Expression of human factor VIII under control of the platelet-specific αIIb promoter in megakaryocytic cell line as well as storage together with VWF

Molecular Genetics and Metabolism

Volumn 79, Issue 1, 2003, Pages 25-33

  Shi, Q ^a,b   Wilcox, D A ^a,b   Fahs, S A ^b   Kroner, P A ^a,b   Montgomery, R R ^a,b,c  

^a Midwest Athletes Against Childhood Cancer Inc   (United States)

^b BLOOD RESEARCH INSTITUTE   (United States)

^c Children's Hospital of Wisconsin   (United States)

Author keywords

Factor VIII; Hemophilia A; Megakaryocytic cell line; VWF; IIb Promoter

Indexed keywords

BLOOD CLOTTING FACTOR 8; VON WILLEBRAND FACTOR;

ARTICLE; BLOOD VESSEL INJURY; CELL LINE; GENE CASSETTE; GENE THERAPY; GENETIC TRANSFECTION; HEMATOPOIETIC CELL; HEMOPHILIA A; HEMOSTASIS; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; MEGAKARYOCYTE; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN EXPRESSION; PROTEIN SECRETION; PROTEIN SYNTHESIS; TARGET CELL; THROMBOCYTE; THROMBOCYTE ACTIVATION;

CUCUMBER MOSAIC VIRUS;

EID: 0038522536     PISSN: 10967192     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1096-7192(03)00049-0     Document Type: Article

References (52)

