Safe and effective regulation of hematocrit by gene gun administration of an erythropoietin-encoding DNA plasmid

Human Gene Therapy

Volumn 10, Issue 4, 1999, Pages 659-665

  Klinman, Dennis M ^a,c   Conover, Jacqueline ^a   Leiden, Jeffrey M ^b   Rosenberg, Amy S ^a   Sechler, Joan M G ^a  

^a Center for Biologics Evaluation/Res   (United States)

^b UNIVERSITY OF CHICAGO   (United States)

^c CENTER FOR DRUG EVALUATION AND RESEARCH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ERYTHROPOIETIN; PLASMID DNA; PLASMID VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CELL MIGRATION; CONTROLLED STUDY; DENDRITIC CELL; DNA TRANSFER; FEMALE; GENE GUN; GENETIC CODE; GENETIC REGULATION; GENETIC TRANSFECTION; HEMATOCRIT; KERATINOCYTE; MOUSE; NONHUMAN; SECRETION; SKIN GRAFT;

ANEMIA; ANIMALS; BASE SEQUENCE; BIOLISTICS; DNA; DNA PRIMERS; ERYTHROPOIETIN; FEMALE; HEMATOCRIT; MICE; MICE, INBRED BALB C; PLASMIDS;

ANIMALIA; MURINAE;

EID: 0033105454     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950018733     Document Type: Article

References (38)

