Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

Human Gene Therapy

Volumn 11, Issue 5, 2000, Pages 729-738

  Chuah, Marinee K L ^a   Van Damme, An ^a   Zwinnen, Hans ^a   Goovaerts, Inge ^a   Vanslembrouck, Veerle ^a   Collen, Desire ^a   Vandendriessche, Thierry ^a  

^a DEPARTMENT OF PLANT SYSTEMS BIOLOGY   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; VIRUS ENVELOPE PROTEIN; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BONE MARROW CELL; CONTROLLED STUDY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; HEMOPHILIA A; HUMAN; HUMAN CELL; MOLONEY LEUKEMIA ONCOVIRUS; MOUSE; NONHUMAN; SCID MOUSE;

ANIMALS; BONE MARROW CELLS; BONE MARROW TRANSPLANTATION; FACTOR VIII; HUMANS; MICE; MICE, INBRED NOD; MICE, SCID; RECOMBINANT PROTEINS; RETROVIRIDAE; STROMAL CELLS;

ALOCASIA MACRORRHIZOS; ANIMALIA; GIBBON APE LEUKEMIA VIRUS; HYLOBATES SP.; MOLONEY MURINE LEUKEMIA VIRUS; MURINAE; ONCOVIRINAE;

EID: 0034689505     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340050015626     Document Type: Article

References (44)

