Improved antibiotic-free plasmid vector design by incorporation of transient expression enhancers

Gene Therapy

Volumn 18, Issue 4, 2011, Pages 334-343

  Luke, J M ^a   Vincent, J M ^a   Du, S X ^b,d   Gerdemann, U ^c   Leen, A M ^c   Whalen, R G ^b,e   Hodgson, C P ^a   Williams, J A ^a  

^a NATURE TECHNOLOGY CORPORATION   (United States)

^b MAXYGEN INC   (United States)

^c CENTER FOR CELL AND GENE THERAPY   (United States)

^d Jiangsu Simcere Pharmaceutical Research Institute   (China)

^e Altravax Inc   (United States)

Author keywords

antibiotic free; DNA vaccination; HIV; non viral vector; plasmid

Indexed keywords

ANTIBIOTIC AGENT; DNA VACCINE; GLYCOPROTEIN GP 120; MESSENGER RNA; PLASMID VECTOR; PROTEIN KINASE R; VIRUS RNA;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CYTOMEGALOVIRUS; ELECTROPORATION; EXPRESSION VECTOR; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; PROMOTER REGION; RABBIT; RNA TRANSLATION; SIMIAN VIRUS 40; TRANSIENT EXPRESSION;

ANIMALS; CELL LINE; EIF-2 KINASE; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; PLASMIDS; RNA, MESSENGER; TRANSGENES;

CYTOMEGALOVIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN T-LYMPHOTROPIC VIRUS 1; MAMMALIA; ORYCTOLAGUS CUNICULUS;

EID: 79953777976     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.149     Document Type: Article

References (49)

