Towards effective non-viral gene delivery vector

Biotechnology and Genetic Engineering Reviews

Volumn 31, Issue 1-2, 2015, Pages 82-107

  Šimčíková, Michaela ^a,b   Prather, Kristala L J ^a,c   Prazeres, Duarte M F ^a,c,d   Monteiro, Gabriel A ^a,c,d  

^a MIT Portugal Program   (Portugal)

^b Instituto de Biotecnologia e Bioengenharia Lisboa   (Portugal)

^c Massachusetts Institute of Technology Cambridge   (United States)

^d INSTITUTO SUPERIOR TÉCNICO   (Portugal)

Author keywords

DNA vaccine; gene therapy; minicircle; minimal vectors; recombination; transgene silencing

Indexed keywords

BIOCHEMISTRY; BIOMOLECULES; DNA; GENE THERAPY; GENE TRANSFER;

CLINICAL TRIAL; DNA VACCINE; GENE DELIVERY VECTORS; MINICIRCLE; MINIMAL VECTOR; NON-VIRAL GENE DELIVERY; PERFORMANCE; RECOMBINATION; SAFETY RECORDS; TRANSGENE SILENCING;

VECTORS;

ALPHA 1 ANTITRYPSIN; ARABINOSE; ARAC PROTEIN; BETA LACTAM ANTIBIOTIC; BLOOD CLOTTING FACTOR 9; CPG OLIGODEOXYNUCLEOTIDE; CRE RECOMBINASE; CYTOSINE; DNA BINDING PROTEIN; DNA TOPOISOMERASE; DNA VACCINE; ENDONUCLEASE; GAMMA INTERFERON; GENOMIC DNA; GUANINE; HETEROCHROMATIN PROTEIN 1; HYPOXIA INDUCIBLE FACTOR 1ALPHA; INTEGRASE; LEVO IDURONIDASE; MANGANESE SUPEROXIDE DISMUTASE; MICRORNA; MINICIRCLE DNA; NUCLEASE; PHOSPHATE; PLASMID DNA; PLASMID VECTOR; RECOMBINASE; RESOLVASE; VASCULOTROPIN; DNA;

ADIPOSE DERIVED STEM CELL; ANTIGEN EXPRESSION; ARTICLE; BACTERIOPHAGE; BIOAVAILABILITY; CODON; CONSENSUS SEQUENCE; DNA DENATURATION; DNA IMMUNIZATION; DNA REPLICATION ORIGIN; ESCHERICHIA COLI; GENE CASSETTE; GENE EXPRESSION; GENE EXPRESSION SYSTEM; GENE SILENCING; GENE VECTOR; GENETIC RECOMBINATION; GENETIC TRANSFECTION; HISTONE MODIFICATION; HUMAN; INDUCED PLURIPOTENT STEM CELL; INTRON; INVERTED REPEAT; LEISHMANIA; MATRIX ATTACHMENT REGION; MINIMALISTIC IMMUNOGENIC DEFINED GENE EXPRESSION VECTOR; MUTAGENESIS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; NUCLEAR LOCALIZATION SIGNAL; PLASMID; POLYADENYLATION; PROMOTER REGION; PROTEIN EXPRESSION; TRANSGENE; VACCINIA VIRUS; ANIMAL; GENE TRANSFER; GENETICS;

ANIMALS; DNA; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HUMANS; PLASMIDS; PROMOTER REGIONS, GENETIC; REPLICATION ORIGIN; TRANSGENES;

EID: 84969718630     PISSN: 02648725     EISSN: 20465556     Source Type: Journal    
DOI: 10.1080/02648725.2016.1178011     Document Type: Article

References (102)