1. Allay, J.A., Dennis, J.E., Haynesworth, S.E., Majumdar, M.K., Clapp, D.W., Shultz, L.D., Caplan, A.I., and Gerson, S.L. (1997). LacZ and interleukin-3 expression in vivo after retroviral transduction of marrow-derived human osteogenic mesenchymal progenitors. Hum. Gene Ther. 8, 1417-1427.
2. Bienzle, D., Abrams Ogg, A.C., Kruth, S.A., Ackland Snow, J., Carter, R.F., Dick, J.E., Jacobs, R.M., Kamel Reid, S., and Dube, I.D. (1994). Gene transfer into hematopoietic stem cells: Long-term maintenance of in vitro activated progenitors without marrow ablation. Proc. Natl. Acad. Sci. U.S.A. 91, 350-354.
3. Chiang, C.G., Rubin, H.L., Cherington, V., Wang, T., Sobolewski, J., McGrath, C.A., Gaffney, A., Emami, S., Sarver, N., Levine, P.H., Greenberger, J.S., and Hurwitz, D.R. (1999). 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. Hum. Gene Ther. 10, 61-76.
4. 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.
5. Chuah, M.K., Collen, D., and Vandendriessche, T. (1998a). Gene therapy for hemophilia: Hopes and hurdles. Crit. Rev. Oncol. Hematol. 28, 153-171.
6. Chuah, M.K., Brems, H., Vanslembrouck, V., Collen, D., and Vandendriessche, T. (1998b). Bone marrow stromal cells as potential targets for gene therapy of hemophilia. A. Hum. Gene Ther. 9, 353-365.
7. Clark, L.N., Koehler, U., Ward, D.C., Wienberg, J., and Hewitt, J.E. (1996). Analysis of the organisation and localisation of the FSHD-associated tandem array in primates: Implications for the origin and evolution of the 3.3 kb repeat family. Chromosoma 105, 180-189.
8. Connelly, S., Smith, T.A., Dhir, G., Gardner, J.M., Mehaffey, M.G., Zaret, K.S., McClelland, A., and Kaleko, M. (1995). In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice. Hum. Gene Ther. 6, 185-193.
9. Connelly, S., Mount, J., Mauser, A., Gardner, J.M., Kaleko, M., McClelland, A., and Lothrop, C.D., Jr. (1996). Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood 88, 3846-3853.
10. Connelly, S., Andrews, J.L., Gallo, A.M., Kayda, D.B., Qian, J., Hoyer, L., Kadan, M.J., Gorziglia, M.I., Trapnell, B.C., McClelland, A., and Kaleko, M. (1998). Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood 91, 3273-3281.
11. Dick, J.E. (1996). Normal and leukemic human stem cells assayed in SCID mice. Semin. Immunol. 8, 197-206.
12. Drize, N.J., Surin, V.L., Gan, O.I., Deryugina, E.I., and Chertkov, J.L. (1992). Gene therapy model for stromal precursor cells of hematopoietic microenvironment. Leukemia 6, 1745-1755.
13. Dwarki, V.J., Belloni, P., Nijjar, T., Smith, J., Couto, L., Rabier, M., Clift, S., Berns, A., and Cohen, L.K. (1995). Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice. Proc. Natl. Acad. Sci. U.S.A. 92, 1023-1027.
14. Evans, G.L., and Morgan, R.A. (1998). Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc. Natl. Acad. Sci. U.S.A. 95, 5734-5739.
15. Flanagan, J.R., Becker, K.G., Ennist, D.L., Gleason, S.L., Driggers, P.H., Levi, B.Z., Appella, E., and Ozato, K. (1992). Cloning of a negative transcription factor that binds to the upstream conserved region of Moloney murine leukemia virus. Mol. Cell. Biol. 12, 38-44.
16. Hoeben, R.C., Van Der Jagt, R.C., Schoute, F., Van Tilburg, N.H., Verbeet, M.P., Briet, E., Van Ormondt, H., and Van Der Eb, A.J. (1990). Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer. J. Biol. Chem. 265, 7318-7323.
17. Hoeben, R.C., Migchielsen, A.A., Van Der Jagt, R.C., Van Ormondt, H., and Van Der Eb, A.J. (1991). Inactivation of the Moloney murine leukemia virus long terminal repeat in murine fibroblast cell lines is associated with methylation and dependent on its chromosomal position. J. Virol. 65, 904-912.
18. Hoeben, R.C., Einerhand, M.P., Briet, E., Van Ormondt, H., Valerio, D., and Van Der Eb, A.J. (1992). Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb. Haemost. 67, 341-345.
19. Hoeben, R.C., Fallaux, F.J., Van Tilburg, N.H., Cramer, S.J., Van Ormondt, H., Briet, E., and Van Der Eb, A.J. (1993). Toward gene therapy for hemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplanation into immunodeficient mice. Hum. Gene Ther. 4, 179-186.
20. Israel, D.I., and Kaufman, R.J. (1990). Retroviral-mediated transfer and amplification of a functional human factor VIII gene. Blood 75, 1074-1080.
21. Kasper, C.K., Aledort, L., and Aronson, D. (1975). Proceedings: A more uniform measurement of factor VIII inhibitors. Thromb. Diath. Haemorrh. 34, 612-614.
22. Kaufman, R.J. (1991). Insight into the structure, function, and biosynthesis of factor VIII through recombinant DNA technology. Ann. Hematol. 63, 155-165.