1. Liu L, Marti GP, Wei X, Zhang X, Zhang H, Liu YV et al. Age-dependent impairment of HIF-1a expression in diabetic mice: correction with electroporation-facilitated gene therapy increases wound healing, angiogenesis, and circulating angiogenic cells. J Cell Physiol 2008; 217: 319-327.
2. Bodles-Brakhop AM, Heller R, Draghia-Akli R. Electroporation for the delivery of DNA-based vaccines and immunotherapeutics: current clinical developments. Mol Ther 2009; 17: 585-592.
3. Luke J, Carnes AE, Hodgson CP, Williams JA. Improved antibiotic-free DNA vaccine vectors utilizing a novel RNA based plasmid selection system. Vaccine 2009; 27: 6454-6459.
4. Williams JA, Luke J, Johnson L, Hodgson CP. pDNAVACCultra vector family: high throughput intracellular targeting DNA vaccine plasmids. Vaccine 2006; 24: 4671-4676. (Pubitemid 43776763)
5. Williams JA, Carnes AE, Hodgson CP. Plasmid DNA vector design; impact on efficacy, safety and upstream production. Biotechnol Adv 2009; 27: 353-370.
6. Williams JA, Luke J, Langtry S, Anderson S, Hodgson CP, Carnes AE. Generic plasmid DNA production platform incorporating low metabolic burden seed-stock and fed-batch fermentation processes. Biotechnol Bioeng 2009; 103: 1129-1143.
7. Carnes AE, Williams JA. Low metabolic burden plasmid production. Genetic Eng Biotech News 2009; 29: 56-57.
8. Carnes AE, Hodgson CP, Luke J, Vincent J, Williams JA. Plasmid DNA production combining antibiotic-free selection, inducible high yield fermentation, and novel autolytic purification. Biotechnol Bioeng 2009; 104: 505-515.
9. FDA. Guidance for Industry: Considerations for Plasmid DNA Vaccines for Infectious Disease Indications 2007: US Food and Drug Administration: Rockville, MD, USA.
10. EMA. Non-clinical Studies Required before First Clinical use of Gene Therapy Medicinal Products 2008: European Medicines Agency: London, England.
11. Wang Z, Troilo PJ, Wang X, Griffiths TG, Pacchione SJ, Barnum AB et al. Detection of integration of plasmid DNA into host genomic DNA following intramuscular injection and electroporation. Gene Ther 2004; 11: 711-721. (Pubitemid 38519279)
12. Van Hall T, Van de Rhee NE, Schoenberger SP, Vierboom MP, Verreck FA, Melief CJ et al. Cryptic open reading frames in plasmid vector backbone sequences can provide highly immunogenic cytotoxic T-lymphocyte epitopes. Cancer Res 1998; 58: 3087-3093. (Pubitemid 28335091)
13. Schirmbeck R, Riedl P, Fissolo N, Lemonnier FA, Bertoleti A, Reimann J. Translation from cryptic reading frames of DNA vaccines generates an extended repertoire of immunogenic, MHC class I-restricted epitopes. J Immunol 2005; 174: 4647-4656.
14. Maness NJ, Wilson NA, Reed JS, Piaskowski SM, Sacha JB, Walsh AD et al. Robust, vaccine-induced CD8+ T lymphocyte response against an out-of-frame epitope. J Immunol 2010; 184: 67-72.
15. Yuh CH, Ting LP. The genome of hepatitis B virus contains a second enhancer: cooperation of two elements within this enhancer is required for its function. J Virol 1990; 64: 4281-4287.
16. Mariati Ho SC, Yap MG, Yang Y. Evaluating post-transcriptional regulatory elements for enhancing transient gene expression levels in CHO K1 and HEK293. Protein Expr Purif 2010; 69: 9-15.
17. Takebe Y, Seiki M, Fujisawa J, Hoy P, Yokata K, Arai K et al. SR alpha promoter: an efficient and versatile mammalian cDNA expression system composed of the simian virus 40 early promoter and the R-U5 segment of human T-cell leukemia virus type 1 long terminal repeat. Mol Cell Biol 1988; 8: 466-472. (Pubitemid 18015592)
18. Barouch DH, Yang ZY, Kong WP, Korioth-Schmitz B, Sumida SM, Truitt DM et al. A human T-cell leukemia virus type 1 regulatory element enhances the immunogenicity of human immunodeficiency virus type 1 DNA vaccines in mice and nonhuman primates. J Virol 2005; 79: 8828-8834. (Pubitemid 40934793)
19. Williams BR. PKR; a sentinel kinase for cellular stress. Oncogene 1999; 18: 6112-6120.
20. Garcia MA, Gil J, Ventoso I, Guerra S, Domingo E, Rivas C et al. Impact of protein kinase PKR in cell biology: from antiviral to antiproliferative action. Microbiol Mol Biol Rev 2006; 70: 1032-1060. (Pubitemid 44958263)
21. Lee ES, Yoon CH, Kim YS, Bae YS. The double-strand RNA-dependent protein kinase PKR plays a significant role in a sustained ER stress-induced apoptosis. FEBS Lett 2007; 581: 4325-4332.
22. Groskreutz D, Schenborn E. Increased gene expression in mammalian cell lines using pAdVAntage DNA as a cotransfectant. Promega Notes 1994; 48: 8-13.
23. Lei M, Liu Y, Samuel CE. Adenovirus VAI RNA antagonizes the RNA-editing activity of the ADAR adenosine deaminase. Virology 1998; 245: 188-196. (Pubitemid 28394181)
24. Andersson MG, Haasnoot PCJ, Xu N, Berenjian S, Berkhout B, Akusjarvi G. Suppression of RNA interference by adenovirus virus-associated RNA. J Virol 2005; 79: 9556-9565. (Pubitemid 41022298)
25. Lu S, Cullen BR. Adenovirus VA1 noncoding RNA can inhibit small interfering RNA and microRNA biogenesis. J Virol 2004; 78: 12868-12876. (Pubitemid 39507795)