1. S.André,, B.Seed,, J.Eberle,, W.Schraut,, A.Bültmann,, J.Haas,, & A.Bu, (1998). Increased immune response elicited by DNA vaccination with a synthetic gp120 sequence with optimized codon usage increased immune response elicited by DNA vaccination with a synthetic gp120 sequence with optimized codon usage. Journal of Virology, 72, 1497–1503.
2. O.Argyros,, S.P.Wong,, C.Fedonidis,, O.Tolmachov,, S.N.Waddington,, S.J.Howe, … R.P.Harbottle, (2011). Development of S/MAR minicircles for enhanced and persistent transgene expression in the mouse liver. Journal of Molecular Medicine, 89, 515–529.10.1007/s00109-010-0713-3
3. O.Argyros,, S.P.Wong,, M.Niceta,, S.N.Waddington,, & S.J.Howe,, C.Coutelle,, … R.P.Harbottle, (2008). Persistent episomal transgene expression in liver following delivery of a scaffold/matrix attachment region containing non-viral vector. Gene Therapy, 15, 1593–1605.10.1038/gt.2008.113
4. A.R.Azzoni,, S.C.Ribeiro,, G.A.Monteiro,, & D.M.F.Prazeres, (2007). The impact of polyadenylation signals on plasmid nuclease-resistance and transgene expression. The Journal of Gene Medicine, 9, 392–402.10.1002/(ISSN)1521-2254
5. A.Baiker,, C.Maercker,, C.Piechaczek,, S.B.Schmidt,, J.Bode,, C.Benham,, & H.J.Lipps, (2000). Mitotic stability of an episomal vector containing a human scaffold/matrix-attached region is provided by association with nuclear matrix. National Cell Biology, 2, 182–184.
6. B.W.Bigger,, O.Tolmachov,, J.M.Collombet,, M.Fragkos,, I.Palaszewski,, & C.Coutelle, (2001). An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy. Journal of Biological Chemistry, 276, 23018–23027.10.1074/jbc.M010873200
7. C.Bode,, G.Zhao,, F.Steinhagen,, T.Kinjo,, & D.M.Klinman, (2011). CpG DNA as a vaccine adjuvant. Expert Review of Vaccines, 10, 499–511.10.1586/erv.10.174
8. D.M.Bower,, & K.L.J.Prather, (2009). Engineering of bacterial strains and vectors for the production of plasmid DNA. Applied Microbiology and Biotechnology, 82, 805–813.10.1007/s00253-009-1889-8
9. R.L.Brinster,, J.M.Allen,, R.R.Behringer,, R.E.Gelinas,, & R.D.Palmiter, (1988). Introns increase transcriptional efficiency in transgenic mice. Proceedings of the National Academy of Sciences, 85, 836–840.10.1073/pnas.85.3.836
10. S.Broll,, A.Oumard,, K.Hahn,, A.Schambach,, & J.Bode, (2010). Minicircle performance depending on S/MAR-nuclear matrix interactions. Journal of Molecular Biology, 395, 950–965.10.1016/j.jmb.2009.11.066
11. B.Brondyk, (1994). pCI and pSI Mammalian Expression Vectors. Promega Notes. 49:7–11.
12. B.D.Brown,, B.Gentner,, A.Cantore,, S.Colleoni,, M.Amendola,, A.Zingale,, … L.Naldini, (2007). Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nature Biotechnology, 25, 1457–1467.10.1038/nbt1372
13. K.Cashman, K.Broderick,, E.Wilkinson,, C.Shaia,, T.Bell,, A.Shurtleff,, … C.Schmaljohn, (2013). Enhanced efficacy of a codon-optimized DNA vaccine encoding the glycoprotein precursor gene of lassa virus in a Guinea pig disease model when delivered by dermal electroporation. Vaccines, 1, 262–277.10.3390/vaccines1030262
14. D.J.Catanese,, J.M.Fogg,, D.E.Schrock,, B.E.Gilbert,, & L.Zechiedrich, (2012). Supercoiled minivector DNA resists shear forces associated with gene therapy delivery. Gene Therapy, 19, 94–100.10.1038/gt.2011.77
15. Z.Y.Chen,, C.Y.He,, A.Ehrdfardt,, & M.A.Kay, (2003). Minicircle DNA vectors devoid of bacterial DNA results in persistent and high-level transgene expression in vivo. Molecular Therapy, 8, 495–500.10.1016/S1525-0016(03)00168-0
