Use of poly(N-vinyl pyrrolidone) with noncondensed plasmid DNA formulations for gene therapy and vaccines

Polymeric Gene Delivery: Principles and Applications

Volumn , Issue , 2004, Pages 329-344

  Nicolaou, Michael ^a   Chang, Polly ^b   Newman, Mark J ^a  

^a Epimmune   (United States)

^b SRI INTERNATIONAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

EID: 84923346889     PISSN: None     EISSN: None     Source Type: Book    
DOI: 10.1201/9780203500477     Document Type: Chapter

References (76)

1. Markovitz, N.S. and Roizman, B., Replication-competent herpes simplex viral vectors for cancer therapy, in Advances in Virus Research, Maramorosch, K., Murphy, F.A., and Shatkin, A.J. (Eds.), Academic Press, New York, NY, 2000, 409.
2. Hitt, M. and Graham, F.L., Adenovirus vectors for human gene therapy, in Advances in Virus Research, Maramorosch, K., Murphy, F.A., and Shatkin, A.J. (Eds.), Academic Press, New York, NY, 2000, 479.
3. Schlesinger, S., Alphavirus expression vectors, in Advances in Virus Research, Maramorosch, K., Murphy, F.A., and Shatkin, A.J. (Eds.), Academic Press, New York, NY, 2000, 565.
4. Cairns, J.S. and Sarver, N., New viral vectors for HIV vaccine delivery, AIDS Res. Hum. Retrovir. 14, 1501, 1998.
5. Voltan, R. and Robert-Guroff, M., Live recombinant vectors for AIDS vaccine development, Curr. Mol. Med. 3, 273, 2003.
6. Davis, H.L., Michel, M.L., Mancini, M., Schleef, M., and Whalen, R.G., Direct gene transfer in skeletal muscle: plasmid DNA-based immunization against the hepatitis B virus surface antigen, Vaccine 12, 1503, 1994.
7. Robinson, H.L. and Pertmer, T.M., DNA vaccines for viral infections: basic studies and applications, in Advances in Virus Research, Maramorosch, K., Murphy, F.A., and Shatkin, A.J. (Eds.), Academic Press, New York, NY, 2000, 1.
8. Weiner, D. B. and Kennedy, R. C. Genetic vaccines, Sci. Am. 281, 50, 1999.
9. Donnelly, J.J., Ulmer, J.B., and Liu, M.A., Immunization with DNA, J. Immunol. Methods 176, 145, 1994.
10. Moelling, K., DNA for genetic vaccination and therapy, Cytokines Cell Mol. Ther. 3, 127, 1997.
11. Wang, R., Doolan, D., Le, T., Hedstrom, R., Coonan, K., Charoenvit, Y., Jones, T., Hobart, P., Margalith, M., Ng, J., Weiss, W., Sedegah, M., De Taisne, C., Norman, J., and Hoffman, S., Induction of antigen-specific cytotoxic T lymphocytes in humans by a malaria DNA vaccine, Science 282, 476, 1998.
12. Le, T.P., Coonan, K.M., Hedstrom, R.C., Charoenvit, Y., Sedegah, M., Epstein, J.E., Kumar, S., Wang, R., Doolan, D.L., Maguire, J.D., Parker, S.E., Hobart, P., Norman, J., and Hoffman, S.L., Safety, tolerability and humoral immune responses after intramuscular administration of a malaria DNA vaccine to healthy adult volunteers, Vaccine 18, 1893, 2000.
13. Wang, R., Epstein, J., Baraceros, F.M., Gorak, E.J., Charoenvit, Y., Carucci, D.J., Hedstrom, R.C., Rahardjo, N., Gay, T., Hobart, P., Stout, R., Jones, T.R., Richie, T.L., Parker, S.E., Doolan, D.L., Norman, J., and Hoffman, S.L., Induction of CD4(+) T cell-dependent CD8(+) type 1 responses in humans by a malaria DNA vaccine, Proc. Nat. Acad. Sci. U.S.A. 98, 10817, 2001.