1. R.J. Kaufman, S.E. Antonarakis, in: R. Hoffman, E.Z. Benz, S.J. Shattil, H.J. Furieb Cohen, L.E. Siberstein, P.P. McGlare, Structure, Biology, and Practice, third ed., 2000, pp. 1850-1868.
2. Rosendaal F.R., Smit C., Briët E. Hemophilia treatment in historical perspective: a review of medical and social developments. Ann. Hematol. 62:1991;5-15.
3. Hedner U. Treatment of patients with factor VIII and factor IX inhibitors with special focuses on the use of recombinant factor VIIa. Thromb. Haemost. 82:1999;531-539.
4. Dwarki V.J., Belloni P., Nijjar T., Smith J., Couto L., Rabier M., Clift S., Berns A., Cohen L.K. Gene therapy of hemophilia A: production of therapeutic levels of human factor VIII in vivo in mice. Proc. Natl. Acad. Sci. USA. 92:1995;1023-1027.
5. Balague C., Zhou J., Dai Y., Alemany R., Josephs S.F., Andreason G., Hariharan M., Sethi E., Prokopenko E., Jan H.Y., Lou Y.C., Hubert-Leslie D., Ruiz L., Zhang W.W. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector. Blood. 95:2000;820-828.
6. Chao H., Mao L., Bruce A.T., Walsh C.F. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood. 95:2000;1594-1599.
7. Chuah M.K., Vandendriessche T., Morgan R.A. Development and analysis of retroviral vector expressing human factor VIII as a potential gene therapy for hemophilia A. Hum. Gene Ther. 6:1995;1363-1377.
8. Chuah M., Chuah M.K., Brems H., Vanlembrouck V., Collen D., Vandendriessche T. Bone marrow stromal cells as targets for gene therapy of hemophilia A. Hum. Gene Ther. 9:1998;353-365.
9. Roth D.A., Tawa N.E. Jr., O'Brien J.M., Treco D.A., Selden R.F. Factor VIII transkaryotic therapy study group nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N. Engl. J. Med. 344:2001;1735-1742.
10. Gnatenko D.V., Saenko E.L., Jesty J., Cao L.X., Hearing P., Bahou W.F. Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to hemophilia A gene therapy. Br. J. Hematol. 104:1999;27-36.
11. Wion K.J., Kelly D., Summerfield J.A., Tuddenham E.G., Cawn R.M. Distribution of factor VIII mRNA and antigen in human liver and other tissues. Nature. 3317:1985;726-729.
12. Hollestelle M.J., Thinnes T., Crain K., Stiko A., Kruijt J.K., van Berkel T.J., Loskutoff D.J., van Mourik J.A. Tissue distribution of factor VIII gene expression in vitro-a closer look. Thromb. Haemost. 86:2001;855-861.
13. Levinson B., Kenwrick S., Gamel P., Fisher K., Gitchier J. Evidence for a third transcript from the human factor VIII gene. Genomics. 14:1992;585-589.
14. Do H., Healey J.F., Waller E.K., Lollar P. Expression of factor VIII by murine liver sinusoidal endothelial cells. J. Biol. Chem. 274:1999;19587-19592.
15. Rosenberg J.B., Foster P.A., Kaufman R.J., Vokac E.A., Moussalli M., Kroner P.A., Montgomery R.R. Intracellular trafficking of factor VIII to von Willebrand factor storage granules. J. Clin. Invest. 72:1998;1968-1977.
16. Rosenberg J.B., Greengard J.S., Montgomery R.R. Genetic induction of a releasable pool of factor VIII in human endothelial cells. Arterioscl. Thromb. Vas. 20:2000;2689-2695.
17. Doering C.B., Josephson C.D., Craddock H.N., Lollar P. Factor VIII expression in azoxymethane-induced murine fulminant hepatic failure. Blood. 100:2002;143-147.
18. Connelly S., Andrews J.L., Gallo-Penn A.M., Tagliavacca L., Kaufman R.J., Kaleko M. Evaluation of an adenoviral vector encoding full-length human factor VIII in hemophiliac mice. Thromb. Haemost. 81:1999;234-239.
19. Miao C.H., Ye X., Thompson A.R. High-level gene expression of human factor VIII in vivo was achieved by a liver-specific construct containing Apo E-HCR and a heterogous intron. Blood. 98:2001;425a.
20. Hoeben R.C., van der Jagt R.C., Schoute F., van Tilburg N.H., Verbeet M.P., Briet E., van Ormondt H., van der Eb A.J. Expression of functional factor VIII in primary human skin fibroblasts after retrovirus mediated transfer. J. Biol. Chem. 265:1990;7318-7323.
21. Israel D.I., Kaufman R.H. Retroviral mediated transfer and amplification of a function human factor VIII gene. Blood. 75:1990;1074-1080.
22. Lynch C.M., Israel D.I., Millet A.D. Toward somatic cell gene therapy using endothelial cells. J. Cell Biochem. 14E:1990;222.
23. Lin Y., Chang L., Solovey A., Healey J.F., Lollar P., Hebbel R.P. Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood. 99:2002;457-462.
24. Wagner D.D. Cell biology of von Willebrand factor. Annu. Rev. Cell Biol. 6:1990;217-246.
25. Mayadas T.N., Wagner D.D. von Willebrand factor biosynthesis and processing. Ann. NY Acad. Sci. 614:1991;153-166.
26. Jaffe E.A., Hoyer L.W., Nachman R.L. Synthesis of antihemophilic factor antigen by cultured human endothelial cells. J. Clin. Invest. 52:1973;2757-2764.
