Role of Vector in Activation of T Cell Subsets in Immune Responses against the Secreted Transgene Product Factor IX

Molecular Therapy

Volumn 1, Issue 3, 2000, Pages 225-235

  Fields, Paul A ^a   Kowalczyk, Dariusz W ^b   Arruda, Valder R ^a   Armstrong, Elina ^a   McCleland, Mark L ^a   Hagstrom, J Nathan ^a   Pasi, K John ^c   Ertl, Hildegund C J ^b   Herzog, Roland W ^a   High, Katherine A ^a,b  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c ROYAL FREE HOSPITAL   (United Kingdom)

Author keywords

Factor IX; adeno associated virus; adenovirus; muscle; plasmid; antibodies; T helper cells; CTL; gene transfer; hemophilia B

Indexed keywords

BLOOD CLOTTING FACTOR 9; CYTOKINE; IMMUNOGLOBULIN G;

ADENOVIRUS; ADOPTIVE TRANSFER; ANIMAL; ARTICLE; BIOSYNTHESIS; C57BL MOUSE; CYTOLOGY; CYTOTOXIC T LYMPHOCYTE; FLUORESCENT ANTIBODY TECHNIQUE; GENE TRANSFER; GENE VECTOR; GENETICS; HEMOPHILIA B; IMMUNOLOGY; METABOLISM; MOUSE; T LYMPHOCYTE SUBPOPULATION; TH1 CELL; TH2 CELL; TRANSGENE;

ADENOVIRIDAE; ADOPTIVE TRANSFER; ANIMALS; CYTOKINES; FACTOR IX; FLUORESCENT ANTIBODY TECHNIQUE; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMOPHILIA B; IMMUNOGLOBULIN G; MICE; MICE, INBRED C57BL; T-LYMPHOCYTE SUBSETS; T-LYMPHOCYTES, CYTOTOXIC; TH1 CELLS; TH2 CELLS; TRANSGENES;

EID: 0034151775     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0032     Document Type: Article

References (39)