1. AL-JANADI, M., AL-BALLA, S., AL-DALAAN, A., and RAZIUDDIN, S. (1993). Cytokine profile in systemic lupus erythematosus, rheumatoid arthritis, and other rheumatic diseases. J. Clin. Immunol. 13, 58-67.
2. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
3. BOHL, D., and HEARD, J. (1997). Modulation of erythropoietin delivery from engineered muscles in mice. Hum. Gene Ther. 8, 195-204.
4. CONDON, C., WATKINS, S.C., CELLUZZI, C.M., THOMPSON, K., and FALO, L.D. (1997). DNA-based immunization by in vivo transfection of dendritic cells. Nature Med. 2, 1122-1128.
5. CONROY, R.M. (1996). Selected strategies to augment polynucleotide immunization. Gene Ther. 3, 67-74.
6. DAVIS, H.L., MICHEL, M., and WHALEN, R.G. (1993). DNA-based immunization induces continuous secretion of hepatitis B surface antigen and high levels of circulating antibody. Hum. Mol. Genet. 2, 1847-1851.
7. DHAWAN, J., PAN, L.C., PAVLATH, J.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1991). Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
8. ERSLEV, A.J. (1990). Relative polycythemia. In Hematology, 4th Ed. W.J. Williams, E. Beutler, A.J. Ersley, and M.A. Lichtman, eds. (McGraw-Hill, New York) pp. 715-717.
9. FYNAN, E.F., WEBSTER, R.G., FULLER, D.H., HAYNES, J.R., SANTORO, J.C., and ROBINSON, H.L. (1993). DNA vaccines: Protective immunizations by parenteral, mucosal, and gene-gun inoculations. Proc. Natl. Acad. Sci. U.S.A. 90, 11478-11482.
10. FYNAN, E.F., WEBSTER, R.G., FULLER, D.H., HAYNES, J.R., SANTORO, J.C., AND ROBINSON, H.L. (1995). DNA vaccines: A novel approach to immunization. Int. J Immunopharmacol. 17, 79-83.
11. HAM, A.W. (1969). Histology, 6th Ed. (Lippincott, Philadelphia) p. 188.
12. HARTIKKA, J., SAWDEY, M., CORNEFERT-JENSEN, F., MARGALITH, M., BARNHART, K., NOLASCO, M., VAHLSING, H.L., MEEK, J., MARQUET, M., HOBART, P., NORMAN, J., and MANTHORPE, M. (1996). An improved plasmid DNA expression vector for direct injection into skeletal muscle. Hum. Gene Ther. 7, 1205-1217.
13. HENGGE, U.R., CHAN, E.F., FOSTER, R.A., WALKER, P.S., and VOGEL, J.C. (1995). Cytokine gene expression in epidermis with biological effects following injection of naked DNA. Nature Genet. 10, 161-166.
14. HUARD, J., AKKARAJU, G., WATKINS, S.C., PIKE-CAVALCOLI, M., and GLORIOSO, J. (1997). LacZ gene transfer to skeletal muscle using a replication defective herpes simplex virus type 1 mutant vector. Hum. Gene Ther. 8, 439-452. [Abstract]
15. KLINMAN, D.M., TAKENO, M., ICHINO, M., GU, M., YAMSHCHIKOV, G., MOR, G., and CONOVER, J. (1997). DNA vaccines: Safety and efficacy issues. Springer Semin. Immunopathol. 19, 245-256.
16. KLINMAN, D.M., SECHLER, J.M.G., CONOVER, J., GU, M., and ROSENBERG, A.S. (1998). Contribution of cells at the site of DNA vaccination to the generation of antigen specific immunity and memory. J. Immunol. 160, 2388-2394.
17. KREMMER, E.J., and PERRICAUDET, M. (1992). Adenovirus and adeno-associated virus mediated gene transfer. Br. Med. Bull. 51, 31-44.
18. MANTHORPE, M., CORNEFERT, J.F., HARTIKKA, J., FELGNER, J., RUNDELL, A., MARGALITH, M., and DWARKI, V. (1993). Gene therapy by intramuscular injection of plasmid DNA: Studies on firefly luciferase gene expression in mice. Hum. Gene Ther. 4, 419-431.
19. MENDELL, J.F., KISSEL, J.T., AMATO, A.A., KLING, W., MORRIS, G.E., and BURGHES, A.H.M. (1995). Myoblast transfer in the treatment of Duchennes muscular dystrophy. N. Engl. J. Med. 333, 832-838.
20. MOR, G., KLINMAN, D.M., SHAPIRO, S., HAGIWARA, E., SEDEGAH, M., NORMAN, J.A., HOFFMAN, S.L., and STEINBERG, A.D. (1995). Complexity of the cytokine and antibody response elicited by immunizing mice with Plasmodium yoelii circumsporozoite protein plasmid DNA. J. Immunol. 155, 2039-2046.
21. MOR, G., SINGLA, M., STEINBERG, A.D., HOFFMAN, S.L., OKUDA, K., and KLINMAN, D.M. (1997). Do DNA vaccines induce autoimmune disease? Hum. Gene Ther. 8, 293-300.
22. NAFFAKH, N., HENRI, A., VILLEVAL, J.L., ROUYER, P., MOULLIER, P., BLUMENFELD, N., DANOS, O., VAINCHENKER, W., HEARD, I.M., and BEUZARD, Y. (1995). Sustained delivery of erythropoietin in mice by genetically modified skin fibroblasts. Proc. Natl. Acad. Sci. U.S.A. 92, 3194-3198.
23. QUANTIN, B., PERRICAUDET, L.D., TAIBAKHSH, J., and MANDEL, J. (1992a). Adenovirus as an expression vector in muscle cells in vivo. Proc. Natl. Acad. Sci. U.S.A. 89, 2581-2584.
24. QUANTIN, B., PERRICAUDET, L.D., TAJBAKHSH, S., and MANDEL, J.L. (1992b). Adenovirus as an expression vector in muscle cells in vivo. Proc. Natl. Acad. Sci. U.S.A. 89, 2581-2584.
25. RAZ, E., WATANABE, A., BAIRD, S.M., EISENBERG, R.A., PARR, T.B., LOTZ, M., KIPPS, T.J., and CARSON, D.A. (1993). Systemic immunological effects of cytokine genes injected into skeletal muscle. Proc. Natl. Acad. Sci. U.S.A. 90, 4523-4527.
26. ROBERTSON, J.S. (1994). Safety considerations for nucleic acid vaccines. Vaccine 12, 1526-1528.
27. SEDEGAH, M., HEDSTROM, R., HOBART, P., and HOFFMAN, S.L. (1994). Protection against malaria by immunization with plasmid DNA encoding circumsporozoite protein. Proc. Natl. Acad. Sci. U.S.A. 91, 9866-9870.
28. SMITH, T.A.G., MEHAFFEY, M.G., KAYDA, D.B., SAUNDERS, J.M., YEI, S., TRAPNELL, B.C., MCCLELLAND, A., and KALEKO, M. (1993). Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice. Nature Genet. 5, 397-402.
29. SNYDER, R.O., SPRATT, S.K., LAGARDE, C., BOHL, D., KASPAR, B., SLOAN, B., COHEN, L.K., and DANOS, O. (1997). Efficient and stable adeno-associated virus-mediated transduction in the skeletal muscle of adult immunocompetent mice. Hum. Gene Ther. 8, 1891-1900.
30. SVENSSON, E.C., BLACK, H.B., DUGGER, D.L., TRIPATHY, S.K., GOLDWASSER, E., HAO, Z., CHU, L., and LEIDEN, J.M. (1997). Long-term erythropoietin expression in rodents and non-human primates following intramuscular injection of a replication-defective adenoviral vector. Hum. Gene Ther. 8, 1797-1806.
31. TANG, B., MATSUDA, T., AKIRA, S., NAGATA, N., IKEHARA, S., HIRANO, T., and KISHIMOTO, T. (1991). Age-associated increase in interleukin 6 in MRL/lpr mice. Int. Immunol. 3, 273-278.
32. TRIPATHY, S.K., BLACK, H.B., GOLDWASSER, E., and LEIDEN, J.M. (1996a). Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 45-550.
33. TRIPATHY, S.K., GOLDWASSER, E., LU, M.M., BARR, E., and LEIDEN, J.M. (1996b). Long term expression of erythropoietin in the systemic circulation of mice after intramuscular injection of a plasmid DNA vector. Proc. Natl. Acad. Sci. U.S.A. 91, 10876-10880.
34. ULMER, J.B., DONNELLY, J.J., PARKER, S.E., RHODES, G.H., FELGNER, P.L., DWARKI, V.J., GROMKOSKI, S.H., DECK, R.R., DEWITT, C.M., and FRIEDMAN, A. (1993). Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 259, 1745-1749. [See comments].
35. WILLIAMS, R.S., JOHNSTON, S.A., RIEDY, M., DEVIT, M.J., MCELLIGOTT, S.G., and SANFORD, J.C. (1991). Introduction of foreign genes into tissues of living mice by DNA-coated microprojectiles. Proc. Natl. Acad. Sci. U.S.A. 88, 2726-2730.
36. WOLFF, J.A., MALONE, R.W., WILLIAMS, P., CHONG, W., ASCADI, G., JANI, A., and FELGNER, P.L. (1990). Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468.
37. WOLFF, J.A., LUDTKE, J.J., ACSADI, G., WILLIMANS, P., and JANI, A. (1992). Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1363, 363-369.
38. YANG, Y., LI, Q., ERTL, H.C., and WILSON, J.M. (1996). Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. U.S.A. 91, 4407-4411.