Enhancer and promoter chimeras in plasmids designed for intramuscular injection: A comparative in vivo and in vitro study

Human Gene Therapy

Volumn 9, Issue 17, 1998, Pages 2545-2553

  Barnhart, Kerry M ^a   Hartikka, Jukka ^a   Manthorpe, Marston ^a   Norman, Jon ^a   Hobart, Peter ^a  

^a VICAL INC   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

LUCIFERASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL DIFFERENTIATION; CELL STRAIN BHK; CHIMERA; ENHANCER REGION; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN CYTOMEGALOVIRUS; KIDNEY CELL; MOUSE; MUSCLE; MYOBLAST; NONHUMAN; PLASMID; PROMOTER REGION; REPORTER GENE; TISSUE SPECIFICITY;

ANIMALS; BASE SEQUENCE; CELL LINE; CHIMERA; CRICETINAE; CYTOMEGALOVIRUS; DNA PRIMERS; ENHANCER ELEMENTS (GENETICS); GENE EXPRESSION; GENES, IMMEDIATE-EARLY; INJECTIONS, INTRAMUSCULAR; LUCIFERASES; MICE; PLASMIDS; PROMOTER REGIONS (GENETICS);

ANIMALIA; CRICETINAE; CUCUMBER MOSAIC VIRUS; DNA VIRUSES; HUMAN HERPESVIRUS 5;

EID: 0032553683     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349850019382     Document Type: Article

References (38)