14. Boyer, J.D., Chattergoon, M.A., Ugen, K.E., Shah, A., Bennett, M., Cohen, A., Nyland, S., Lacy, K.E., Bagarazzi, M.L., Higgins, T.J., Baine, Y., Ciccarelli, R.B., Ginsberg, R.S., MacGregor, R.R., and Weiner, D.B., Enhancement of cellular immune response in HIV-1 seropositive individuals: a DNA-based trial, Clin. Immunol. 90, 100, 1999.
15. MacGregor, R.R., Boyer, J., Ugen, K., Lacy, K., Gluckman, S., Bagarazzi, M., Chattergoon, M., Baine, Y., Higgins, T., Ciccarelli, R., Coney, L., Ginsberg, R.S., and Weiner, D., First human trial of a DNAbased vaccine for treatment of Human Immunodeficiency Virus Type 1 infection: safety and host response, J. Infect. Dis. 178, 92, 1998.
16. Boyer, J.D., Cohen, A.D., Vogt, S., Schumann, K., Nath, B., Ahn, L., Lacy, K., Bagarazzi, M.L., Higgins, T.J., Baine, Y., Ciccarelli, R.B., Ginsberg, R.S., MacGregor, R.R., and Weiner, D.B., Vaccination of seronegative volunteers with a Human Immunodeficiency Virus Type 1 env/rev DNA vaccine induces antigen-specific proliferation and lymphocyte production of β-chemokines, J. Infect. Dis. 181, 476, 2000.
17. Calarota, S., Leandersson, A.C., Bratt, G., Hinkula, J., Klinman, D.M., Weinhold, K.J., Sandström, E., and Wahren, B., Immune responses in asymptomatic HIV-1-infected patients after HIV-DNA immunization followed by highly active antiretroviral treatment, J. Immunol. 163, 2330, 1999.
18. Rolland, A.P., From genes to gene medicines: recent advances in nonviral gene delivery, Crit. Rev. Ther. Drug Carrier Syst. 15, 143, 1998.
19. Mahato, R.I., Rolland, A.P., and Tomlinson, E., Cationic lipid-based gene delivery systems: pharmaceutical perspectives, Pharm. Res. 14, 853-859, 1997.
20. Felgner, P.L., Tsai, Y.J., Sukhu, L., Wheeler, C.J., Manthrope, M., Marshall, J.A., and Cheng, S.H., Improved cationic lipid formulations for in vivo gene therapy, Ann. NY Acad. Sci. 27, 126, 1995.
21. O’Hagan, D., Singh, M., Ugozzoli, M., Wild, C., Barnett, S., Chen, M., Schaefer, M., Doe, B., Otten, G.R., and Ulmer, J.B., Induction of potent immune responses by cationic microparticles with adsorbed Human Immunodeficiency Virus DNA vaccines, J. Virol. 75, 9037, 2001.
22. Fu, T.M., Guan, L., Friedman, A., Ulmer, J.B., Liu, M.A., and Donnelly, J.J., Induction of MHC class I-restricted CTL response by DNA immunization with ubiquitin-influenza virus nucleoprotein fusion antigens, Vaccine 16, 1711, 1998.
23. Yokoyama, M., Zhang, J., and Whitton, J.L., DNA immunization: Effects of vehicle and route of administration on the induction of protective antiviral immunity, FEMS Immunol. Med. Microbiol. 14, 221, 1996.
24. Rodriguez, F., Harkins, S., Redwine, J.M., de Pereda, J.M., and Whitton, J.L., CD4(+) T cells induced by a DNA vaccine: immunological consequences of epitope-specific lysosomal targeting, J. Virol. 75, 10421, 2001.
25. Whitton, J.L., Rodriguez, F., Zhang, J., and Hassett, D.E., DNA immunization: mechanistic studies, Vaccine 17, 1612, 1999.