16. Z.Y.Chen,, C.Y.He,, & M.A.Kay, (2005). Improved production and purification of minicircle DNA vector free of plasmid bacterial sequences and capable of persistent transgene expression in vivo. Human Gene Therapy, 16, 126–131.10.1089/hum.2005.16.126
17. Z.Y.Chen,, C.Y.He,, L.Meuse,, & M.A.Kay, (2004). Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Therapy, 11, 856–864.10.1038/sj.gt.3302231
18. Z.Y.Chen,, E.Riu,, C.Y.He,, H.Xu,, & M.A.Kay, (2008). Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Molecular Therapy, 16, 548–556.10.1038/sj.mt.6300399
19. T.Choi,, M.Huang,, C.Gorman,, & R.Jaenisch, (1991). A generic intron increases gene expression in transgenic mice. Molecular and Cellular Biology, 11, 3070–3074.10.1128/MCB.11.6.3070
20. J.R.Cooke,, E.A.McKie,, J.M.Ward,, & E.Keshavarz-Moore, (2004). Impact of intrinsic DNA structure on processing of plasmids for gene therapy and DNA vaccines. Journal of Biotechnology, 114, 239–254.10.1016/j.jbiotec.2004.06.011
21. F.Cramer,, C.L.Christensen,, T.T.Poulsen,, M.A.Badding,, D.A.Dean,, & H.S.Poulsen, (2012). Insertion of a nuclear factor kappa B DNA nuclear-targeting sequence potentiates suicide gene therapy efficacy in lung cancer cell lines. Cancer Gene Therapy, 19, 675–683.10.1038/cgt.2012.54
22. A.M.Darquet,, B.Cameron,, P.Wils,, D.Scherman,, & J.Crouzet, (1997). A new DNA vehicle for nonviral gene delivery: Supercoiled minicircle. Gene Therapy, 4, 1341–1349.10.1038/sj.gt.3300540
23. A.M.Darquet,, R.Rangara,, P.Kreiss,, B.Schwartz,, S.Naimi,, P.Delaère,, … D.Sherman, (1999). Minicircle: An improved DNA molecule for in vitro and in vivo gene transfer. Gene Therapy, 6, 209–218.10.1038/sj.gt.3300816
24. D.A.Dean, (2013). Cell-specific targeting strategies for electroporation-mediated gene delivery in cells and animals. The Journal of Membrane Biology, 246, 737–744.10.1007/s00232-013-9534-y
25. D.A.Dean,, B.S.Dean,, S.Muller,, & L.C.Smith, (1999). Sequence requirements for plasmid nuclear import. Experimental Cell Research, 253, 713–722.10.1006/excr.1999.4716
26. W.M.Dietz,, N.E.B.Skinner,, S.E.Hamilton,, M.D.Jund,, S.M.Heitfeld,, A.J.Litterman,, … M.J.Osborn, (2013). Minicircle DNA is superior to plasmid DNA in eliciting antigen-specific CD8(+) T-cell responses. Molecular Therapy, 21, 1526–1535.10.1038/mt.2013.85
27. Y.Dong,, A.Aied,, J.Li,, Q.Wang,, X.Hu,, & W.Wang, (2013). An in vitro approach for production of non-scar minicircle DNA vectors. Journal of Biotechnology, 2, 8–11.
28. L.Eberl,, C.S.Kristensen,, M.Givskov,, E.Grohmann,, M.Gerlitz,, & H.Schwab, (1994). Analysis of the multimer resolution system encoded by the parCBA operon of broad-host-range plasmid RP4. Molecular Microbiology, 12, 131–141.10.1111/mmi.1994.12.issue-1
29. EMEA. (2001). Note for guidance on the quality, preclinical and clinical aspects of gene transfer medicinal products. CPMP/BWP/3088/99. 0–32. London: Author.
30. F.Faurez,, D.Dory,, V.Le Moigne,, R.Gravier,, & A.Jestin, (2010). Biosafety of DNA vaccines: New generation of DNA vectors and current knowledge on the fate of plasmids after injection. Vaccine., 28, 3888–3895.10.1016/j.vaccine.2010.03.040
31. FDA. (1996). Points to consider on plasmid DNA vaccines for preventive infectious disease indications. Docket No 96N-0400. Rockville, MD: Author.
