.P. Gene therapy for hemophilia. Curr. Opin. Hematol. 2006, 13:301-307.
    • (2006) Curr. Opin. Hematol. , vol.13 , pp. 301-307
    • Ponder, K.P.1
  • 142
    • 0019426232 scopus 로고
    • Human megakaryocytes. II. Expression of platelet proteins in early marrow megakaryocytes
    • Rabellino E.M., Levene R.B., Leung L.L., Nachman R.L. Human megakaryocytes. II. Expression of platelet proteins in early marrow megakaryocytes. J. Exp. Med. 1981, 154:88-100.
    • (1981) J. Exp. Med. , vol.154 , pp. 88-100
    • Rabellino, E.M.1    Levene, R.B.2    Leung, L.L.3    Nachman, R.L.4
  • 143
    • 0030307624 scopus 로고    scopus 로고
    • The platelet glycoprotein Ib-V-IX system: regulation of gene expression
    • Roth G.J., Yagi M., Bastian L.S. The platelet glycoprotein Ib-V-IX system: regulation of gene expression. Stem Cells 1996, 14 Suppl 1:188-193.
    • (1996) Stem Cells , pp. 188-193
    • Roth, G.J.1    Yagi, M.2    Bastian, L.S.3
  • 145
    • 0023098325 scopus 로고
    • Structure of the rat platelet factor 4 gene: a marker for megakaryocyte differentiation
    • Doi T., Greenberg S.M., Rosenberg R.D. Structure of the rat platelet factor 4 gene: a marker for megakaryocyte differentiation. Mol. Cell Biol. 1987, 7:898-904.
    • (1987) Mol. Cell Biol. , vol.7 , pp. 898-904
    • Doi, T.1    Greenberg, S.M.2    Rosenberg, R.D.3
  • 146
    • 0029144833 scopus 로고
    • Regulation of gene transcription during the differentiation of megakaryocytes
    • Uzan G., Prandini M.H., Berthier R. Regulation of gene transcription during the differentiation of megakaryocytes. Thromb. Haemost. 1995, 74:210-212.
    • (1995) Thromb. Haemost. , vol.74 , pp. 210-212
    • Uzan, G.1    Prandini, M.H.2    Berthier, R.3
  • 148
    • 0032005935 scopus 로고    scopus 로고
    • Thrombopoietin and its receptor, c-mpl, are constitutively expressed by mouse liver endothelial cells: evidence of thrombopoietin as a growth factor for liver endothelial cells
    • Cardier J.E., Dempsey J. Thrombopoietin and its receptor, c-mpl, are constitutively expressed by mouse liver endothelial cells: evidence of thrombopoietin as a growth factor for liver endothelial cells. Blood 1998, 91:923-929.
    • (1998) Blood , vol.91 , pp. 923-929
    • Cardier, J.E.1    Dempsey, J.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.