26. Kim, J.J., Trivedi, N.N., Nottingham, L.K., Morrison, L., Tsai, A., Hu, Y., Mahalingam, S., Dang, K.S., Ahn, L., Doyle, N.K., Wilson, D.M., Chattergoon, M.A., Chalian, A.A., Boyer, J.D., Agadjanyan, M.G., and Weiner, D.B., Modulation of amplitude and direction of in vivo immune responses by coadministration of cytokine gene expression cassettes with DNA immunogens, Eur. J. Immunol. 28, 1089, 1998.
27. Sin, J.I. and Weiner, D.B., Improving DNA vaccines targeting viral infection, Intervirology 43, 233, 2000.
28. Boyer, J.D., Chattergoon, M., Muthumani, K., Kudchodkar, S., Kim, J., Bagarazzi, M., Pavlakis, G., Sekaly, R., and Weiner, D.B., Next generation DNA vaccines for HIV-1, J. Liposome Res. 12, 137, 2002.
29. Legendre, J.Y. and Szoka, F.C., Jr., Delivery of plasmid DNA into mammalian cell lines using pHsensitive liposomes: comparison with cationic liposomes, Pharm. Res. 9, 1235, 1992.
30. Freimark, B.D., Blezinger, H.P., Florack, V.J., Nordstrom, J.L., Long, S.D., Deshpande, D.S., Nochumson, S., and Petrak, K.L., Cationic lipids enhance cytokine and cell influx levels in the lung following administration of plasmid: cationic lipid complexes, J. Immunol. 160, 4580, 1998.
31. Mahato, R.I., Monera, O.D., Smith, L.C., and Rolland, A.P., Peptide-based gene delivery, Curr. Top. Microbiol. Immunol. 1, 226, 1999.
32. MacLaughlin, F.C., Mumper, R.J., Wang, J., Tagliaferri, J.M., Gill, In., Hinchcliffe, M., and Rolland, A.P., Chitosan and depolymerized chitosan oligomers as condensing carriers for in vivo plasmid delivery, J. Control. Release 56, 259, 1998.
33. Kaneda, Y., Iwai, K., and Uchida, T., Increased expression of DNA cointroduced with nuclear protein in adult rat liver, Science 243, 375, 1989.
34. Kato, K., Nakanishi, M., Kaneda, Y., Uchida, T., and Okada, Y., Expression of hepatitis B virus surface antigen in adult rat liver. Co-introduction of DNA and nuclear protein by a simplified liposome method, J. Biol. Chem. 266, 3361, 1991.
35. Lunsford, L., McKeever, U., Eckstein, V., and Hedley, M.L., Tissue distribution and persistence in mice of plasmid DNA encapsulated in a PLGA-based microsphere delivery vehicle, J. Drug Target. 8, 39, 2000.
36. Hao, T., McKeever, U., and Hedley, M.L., Biological potency of microsphere encapsulated plasmid DNA, J. Control. Release 69, 249, 2000.
37. Hedley, M.L., Curley, J., and Urban, R., Microspheres containing plasmid-encoded antigens elicit cytotoxic T-cell responses. Nat. Med. 4, 365, 1998.
38. McKeever, U., Barman, S., Hao, T., Chambers, P., Song, S., Lunsford, L., Hsu, Y.Y., Roy, K., and Hedley, M.L., Protective immune responses elicited in mice by immunization with formulations of poly(lactide-co-glycolide) microparticles. Vaccine 20, 1524, 2002.
39. Sheets, E.E., Urban, R.G., Crum, C.P., Hedley, M.L., Politch, J.A., Gold, M.A., Muderspach, L.I., Cole, G.A., and Crowley-Nowick, P.A., Immunotherapy of human cervical high-grade cervical intraepithelial neoplasia with microparticle-delivered human papillomavirus 16 E7 plasmid DNA, Am. J. Obstet. Gynecol. 188, 916, 2003.