26. Ma Y, Mathews MB. Secondary and tertiary structure in the central domain of adenovirus type 2 VA RNA I. RNA 1996; 2: 937-951.
27. Rahman A, Malhotra P, Shar R, Kewalramani T, Thimmapaya B. Effect of single-base substitutions in the central domain of virus-associated RNA I on its function. J Virol 1995; 69: 4299-4307.
28. Lam AP, Dean DA. Progress and prospects: nuclear import of nonviral vectors. Gene Ther 2010; 61: 603-613.
29. Wagstaff KM, Jans DA. Nucleocytoplasmic transport of DNA: enhancing non-viral gene transfer. Biochem J 2007; 406: 185-202. (Pubitemid 47310949)
30. Miller AM, Munkonge FM, Alton EW, Dean DA. Identification of protein cofactors necessary for sequence-specific plasmid DNA nuclear import. Mol Ther 2009; 17: 1897-1903.
31. Li S, MacLaughlin FC, Fewell JG, Gondo M, Wang J, Nicol F et al. Muscle-specific enhancement of gene expression by incorporation of SV40 enhancer in the expression plasmid. Gene Ther 2001; 8: 494-497. (Pubitemid 32266405)
32. Blomberg P, Eskandarpour M, Xia S, Sylven C, Islam KB. Electroporation in combination with a plasmid vector containing SV40 enhancer elements results in increased and persistent gene expression in mouse muscle. Biochem Biophys Res Commun 2002; 298: 505-510. (Pubitemid 35333687)
33. Williams JA. Vectors and methods for genetic immunization. World Patent Application 2006; WO2006078979.
34. Angulo A, Kerry D, Huang H, Borst EM, Razinsky A, Wu J et al. Identification of a boundary domain adjacent to the potent human cytomegalovirus enhancer that represses transcription from the divergent UL127 promoter. J Virol 2000; 74: 2826-2839. (Pubitemid 30117594)
35. Chao SH, Harada JN, Hyndman F, Gao X, Nelson CG, Chanda SK et al. PDX1, a cellular homeoprotein, binds to and regulates the activity of human cytomegalovirus immediate early promoter. J Biol Chem 2004; 279: 16111-16120. (Pubitemid 38509302)
36. Lee J, Klase Z, Gao X, Caldwell JS, Stinski MJ, Kashanchi F et al. Cellular homeo-proteins, SATB1 and CDP, bind to the unique region between the human cytomegalo-virus UL127 and major immediate-early genes. Virology 2007; 366: 117-125. (Pubitemid 47314404)
37. Mesika A, Grigoreva I, Zohar M, Reich Z. A regulated NFkB-assisted import of plasmid DNA into mammalian cell nuclei. Mol Ther 2001; 3: 653-657. (Pubitemid 32509861)
38. Munkonge FM, Amin V, Hyde SC, Green AM, Pringle IA, Gill DR et al. Identification and functional characterization of cytoplasmic determinants of plasmid DNA nuclear import. J Biol Chem 2009; 284: 26978-26987.
39. Hilton TL, Li Y, Dunphy EL, Wang EH. TAF1 histone acetyltransferase activity in Sp1 activation of the cyclin D1 promoter. Mol Cell Biol 2005; 25: 4321-4332. (Pubitemid 40617676)
40. Thakur RK, Kumar P, Halder K, Verma A, Kar A, Parent JL et al. Metastases suppressor NM23-H2 interaction with G-quadruplex DNA within c-MYC promoter nuclease hypersensitive element induces c-MYC expression. Nucleic Acids Res 2009; 37: 172-183.
41. Carnes AE, Luke JM, Vincent JM, Anderson S, Schukar A, Hodgson CP et al. Critical design criteria for minimal antibiotic-free plasmid vectors necessary to combine robust RNA Pol II and Pol III-mediated eukaryotic expression with high bacterial production yields. J Gene Med 2010; 12: 818-831.
42. Kaufman RJ, Murtha P. Translational control mediated by eukaryotic initiation factor-2 is restricted to specific mRNAs in transfected cells. Mol Cell Biol 1987; 7: 1568-1571. (Pubitemid 17045251)
43. Terenzi F, deVeer MJ, Ying H, Restifo NP, Williams BR, Silverman RH. The antiviral enzymes PKR and RNase L suppress gene expression from viral and non-viral based vectors. Nucleic Acids Res 1999; 27: 4369-4375. (Pubitemid 29533176)
44. Breuzard G, Tertil M, Goncalves C, Cheradame H, Geguan P, Pichon C et al. Nuclear delivery of NFB-assisted DNA/polymer complexes: plasmid DNA quantification by confocal laser scanning microscopy and evidence of nuclear polyplexes by FRET imaging. Nucleic Acids Res 2008; 36: e71.
45. Molnar MJ, Gilbert R, Lu Y, Liu AB, Guo A, Larochelle N et al. Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles. Mol Ther 2004; 10: 447-455. (Pubitemid 39186108)
46. Williams JA. Vectors and methods for genetic immunization. World Patent Application 2008; WO2008153733.
47. Lednicky J, Folk WR. Two synthetic Sp1-binding sites functionally substitute for the 21-base-pair repeat region to activate simian virus 40 growth in CV-1 cells. J Virol 1992; 66: 6379-6390.
48. Du SX, Idiart RJ, Mariano EB, Chen H, Jiang P, Xu L et al. Effect of trimerization motifs on quaternary structure, antigenicity, and immunogenicity of a noncleavable HIV-1 gp120 envelope glycoprotein. Virology 2009; 395: 33-44.
49. Gerdemann U, Christin AS, Vera JF, Ramos CA, Fujita Y, Liu H et al. Nucleofection of DCs to generate multivirus-specific T cells for prevention or treatment of viral infections in the immunocompromised host. Mol Ther 2009; 17: 1616-1625.