32. B.Ferraro,, M.P.Morrow,, N.A.Hutnick,, T.H.Shin,, C.E.Lucke,, & D.B.Weiner, (2011). Clinical applications of DNA vaccines: Current progress. Clinical Infectious Diseases, 53, 296–302.10.1093/cid/cir334
33. M.Gellert,, & H.Nash, (1987). Communication between segments of DNA during site-specific recombination. Nature, 325, 401–404.10.1038/325401a0
34. Gene Therapy Clinical Trials Worldwide. (2016). Journal of Gene Medicine. Retrieved January 7, 2016, from, http://www.abedia.com/wiley/
35. M.Gerlitz,, O.Hrabak,, & H.Schwab, (1990). Partitioning of broad-host-range plasmid Rp4 is a complex system involving site-specific recombination. Journal of Bacteriology, 172, 6194–6203.
36. L.E.Gracey Maniar,, J.M.Maniar,, Z.Y.Chen,, J.Lu,, A.Z.Fire,, & M.A.Kay, (2013). Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Molecular Therapy, 21, 131–138.10.1038/mt.2012.244
37. N.D.F.Grindley,, K.L.Whiteson,, & P.A.Rice, (2006). Mechanisms of site-specific recombination. Annual Review of Biochemistry, 75, 567–605.10.1146/annurev.biochem.73.011303.073908
38. M.Gürsel,, D.Verthelyi,, I.Gürsel,, K.J.Ishii,, & D.M.Klinman, (2002). Differential and competitive activation of human immune cells by distinct classes of CpG oligodeoxynucleotide. Journal of Leukocyte Biology, 71, 813–820.
39. L.M.Guzman,, D.Belin,, M.J.Carson,, & J.Beckwith, (1995). Tight regulation, modulation, and high-level expression by vectors containing the arabinose PBAD promoter. Journal of Bacteriology, 177, 4121–4130.
40. R.Haase,, O.Argyros,, S.P.Wong,, R.P.Harbottle,, H.J.Lipps,, M.Ogris,, … A.Baiker, (2010). pEPito: A significantly improved non-viral episomal expression vector for mammalian cells. BMC Biotechnology, 10, 20.10.1186/1472-6750-10-20
41. C.Hagedorn,, S.-P.Wong,, R.Harbottle,, & H.J.Lipps, (2011). Scaffold/matrix attached region-based nonviral episomal vectors. Human Gene Therapy, 22, 915–923.10.1089/hum.2011.084
42. N.Harraghy,, A.Gaussin,, & N.Mermod, (2008). Sustained transgene expression using MAR elements. Current Gene Therapy, 8, 353–366.10.2174/156652308786071032
43. M.Huang,, Z.Chen,, S.Hu,, F.Jia,, Z.Li,, & G.Hoyt,, … J.C.Wu, (2009). Novel minicircle vector for gene therapy in murine myocardial infarction. Circulation, 120, S230–S237.10.1161/CIRCULATIONAHA.108.841155
44. S.C.Hyde,, I.A.Pringle,, S.Abdullah,, A.E.Lawton,, L.A.Davies,, A.Varathalingam,, … D.R.Gill, (2008). CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. Nature Biotechnology, 26, 549–551. 10.1038/nbt1399
45. W.Jechlinger,, C.Azimpour Tabrizi,, W.Lubitz,, P.Mayrhofer, (2004). Minicircle DNA immobilized in bacterial ghosts: In vivo production of safe non-viral DNA delivery vehicles. Journal of Molecular Microbiology and Biotechnology, 8, 222–231.10.1159/000086703
46. F.Jia,, K.D.Wilson,, N.Sun,, D.M.Gupta,, M.Huang,, Z.Li,, … J.C.Wu, (2010). A nonviral minicircle vector for deriving human iPS cells. Nature Methods, 7, 197–199.10.1038/nmeth.1426
47. M.A.Kay,, C.Y.He,, & Z.Y.Chen, (2010). A robust system for production of minicircle DNA vectors. Nature Biotechnology, 28, 1287–1289.10.1038/nbt.1708
48. A.Khlebnikov,, K.A.Datsenko,, T.Skaug,, B.L.Wanner,, & J.D.Keasling, (2001). Homogeneous expression of the PBAD promoter in Escherichia coli by constitutive expression of the low-affinity high-capacity AraE transporter. Microbiology, 147, 3241–3247.10.1099/00221287-147-12-3241