40. Mumper, R.J., Duguid, J.G., Anwer, K., Barron, M.K., Nitta, H., and Rolland, A.P., Polyvinyl derivatives as novel interactive polymers for controlled gene delivery to muscle, Pharm. Res. 13, 701, 1996.
41. Mumper, R.J., Wang, J., Klakamp, S.L., Nitta, H., Anwer, K., Tagliaferri, F., and Rolland, A.P., Protective interactive noncondensing (PINC) polymers for enhanced plasmid distribution and expression in rat skeletal muscle, J. Control. Release 52, 191, 1998.
42. Alila, H., Coleman, M.E., Nitta, H., French, M., Anwer, K., Liu, Q., Meyer, T., Wang, J., Mumper, R.J., Oubari, D., Long, S.D., Nordstrom, J.L., and Rolland, A.P., Expression of biologically active human insulin-like growth factor-I following intramuscular injection of a formulated plasmid in rats, Hum. Gene Ther. 8, 1785, 1997.
43. Coleman, M., Muller, S., Quezada, A., Mendiratta, S.K., Wang, J., Thull, N.M., Bishop, J., Matar, M., Mester, J., and Pericle, F., Nonviral interferon alpha gene therapy inhibits growth of established tumors by eliciting a systemic immune response, Hum. Gene Ther. 9, 2223, 1998.
44. Bishop, J.S., Thull, N.M., Matar, M., Quezada, A., Munger, W.E., Batten, T.L., Muller, S., and Pericle, F., Antitumoral effect of a nonviral interleukin-2 gene therapy is enhanced by combination with 5- fluorouracil, Cancer Gene Ther. 7, 1165, 2000.
45. Mendiratta, S.K., Quezada, A., Matar, M., Wang, J., Hebel, H.L., Long, S., Nordstrom, J.L., and Pericle, F., Intratumoral delivery of IL-12 gene by polyvinyl polymeric vector system to murine renal and colon carcinoma results in potent antitumor immunity, Gene Ther. 6, 833, 1999.
46. Mendiratta, S.K., Quezada, A., Matar, M., Thull, N.M., Bishop, J.S., Nordstrom, J.L., and Pericle, F., Combination of interleukin 12 and interferon alpha gene therapy induces a synergistic antitumor response against colon and renal cell carcinoma, Hum. Gene Ther. 11, 1851, 2000.
47. Anwer, K., Earle, K.A., Shi, M., Wang, J., Mumper, R.J., Proctor, B., Jansa, K., Ledebur, H.C., Davis, S.S., Eaglstein, W., and Rolland, A.P., Synergistic effect of formulated plasmid and needle-free injection for genetic vaccines, Pharm. Res. 16, 889, 1999.
48. Townsend, A. and Bodmer, H., Antigen recognition by Class I-restricted T lymphocytes, Ann. Rev. Immunol. 7, 601, 1989.
49. Germain, R.N. and Margulies, D.H., The biochemistry and cell biology of antigen processing and presentation, Ann. Rev. Immunol. 11, 403, 1993.
50. Bartlett, J.A., Wasserman, S.S., Hicks, C.B., Dodge, R.T., Weinhold, K.J., Tacket, C.O., Ketter, N., Wittek, A.E., Palker, T.J., and Haynes, B.F., Safety and immunogenicity of an HLA-based HIV envelope polyvalent synthetic peptide immunogen, DATRI 010 Study Group, Division of AIDS Treatment Research Initiative, AIDS 12, 1291, 1998.
51. Pinto, L.A., Berzofsky, J.A., Fowke, K.R., Little, R.F., Merced-Galindez, F., Humphrey, R., Ahlers, J., Dunlop, N., Cohen, R.B., Steinberg, S.M., Nara, P., Shearer, G.M., and Yarchoan, R., HIV-specific immunity following immunization with HIV synthetic envelope peptides in asymptomatic HIVinfected patients, AIDS 13, 2003, 1999.