23. Kay, M.A., and High, K. (1999). Gene therapy for the hemophilias. Proc. Natl. Acad. Sci. U.S.A. 96, 9973-9975.
24. Koeberl, D.D., Halbert, C.L., Krumm, A., and Miller, A.D. (1995). Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation. Hum. Gene Ther. 6, 469-479.
25. 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. 7, 1415-1422.
26. Li, K.J., Dilber, M.S., Abedi, M.R., Bjorkstrand, B., Smith, C.I., Garoff, H., Gahrton, G., and Xanthopoulos, K.G. (1995). Retroviral-mediated gene transfer into human bone marrow stromal cells: Studies of efficiency and in vivo survival in SCID mice. Eur. J. Haematol. 55, 302-306.
27. Loh, T.P., Sievert, L.L., and Scott, R.W. (1990). Evidence for a stem cell-specific repressor of Moloney murine leukemia virus expression in embryonal carcinoma cells. Mol. Cell. Biol. 10, 4045-4057.
28. Lynch, C.M., Israel, D.I., Kaufman, R.J., and Miller, A.D. (1993). Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum. Gene Ther. 4, 259-272.
29. Miller, A.D., Garcia, J.V., Von Suhr, N., Lynch, C.M., Wilson, C., and Eiden, J.V. (1991). Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J. Virol. 65, 2220-2224.
30. 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.
31. Palmer, T.D., Thompson, A.R., and Miller, A.D. (1989). Production of human factor IX in animals by genetically modified skin fibroblasts: Potential therapy for hemophilia B. Blood 73, 438-445.
32. Pawliuk, R., Eaves, C.J., and Humphries, R.K. (1997). Sustained high-level reconstitution of the hematopoietic system by preselected hematopoietic cells expressing a transduced cell-surface antigen. Hum. Gene Ther. 8, 1595-1604.
33. Pawliuk, R., Bachelot, T., Wise, R.J., Mathews-Roth, M.M., and Leboulch, P. (1999). Long-term cure of the photosensitivity of murine erythropoietic protoporphyria by preselective gene therapy. Nature Med. 5, 768-773.
34. Pereira, R.F., Halford, K.W., O'Hara, M.D., Leeper, D.B., Sokolov, B.P., Pollard, M.D., Bagasra, O., and Prockop, D.J. (1995). Cultured adherent cells from marrow can serve as long-lasting precursor cells for bone, cartilage, and lung in irradiated mice. Proc. Natl. Acad. Sci. U.S.A. 92, 4857-4861.
35. Pereira, R.F., O'Hara, M.D., Laptev, A.V., Halford, K.W., Pollard, M.D., Class, R., Simon, D., Livezey, K., and Prockop, D.J. (1998). Marrow stromal cells as a source of progenitor cells for nonhematopoietic tissues in transgenic mice with a phenotype of osteogenesis imperfecta. Proc. Natl. Acad. Sci. U.S.A. 95, 1142-1147.
36. Pittenger, M.F., Mackay, A.M., Beck, S.C., Jaiswal, R.K., Douglas, R., Mosca, J.D., Moorman, M.A., Simonetti, D.W., Craig, S., and Marshak, D.R. (1999). Multilineage potential of adult human mesenchymal stem cells. Science 284, 143-147.
37. Prockop, D.J. (1997). Marrow stromal cells as stem cells for nonhematopoietic tissues. Science 276, 71-74.
38. Quesenberry, P.J., Ramshaw, H., Crittenden, R.B., Stewart, F.M., Rao, S., Peters, S., Becker, P., Lowry, P., Blomberg, M., and Reilly, J. (1994). Engraftment of normal murine marrow into nonmyeloablated host mice. Blood Cells 20, 348-350.
39. Robbins, P.B., Skelton, D.C., Yu, X.J., Halene, S., Leonard, E.H., and Kohn, D.B. (1998). Consistent, persistent expression from modified retroviral vectors in murine hematopoietic stem cells. Proc. Natl. Acad. Sci. U.S.A. 95, 10182-10187.
40. Sadler, J.E., and Davie, E.W. (1987). Hemophilia A, hemophilia B, and von Willebrand's disease. In The Molecular Basis of Blood Diseases. A.W. Nienhuis, P. Leder, and P.W. Majerus, eds. (W.B. Saunders, New York) p. 575.
41. Saxe, D.F., Boggs, S.S., and Boggs, D.R. (1984). Transplantation of chromosomally marked syngeneic marrow cells into mice not subjected to hematopoietic stem cell depletion. Exp. Hematol. 12, 277-283.
42. Shultz, L.D., Schweitzer, P.A., Christianson, S.W., Gott, B., Schweitzer, I.B., Tennent, B., McKenna, S., Mobraaten, L., Rajan, T.V., and Greiner, D.L. (1995). Multiple detects in innate and adaptive immunologic function in NOD/LtSz-scid mice. J. Immunol. 154, 180-191.
43. Vandendriessche, T., Vanslembrouck, V., Goovaerts, I., Zwinnen, H., Vanderhaeghen, M.L., Collen, D., and Chuah, M.K. (1999). Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice. Proc. Natl. Acad. Sci. U.S.A. 96, 10379-10384.
44. Zatloukal, K., Cotten, M., Berger, M., Schmidt, W., Wagner, E., and Birnstiel, M.L. (1994). In vivo production of human factor VII in mice after intransplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery. Proc. Natl. Acad. Sci. U.S.A. 91, 5148-5152.