27. Wagner D.D. The Weibel-Palade body: the storage granules for von Willebrand factor and P-selectin. Thromb. Haemost. 70:1993;105-110.
28. Nachman R., Levine R., Jaffe E.A. Synthesis of factor VIII antigen by culture guinea pig megakaryocytes. J. Clin. Invest. 60:1977;914-921.
29. 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. 9:1989;1233-1242.
30. Wilcox D.A., Olsen J.C., Ishizawn L., Griffith M., White G.C. Integrin. αIIb promoter-targeted expression of gene products in megakaryocytes derived from retrovirus transduced human hematopoietic cells Proc. Natl. Acad. Sci. USA. 96:1999;9654-9659.
31. Romeo P.H., Prandini M.H., Joulin V., Mignotte V., Prenant M., Vainchenker W., Marguerie G., Uzan G. Megakaryocytic and erythrocytic lineages share specific transcription factors. Nature. 94:1990;447-449.
32. Fong A.M., Santoro S.A. Transcriptional regulation of αIIb integrin gene expression during megakaryocytic differentiation of K562 cells. Role of a silencer element. J. Biol. Chem. 269:1994;18441-18447.
33. 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. 88:1996;2062-2070.
34. ′ -flanking region of the rat GPIIb gene by studies in a primary rat marrow culture system Blood. 84:1994;3385-3393.
35. Tronik-Le Roux D., Roullet V., Scxhweitzer 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 αIIb promoter. J. Exp. Med. 181:1995;2141-2151.
36. ′ flanking region of the murine glycoprotein αIIb gene is transcriptionally active in primitive hematopoietic progenitor cells Blood. 90:1997;2995-3004.
37. Pittman R.J., Marquette K.A., Kaufman R.J. Role of the B domain for factor VIII expression and function. Blood. 84:1994;4214-4225.
38. Kroner P.A., Friedman K.D., Fahs S.A., Scott J.P., Montgomery R.R. Abnormal binding of factor VIII is linked with the substitution of glutamine of arginine 91 in von Willebrand factor in a variant form of von Willebrand disease. J. Biol. Chem. 266:1991;1946-1949.
39. Greenberg S.M., Rosenthal D.S., Greenley T.A., Tantravahi R., Handin R.I. Characterization of a new megakaryocytic cell line: the Dami cell. Blood. 72:1988;1968-1977.
40. Ballen K.K., Ritchie A.J., Murphy C., Handin R.I., Ewenstein B.M. Expression and activation of protein kinase C isoforms in a human megakaryocytic cell line. Exp. Hematol. 24:1996;1501-1508.
41. Sandra L.H., Fahs S.A., Montgomery R.R. Von Willebrand factor storage and multimerization: 2 independent intracellular processes. Blood. 96:2000;1808-1815.
42. Moore H.P., Walker M.D., Lee F., Kelly R.B. Expressing a human proinsulin cDNA in a mouse ACTH-secreting cell: intracellular storage, proteolytic, and secretion on stimulation. Cell. 35:1983;531-538.
43. Wagner D.D., Saffaripour S., Bonfanti R., Sadler J.E., Cramer E.M., Chapman B., Mayadas T.N. Induction of specific storage organelles by von Willebrand factor propolypeptide. Cell. 64:1991;403-413.
44. Rodriguez M.H., Enjolras N., Plantier J.L., Rea M., Leboeuf M., Uzan G., Bordet J.C., Negrier C. Expression of coagulation factor IX in a haematopoietic cell line. Thromb. Haemost. 87:2002;366-373.
45. Hoeben R.C., Einerhand M.P., Briet E., van Ormondt H., Valerio D., van der Eb A.J. Toward gene therapy in hemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haemotopoietic progenitor cells. Thromb. Haemost. 67:1992;341-345.
46. Challita P.M., Kohn B.D. Lack of expression from a retroviral vector after transcription of murine haemotopoietic stem cells is associated with methylation in vivo. Proc. Natl. Acad. Sci. USA. 91:1994;2567-2571.
47. Nakai H., Herzog R.W., Hagstrom J.N., Walter J., Kung S.H., Yang E.Y., Tai S.J., Iwaki Y., Kurtzman G.J., Fisher K.J., Colosi P., Couto L.B., High K.A. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood. 91:1998;4600-4607.
48. Baskar J.F., Smith P.P., Ciment G.S., Hoffmann S., Tucker C., Tenney D.J., Colberg-Poley A.M., Nelson J.A., Ghazal P. The enhancer domain of the human cytomegalovirus majoy immediate early promoter determines cell type-specific expression in transgenic mice. J. Virol. 70:1996;3207-3214.
49. Phillipa D.R., Charo I.F., Parise L.V., Fitzgerald L.A. The platelet membrane glycoprotein IIb-IIIa complex. Blood. 71:1988;831-843.
50. Fraser J.K., Leahy M.F., Berridge M.V. Expression of antigens of the platelet glycoprotein IIb/IIIa complex on human hematopoietic stem cells. Blood. 68:1986;762-769.
51. Berkow R.L., Straneva J.E., Bruno E., Beyer G.S., Burgess J.S., Hoffman R. Isolation of human megakaryocytes by density centrifugal elutriation. J. Lab. Clin. Med. 103:1984;811-818.
52. Bowie E.J., Solberg L.A. Jr., Fass D.N., Johnson C.M., Knutson G.J., Stewart M.L., Zoecklein L.J. Transplantation of normal bone marrow into a pig with severe von Willebrand disease. J. Clin. Invest. 78:1986;26-30.