52. Gahéry-Ségard, H., Pialoux, G., Charmeteau, B., Sermet, S., Poncelet, H., Raux, M., Tartar, A., Lévy, J.P. Gras-Masse, H., and Guillet, J.G., Multiepitopic B- and T-cell responses induced in humans by a Human Immunodeficiency Virus Type 1 lipopeptide vaccine, J. Virol. 74, 1694, 2000.
53. An, L.L., Rodriguez, F., Harkins, S., Zhang, J., and Whitton, J.L., Quantitative and qualitative analyses of the immune responses induced by a multivalent minigene DNA vaccine, Vaccine 18, 2132, 2000.
54. An, L.L. and Whitton, J.L., Multivalent minigene vaccines against infectious disease, Curr. Opin. Mol. Ther. 1, 16, 1999.
55. Hanke, T., Schneider, J., Gilbert, S.C., Hill, A.V.S., and McMichael, A., DNA multi-CTL epitope vaccines for HIV and Plasmodium falciparum: immunogenicity in mice, Vaccine 16, 426, 1998.
56. Hanke, T., Neumann, V.C., Blanchard, T.K., Sweeney, P., Hill, A.V.S., Smith, G.L., and McMichael, A., Effective induction of HIV-specific CTL by multi-epitope using gene gun in a combined vaccination regime, Vaccine 17, 589, 1999.
57. Ishioka, G.Y., Fikes, J., Hermanson, G., Livingston, B., Crimi, C., Qin, M., del Guercio, M.F., Oseroff, C., Dahlberg, C., Alexander, J., Chesnut, R.W., and Sette, A., Utilization of MHC Class I transgenic mice for development of minigene DNA vaccines encoding multiple HLA-restricted CTL epitopes, J. Immunol. 162, 3915, 1999.
58. Livingston, B.D., Newman, M., Crimi, C., McKinney, D., Chesnut, R., and Sette, A., Optimization of epitope processing enhances immunogenicity of multi-epitope DNA vaccines, Vaccine 19, 4652, 2001.
59. Livingston, B., Crimi, C., Newman, M., Higashimoto, Y., Appella, E., Sidney, J., and Sette, A.A., Rational strategy to design multi-epitope immunogens based on multiple Th-lymphocyte epitopes, J. Immunol. 168, 5499, 2002.
60. Alexander, J., Sidney, J., Southwood, S., Ruppert, J., Oseroff, C., Maewal, A., Snoke, K., Serra, H.M., Kubo, R.T., and Sette, A., Development of high potency universal DR-restricted helper epitopes by modification of high affinity DR-blocking peptides, Immunity 1, 751, 1994.
61. Wilson, C., McKinney, D.M., Anders, M., MaWhinney, S., Forster, J., Crimi, C., Southwood, S., Sette, A., Chesnut, R., Newman, M., and Livingston, B., Development of a DNA vaccine designed to induce cytotoxic t lymphocyte responses to multiple conserved epitopes in HIV-1, J. Immunol., 171, 5611-5623, 2003.
62. Robinson, B.V., Sullivan, F.M., Borzelleca, J.F., and Schwartz, S.L., PVP: A Critical Review of the Kinetics and Toxicology of Polyvinylpyrrolidone (Povidone), Lewis Publishers, 1990, 1.
63. Quezada, A., Horner, M.J., Loera, D., French, M., Pericle, F., Johnson, R., Perrard, J., Jenkins, M., and Coleman, M., Safety toxicity study of plasmid-based IL-12 therapy in Cynomolgus monkeys, J. Pharm. Pharmacol. 54, 241, 2002.
64. Martin, T., Parker, S.E., Hedstrom, R., Le, T., Hoffman, S.L., Norman, J., Hobart, P., and Lew, D., Plasmid DNA malaria vaccine: the potential for genomic integration after intramuscular injection, Hum. Gene Ther. 10, 759, 1999.