49. D.M.Klinman, (2004). Immunotherapeutic uses of CpG oligodeoxynucleotides. Nature Reviews Immunology, 4, 249–259.10.1038/nri1329
50. D.Kobelt,, M.Schleef,, M.Schmeer,, J.Aumann,, P.M.Schlag,, & W.Walther, (2013). Performance of high quality minicircle DNA for in vitro and in vivo gene transfer. Molecular Biotechnology, 53, 80–89.
51. M.Kozak, (1997). Recognition of AUG and alternative initiator codons is augmented by G in position +4 but is not generally affected by the nucleotides in positions +5 and +6. The EMBO Journal, 16, 2482–2492.10.1093/emboj/16.9.2482
52. P.Kreiss,, B.Cameron,, A.M.Darquet,, D.Scherman,, & J.Crouzet, (1998). Production of a new DNA vehicle for gene transfer using site-specific recombination. Applied Microbiology and Biotechnology, 49, 560–567.10.1007/s002530051213
53. A.M.Krieg, (1999). Direct immunologic activities of CpG DNA and implications for gene therapy. The Journal of Gene Medicine, 1, 56–63.10.1002/(SICI)1521-2254(199901/02)1:1<56::AID-JGM5>3.3.CO;2-Y
54. A.Lacy-Hulbert,, R.Thomas,, X.P.Li,, C.E.Lilley,, R.S.Coffin,, & J.Roes, (2001). Interruption of coding sequences by heterologous introns can enhance the functional expression of recombinant genes. Gene Therapy, 8, 649–653.10.1038/sj.gt.3301440
55. A.Landy, (1989). Dynamic, structural, and regulatory aspects of lambda site-specific recombination. Annual Review of Biochemistry, 58, 913–949.10.1146/annurev.bi.58.070189.004405
56. A.R.Lara,, & O.T.Ramírez, (2012). Plasmid DNA production for therapeutic applications. Methods in Molecular Biology, 824, 271–303.10.1007/978-1-61779-433-9
57. L.López-Fuertes,, E.Pérez-Jiménez,, A.J.Vila-Coro,, F.Sack,, S.Moreno,, S.A.Konig,, … M.Esteban, (2002). DNA vaccination with linear minimalistic (MIDGE) vectors confers protection against Leishmania major infection in mice. Vaccine, 21, 247–257.10.1016/S0264-410X(02)00450-4
58. J.Lu,, F.Zhang,, & M.A.Kay, (2013). A mini-intronic plasmid (MIP): A novel robust transgene expression vector in vivo and in vitro. Molecular Therapy, 21, 954–963.10.1038/mt.2013.33
59. J.Lu,, F.Zhang,, S.Xu,, A.Z.Fire,, & M.A.Kay, (2012). The extragenic spacer length between the 5′ and 3′ ends of the transgene expression cassette affects transgene silencing from plasmid-based vectors. Molecular Therapy, 20, 2111–2119.10.1038/mt.2012.65
60. J.Luke,, A.E.Carnes,, C.P.Hodgson,, & J.A.Williams, (2009). Improved antibiotic-free DNA vaccine vectors utilizing a novel RNA based plasmid selection system. Vaccine, 27, 6454–6459.10.1016/j.vaccine.2009.06.017
61. J.Mahillon,, & M.Chandler, (1998). Insertion sequences. Microbiology and Molecular Biology Reviews, 62, 752–774.
62. P.Mayrhofer,, M.Blaesen,, M.Schleef,, & W.Jechlinger, (2008). Minicircle-DNA production by site specific recombination and protein-DNA interaction chromatography. The Journal of Gene Medicine, 10, 1253–1269.10.1002/jgm.v10:11
63. M.J.McCluskie,, R.D.Weeratna,, & H.L.Davis, (2000). The role of CpG in DNA vaccines. Springer Seminars in Immunopathology, 22, 125–132.10.1007/s002810000014
64. A.Mesika,, I.Grigoreva,, M.Zohar,, & Z.Reich, (2001). A regulated, NFκB-assisted import of plasmid DNA into mammalian cell nuclei. Molecular Therapy, 3, 653–657.10.1006/mthe.2001.0312
65. A.M.Miller,, & D.A.Dean, (2008). Cell-specific nuclear import of plasmid DNA in smooth muscle requires tissue-specific transcription factors and DNA sequences. Gene Therapy, 15, 1107–1115.10.1038/gt.2008.83