1. AMACHER, S.L., BUSKIN, J.N., and HAUSCHKA, S.D. (1993). Multiple regulatory elements contribute differentially to muscle creatine kinase enhancer activity in skeletal and cardiac muscle. Mol. Cell. Biol. 13, 2753-2764.
2. BARRY, M.A., LAI, W.C., and JOHNSTON, S.A. (1995). Protection against mycoplasmic infection using expression library immunization. Nature (London) 377, 632-635.
3. BIBEN, C., KIRSCHBAUM, B.J., GARNER, I., and BUCKINGHAM, M. (1994). Novel muscle-specific enhancer sequences upstream of the cardiac actin gene. Mol. Cell. Biol. 14, 3504-3513.
4. CSERJESI, P., LILLY, B., HINKLEY, C., PERRY, M., and OLSON, E. (1994). Homeodomain protectin MHox and MADS protein myocyte enhancer-binding factor-2 converge on a common element in the muscle creatine kinase enhancer. J. Biol. Chem. 269, 16740-16745.
5. DOH, S.G., VAHLSING, H.L., HARTIKKA, J., LIANG, X., and MANTHORPE, M. (1997). Spatial-temporal patterns of gene expression in mouse skeletal muscle after injection of lacZ plasmid DNA. Gene Ther. 4, 648-663.
6. EDMONDSON, D.G., CHENG, T.-C., CSERJESI, P., CHAKRABORTY, T., and OLSON, E.N. (1992). Analysis of the myogenin promoter reveals an indirect pathway for the positive autoregulation mediated by the muscle-specific enhancer factor MEF-2. Mol. Cell. Biol. 12, 3665-3677.
7. EDMONSON, D., and OLSON, E. (1993). Helix-loop-helix proteins as regulators of muscle-specific transcription. J. Biol. Chem. 268, 755-758.
8. FAZIO, V.M., FAZIO, S., RINALDI, M., CATANI, M.V., ZOTTI, S., CIAFRE, S.A., SERIPA, D., RICCI, G., and FARACE, M.G. (1994). Accumulation of human apoliporprotein-E in rat plasma after in vivo intramuscular injection of naked DNA. Biochem. Biophys. Res. Commun. 200, 298-305.
9. FELGNER, J., KUMAR, R., SRIDHAR, C., WHEELER, C., TSAI, Y., BORDER, R., RAMSEY, P., MARTIN, M., and FELGNER, P. (1994). Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269, 2550-2561.
10. FELGNER, P., and RHODES, G. (1991). Gene therapeutics. Nature (London) 349, 351-352.
11. FERRARI, G., SALVATORI, G., ROSSI, C., COSSU, G., and MAVILIO, F. (1995). A retroviral vector containing a muscle-specific enhancer drives gene expression only in differentiated muscle fibers. Hum. Gene Ther. 6, 733-742.
12. GARNER, I., MINTY, A.J., ALONSO, S., BARTON, P.J., and BUCKINGHAM, M.E. (1986). A 5′ duplication of the α-cardiac actin gene in BALB/c mice is associated with abnormal levels of α-cardiac and α-skeletal actin mRNAs in adult cardiac tissue. EMBO J. 5, 2559-2567.
13. GOSSETT, L., KELVIN, D., STERNBERG, E., and OLSON, E. (1989). A new myocyte-specific enhancer-binding factor that recognizes a conserved element associated with multiple muscle-specific genes. Mol. Cell. Biol. 9, 5022-5033.
14. HARTIKKA, J., SAWDEY, M., CORNEFERT-JENSEN, F., MARGALITH, M., BARNHART, K., NOLASCO, M., VAHLSING, 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.
15. HORN, N., MEEK, J., BUDAHAZI, G., and MARQUET, M. (1995). Cancer gene therapy using plasmid DNA: Purification of DNA for human clinical trials. Hum. Gene Ther. 6, 565-573.
16. HU, M., SHARP, S., and DAVIDSON, N. (1986). The complete sequence of the mouse skeletal α-actin gene reveals several conserved and inverted repeat sequences outside of the protein-coding region. Mol. Cell. Biol. 6, 15-25.
17. JAYNES, J.B., JOHNSON, J.E., BUSKIN, J.N., GARTSIDE, C.L., and HAUSCHKA, S.D. (1988). The muscle creatine kinase gene is regulated by multiple upstream elements, including a muscle-specific enhancer. Mol. Cell. Biol. 8, 62-70.
18. LAEMMLI, U., and TJIAN, R. (1996). A nuclear traffic jam - unraveling multicomponent machines and compartments. Curr. Opin. Cell Biol. 8, 299-302.
19. LEDLEY, F.D. (1995). Nonviral gene therapy: The promise of genes as pharmaceutical products. Hum. Gene Ther. 6, 1129-1144.
20. LEVY, M., BARRON, L., MEYER, K., and SZOKA, F. (1996). Characterization of plasmid DNA transfer into mouse skeletal muscle: Evaluation of uptake mechanism, expression and secretion of gene products into blood. Gene Ther. 3, 201-211.
21. LEW, D., PARKER, S., LATIMER, T., ABAI, A., KUWAHARA-RUNDELL, A., DOH, S., YANG, Z., LAFACE, D., GROMKOWSKI, S., NABEL, G., MANTHORPE, M., and NORMAN, J. (1995). Cancer gene therapy using plasmid DNA: Pharmacokinetic study of DNA following injection into mice. Hum. Gene Ther. 6, 553-564.
22. MA, J., YANG, Z., CHAO, J., and CHAO, L. (1995). Intramuscular delivery of rat kallikrein-binding protein reverses hypotension in transgenic mice expressing human tissue kallikrein. J. Biol. Chem. 270, 451-455.
23. 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, 419-4131.
24. MANTHORPE, M., HARTIKKA, J., VAHLSING, H.L., and SAWDEY, M. (1997). Quantification of plasmid DNA expression in vivo. In Gene Quantification. F. Ferre, ed. (Burkauser, Cambridge, MA).
25. MUSCAT, G., and KEDES, L. (1987). Multiple 5′-flanking regions of the human α-skeletal actin gene synergistically modulate muscle-specific expression. Mol. Cell. Biol. 7, 4089-4099.
26. OLSON, E. (1990). The MyoD family, a paradigm for development? Genes Dev. 4, 1454-1461.
27. OLSON, E.N. (1993). Signal transduction pathways that regulate skeletal muscle gene expression. Mol. Endocrinol. 7, 1369-1378.
28. PARKER, S.E., VAHLSING, L.H., SERFILIPPI, L.M., FRANKLIN, C.L., DOH, S.O., GROMKOWSKI, S.H., LEW, D., MANTHORPE, M., and NORMAN, J. (1995). Cancer gene therapy using plasmid DNA: Safety evaluation in rodents and nonhuman primates. Hum. Gene Ther. 6, 575-590.
29. PARKER, S.E., BORELLINI, F., WENK, M.L., HOBART, P., HOFFMAN, S.L., HEDSTROM, R., LE, T., and NORMAN, J.A. (1998). Plasmid DNA malaria vaccine: Tissue distribution and safety studies in mice and rabbits. Submitted.
30. PARMACEK, M., IP, H., JUNG, F., SHEN, T., MARTIN, J., VORA, A., OLSON, E., and LEIDEN, J. (1994). A novel myogenic regulatory circuit controls slow/cardiac troponin C gene transcription in skeletal muscle. Mol. Cell. Biol. 14, 1870-1885.
31. 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.
32. SKARLI, M., KIRI, A., VRBOVA, G., LEE, C., and GOLDSPINK, G. (1998). Myosin regulatory elements as vectors for gene transfer by intramuscular injection. Gene Ther. 5, 514-520.
33. TJIAN, R. (1996). The biochemistry of transcription in eukaryotes: A paradigm for multisubunit regulatory complexes. Philos. Trans. R. Soc. Lond. B Biol. Sci. 351, 491-499.
34. TRIPATHY, S., SVENSSON, E., BLACK, H., GOLDWASSER, E., MARGALITH, M., HOBART, P., and LEIDEN, J. (1996). 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. 93, 10876-10880.
35. ULMER, J.B., DONNELLY, J.J., PARKER, S.E., RHODES, G.H., FELGNER, P.L., DWARKI, V.J., GROMKOWSKI, S.H., DECK, R.R., DEWITT, C.M., FRIEDMAN, A., and LIU, M. (1993). Heterologous protection against influenza by injection of DNA encoding a viral protein. Science 259, 1745-1749.
36. VERMA, I., and SOMIA, N. (1997). Gene therapy - promises, problems, and prospects. Nature (London) 389, 239-242.
37. YEW, N., WYSOKENSKI, D., WANG, K., ZIEGLER, R., MARSHALL, J., MCNEILLY, D., CHERRY, M., OSBURN, W., and CHENG, S. (1997). Optimization of plasmid vectors for high-level expression in lung epithelial cells. Hum. Gene Ther. 8, 575-584.
38. ZAWEL, L., and REINBERG, D. (1995). Common themes in assembly and function of eukaryotic transcription complexes. Annu. Rev. Biochem. 64, 533-561.