65. Kawabata, K., Takakura, Y., and Hashida, M., The fate of plasmid DNA after intravenous injection in mice: involvement of scavenger receptors in its hepatic uptake, Pharm. Res. 12, 825, 1995.
66. Ledwith, B.J., Manam, S., Troilo, P.J., Barnum, A.B., Pauley, C.J., Griffiths, T.G., Harper, L.B., Beare, C.M., Bagdon, W.J., and Nichols, W.W., Plasmid DNA vaccines: investigation of integration into host cellular DNA following intramuscular injection in mice, Intervirology 43, 258, 2000.
67. Parker, S.E., Borellini, F., Wenk, M.L., Hobart, P., Hoffman, S.L., Hedstrom, R., Le, T., and Norman, J.A., Plasmid DNA malaria vaccine: tissue distribution and safety studies in mice and rabbits, Hum. Gene Ther. 10, 741, 1999.
68. Winegar, R.A., Monforte, J.A., Suing, K.D., O’Loughlin, K.G., Rudd, C.J., and Macgregor, J.T., Determination of tissue distribution of an intramuscular plasmid vaccine using PCR and in situ DNA hybridization, Hum. Gene Ther. 7, 2185, 1996.
69. Bagarazzi, M.L., Boyer, J.D., Ugen, K.E., Javadian, M.A., Chattergoon, M., Shah, A., Bennett, M., Ciccarelli, R., Carrano, R., Coney, L., and Weiner, D.B., Safety and immunogenicity of HIV-1 DNA constructs in chimpanzees, Vaccine 16, 1836, 1998.
70. Haworth, R. and Pilling, A.M., The PCR assay in the preclinical safety evaluation of nucleic acid medicines, Hum. Exp. Toxicol. 19, 267, 2000.
71. Winegar, R.A., Monforte, J.A., Suing, K.D., O’Loughlin, K.G., Rudd, C.J., and Macgregor, J.T., Determination of tissue distribution of an intramuscular plasmid vaccine using PCR and in situ DNA hybridization, Hum. Gene Ther. 7, 2185, 1996.
72. Martin, T., Parker, S.E., Hedstrom, R.C., Le, T., Hoffman, S.L., Norman, J., Hobart, P., and Lew, D., Plasmid DNA malaria vaccine: the potential for genomic integration after intramuscular injection, Hum. Gene Ther. 10, 759, 1999.
73. Ledwith, B.J., Manam, S., Troilo, P.J., Barnum, A.B., Pauley, C.J., Griffiths, T.G., Harper, L.B., Schock, H.B., Zhang, H., Faris, J.E., Way, P.A., Beare, C.M., Bagdon, W.J., and Nichols, W.W., Plasmid DNA vaccines: assay for integration into host genomic DNA, Dev. Biol. 104, 33, 2000.
74. Manam, S., Ledwith, B.J., Barnum, A.B., Troilo, P.J., Pauley, C.J., Harper, L.B., Griffiths II, T.G., Niu, Z., Denisova, L., Follmer, T.T., Pacchione, S.J., Wang, Z., Beare, C.M., Bagdon, W.J., and Nichols, W.W., Plasmid DNA vaccines: tissue distribution and effects of DNA sequence, adjuvants and delivery method on integration into host DNA, Intervirology 43, 273, 2000.
75. Payette, P.J., Weeratna, R.D., McCluskie, M.J., and Davis, H.L., Immune-mediated destruction of transfected myocytes following DNA vaccination occurs via multiple mechanisms, Gene Ther. 8, 1395, 2001.
76. McKinney, D.M., Skvoretz, R., Qin, M.S., Ishioka, G., and Sette, A., Characterization of an in situ IFN-γ ELISA assay which is able to detect specific peptide responses from freshly isolated splenocytes induced by DNA minigene immunization, J. Immunol. Methods 237, 105, 2000.