1. Herzog R. W., Hagstrom J. N., Kung Z-H., Tai S. J., Wilson J. M., Fisher K. J., High K. A. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA. 94:1997;5804-5809.
2. Herzog R. W., Yang E. Y., Couto L. B., Hagstrom J. N., Elwell D., Fields P. A., Burton M., Bellinger D. A., Read M. S., Brinkhous K. M., Podsakoff G. M., Nichols T. C., Kurtzman G. J., High K. A. Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat. Med. 5:1999;56-63.
3. High K. A. Factor IX: Molecular structure, epitopes, and mutations associated with inhibitor formation. Adv. Exp. Med. Biol. 386:1995;79-86.
4. Herzog R. W., High K. A. AAV-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy. Thromb. Haemostasis. 82:1999;540-546.
5. Yang Y., Xiang Z., Ertl H. C. J., Wilson J. M. Upregulation of class I major histocompatibility complex antigens by interferon γ is necessary for T-cell-mediated elimination of recombinant adenovirus-infected hepatocytes in vivo. Proc. Natl. Acad. Sci. USA. 92:1995;7257-7261.
6. Jooss K., Yang Y., Fisher K. J., Wilson J. M. Transduction of dendritic cells by DNA viral vectors directs the immune response to transgene products in muscle fibers. J. Virol. 72:1998;4212-4223.
7. Coutelier J. P., Van der Logt J. T. M., Heessen F. W. A, Warnier G., Van Snick J. IgG2a restriction of murine antibodies elicited by viral infections. J. Exp. Med. 165-169:1987;64.
8. Yang Y. P., Trinchieri G., Wilson J. M. Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nat. Med. 1:1995;890-893.
9. Yang Y., Ehlen Haecker S., Su Q., Wilson J. M. Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum. Mol. Genet. 5:1996;1703-1712.
10. Yang Y., Greenough K., Wilson J. M. Transient immune blockade prevents formation of neutralizing antibody to recombinant adenovirus and allows repeated gene transfer to mouse liver. Gene Ther. 3:1996;412-420.
11. Lagrange P. H., Mackaness G. B., Miller T. E. Influence of dose and route of antigen injection on the immunological induction of T cells. J. Exp. Med. 139:1977;528-542.
12. Nakai H., Herzog R., Hagstrom J. N., Kung J., Walter J., Tai S., Iwaki Y., Kurtzman G., Fisher K., Couto L., High K. A. AAV-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood. 91:1998;4600-4607.
13. Matsushita T., Elliger S., Elliger C., Podsakoff G., Villarreal L., Kurtzman G. J., Iwaki Y., Colosi P. Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther. 5:1998;938-945.
14. Walter J., You Q., Hagstrom J. N., Sands M., High K. A. Successful expression of human factor IX following repeat administration of an adenoviral vector in mice. Proc. Natl. Acad. Sci. USA. 93:1996;3056-3061.
15. Kessler P. D., Podsakoff G. M., Chen X., McQuiston S. A., Colosi P. C., Matelis L. A., Kurtzman G. J., Bryne B. J. Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein. Proc. Natl. Acad. Sci. USA. 93:1996;14082-14087.
16. Kung J., Hagstrom J., Cass D., Tai S., Lin H. F., Stafford D. W., High K. A. Human F.IX corrects the bleeding diathesis of mice with hemophilia B. Blood. 91:1998;784-790.
17. Caamono J. H., Rizzo C. A., Durham S. K., Barton D. S., Raventos-Suarez C., Snapper C. M., Bravo R. Nuclear factor (NF)-kB2 (p100/p52) is required for normal splenic microarchitecture and B cell-mediated immune responses. J. Exp. Med. 187:1998;185-196.
18. Lin K-Y., Guarnieri F. G., Staveley-O'Carroll K. F., Levitsky H. I., August J. T., Pardoll D. M., Wu T-C. Treatment of established tumors with a novel vaccine that enhances major histocompatibility class II presentation of tumor antigen. Cancer Res. 56:1996;21-26.
19. Fisher K. J., Jooss K., Alston J., Yang Y., Ehlen-Haecker S., High K., Pathak R., Raper S. E., Wilson J. M. Recombinant adeno-associated virus for muscle directed gene therapy. Nat. Med. 3:1997;306-312.
20. Mombaerts P., Iacomini J., Johnson R. S., Herrup K., Tonegawa S., Papaioannou V. E. Rag-1-deficient mice have no mature B and T lymphocytes. Cell. 68:1992;869-877.
21. Lin H-F., Maeda N., Smithies O., Straight D. L., Stafford D. W. A coagulation factor IX-deficient mouse model for human hemophilia B. Blood. 90:1997;3962-3966.
22. b H chain constant regions are derived from two distinct isotypic genes. Immunogenetics. 29:1989;92-97.
23. Morgado M. G., Cam P., Gris-Liebe C., Cazenave P-A., Jouvin-Marche E. Further evidence that Balb/c and C57BL/6 γ2a genes originate from distinct isotypes. EMBO J. 8:1989;3245-3251.
24. Corr M., Lee D. J., Carson D. A., Tighe H. Gene vaccination with naked plasmid DNA: Mechanism of CTL priming. J. Exp. Med. 184:1996;1555-1560.
25. Doe B., Selby M., Barnett S., Baenziger J., Walker C. M. Induction of cytotoxic T lymphocytes by intramuscular immunization with plasmid DNA is facilitated by bone marrow-derived cells. Proc. Natl. Acad. Sci. USA. 93:1996;8578-8583.
26. Ulmer J. B., Deck R. R., Dewitt C. M., Donnelly J. J., Liu M. A. Generation of MHC class I-restricted cytotoxic lymphocytes by expression of a viral protein in muscle cell: Antigen presentation by non-muscle cell. Immunology. 89:1996;59-67.
27. Fu T. M., Ulmer J. B., Caufield M. J., Deck R. R., Friedman A., Wang S., Liu X., Donnelly J. J., Liu M. A. Priming of cytotoxic T lymphocytes by DNA vaccines: Requirement for professional antigen presenting cells and evidence for antigen transfer from myotubes. Mol. Med. 3:1997;362-371.
28. Rock K. L., Rothstein L., Gamble S., Fleischhacker C. Characterization of antigen-presenting cells that present exogenous antigens in association with class I MHC molecules. J. Immunol. 150:1993;438.
29. Carbone F. R., Kurts C., Bennett S. R. M., Miller J. F. A. P., Heath W. R. Cross-presentation: A general mechanism for CTL immunity and tolerance. Immunol. Today. 19:1998;368-373.
30. Song S., Morgan M., Ellis T., Poirier A., Chesnut K., Wang J., Brantly M., Muzyczka N., Bryne B. J., Atkinson M., Flotte T. R. Sustained secretion of human alpha-1-antitrypsin from murine muscle transduced with adeno-associated virus vector. Proc. Natl. Acad. Sci. USA. 95:1998;14384-14388.
31. Xiao X., Li J., Samulski R. J. Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector. J. Virol. 70:1996;8098-8108.
32. Snyder R. O., Spratt S. K., Lagarde C., Bohl D., Kaspar B., Sloan B., Cohen L. K., Danos O. Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum. Gene Ther. 8:1997;1891-1900.
33. Manning W. C., Paliard X., Zhou S., Bland M. P., Lee A. Y., Hong K., Walker C. M., Escobedo J. A., Dwarki V. Genetic immunization with adeno-associated virus vectors expressing herpes simplex virus type 2 glycoproteins B and D. J. Virol. 71:1997;7960-7962.
34. Brockstedt D. G., Podsakoff G. M., Fong L., Kurtzman G. J., Mueller-Ruchholtz W., Engleman E. G. Induction of immunity to antigens expressed by recombinant adeno-associated virus depends on the route of administration. Clin. Immunol. 92:1999;67-75.
35. Qian J., Bi L., Kazazian H. H., Hoyer L. W. Inhibitor development and T cell response to human factor VIII in murine hemophilia A. Blood. 88:1996;656a.
36. Nilsson I. M., Berntorp E., Zettervall O. Induction of split tolerance and clinical cure in high-responding hemophiliacs with factor IX antibodies. Proc. Natl. Acad. Sci. USA. 83:1986;9169-9173.
37. Tripathy S. K., Black H. B., Goldwasser E., Leiden J. M. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nat. Med. 2:1996;545-550.
38. Pastore L., Morral N., Zhou H. S., Garcia R., Parks R. J., Kochanek S., Graham F. L., Lee B., Beaudet A. L. Use of a liver-specific promoter reduces immune response to the transgene in adenoviral vectors. Hum. Gene Ther. 10:1999;1773-1781.
39. Dai Y., Schwarz E. M., Gu D., Zhang W-W., Sarvetnick N., Verma I. M. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl. Acad. Sci. USA. 92:1995;1401-1405.