66. H.A.Nash, (1996). Site-specific recombination: Integration, excision, resolution, and Inversion of defined DNA segments. Washington, DC: ASM Press, 2363–2376.
67. K.Nehlsen,, S.Broll,, & J.Bode, (2006). Replicating minicircles: Generation of nonviral episomes for the efficient modification of dividing cells. Gene Therapy and Molecular Biology, 10, 233–244.
68. A.T.Nguyen,, A.C.Dow,, J.Kupiec-Weglinski,, R.W.Busuttil,, & G.S.Lipshutz, (2008). Evaluation of gene promoters for liver expression by hydrodynamic gene transfer. Journal of Surgical Research, 148, 60–66.10.1016/j.jss.2008.02.016
69. G.Olafsdóttir,, V.Svansson,, S.Ingvarsson,, E.Marti,, & S.Torsteinsdóttir, (2008). In vitro analysis of expression vectors for DNA vaccination of horses: The effect of a Kozak sequence. Acta Veterinaria Scandinavica, 50, 44.10.1186/1751-0147-50-44
70. P.H.Oliveira,, & J.Mairhofer, (2013). Marker-free plasmids for biotechnological applications – Implications and perspectives. Trends in Biotechnology, 31, 539–547.10.1016/j.tibtech.2013.06.001
71. P.H.Oliveira,, K.J.Prather,, D.M.F.Prazeres,, & G.A.Monteiro, (2009). Structural instability of plasmid biopharmaceuticals: Challenges and implications. Trends in Biotechnology, 27, 503–511.10.1016/j.tibtech.2009.06.004
72. M.J.Osborn,, R.T.McElmurry,, C.J.Lees,, A.P.DeFeo,, Z.Y.Chen,, M.A.Kay,, … B.R.Blazar, (2011). Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of alpha-L-Iduronidase in mice withmucopolysaccharidosis type I. Molecular Therapy, 9, 450–460.10.1038/mt.2010.249
73. R.D.Palmiter,, E.P.Sandgren,, M.R.Avarbock,, D.D.Allen,, & R.L.Brinster, (1991). Heterologous introns can enhance expression of transgenes in mice. Proceedings of the National Academy of Sciences, 88, 478–482.10.1073/pnas.88.2.478
74. C.Piechaczek,, C.Fetzer,, A.Baiker,, J.Bode,, & H.J.Lipps, (1999). A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells. Nucleic Acids Research, 27, 426–428.10.1093/nar/27.2.426
75. K.L.J.Prather,, M.C.Edmonds,, & J.W.Herod, (2006). Identification and characterization of IS1 transposition in plasmid amplification mutants of E. coli clones producing DNA vaccines. Applied Microbiology and Biotechnology, 73, 815–826.10.1007/s00253-006-0532-1
76. D.M.F.Prazeres, (2011). Plasmid biopharmaceuticals. Hoboken, NJ: Wiley.10.1002/9780470939918
77. Promega Technical Buletin. (2007). Expression vector pCI-neo mammalian expression Vector. Madison, WI: Promega.
78. N.Proudfoot, (1991). Poly(A) signals. Cell, 64, 671–674.10.1016/0092-8674(91)90495-K
79. R.Rankin,, R.Pontarollo,, X.Ioannou,, A.M.Krieg,, R.Hecker,, L.A.Babiuk,, …, S.van den Hurk, (2001). CpG motif identification for veterinary and laboratory species demonstrates that sequence recognition is highly conserved. Antisense and Nucleic Acid Drug Development, 11, 333–340.10.1089/108729001753231713
80. A.Razin,, & J.Friedman, (1981). DNA methylation and its possible biological roles. Progress in Nucleic Acid Research and Molecular Biology, 25, 33–52.10.1016/S0079-6603(08)60482-1
81. B.Rehberger,, M.Heine,, C.Wodarczyk,, & R.Wagner, (2013). Methods for semi-synthetically producing highly pure minicircle DNA vectors from plasmids. U.S. Patent EP2625275 A1.
82. S.C.Ribeiro,, P.H.Oliveira,, D.M.F.Prazeres,, & G.A.Monteiro, (2008). High frequency plasmid recombination mediated by 28 bp direct repeats. Molecular Biotechnology, 40, 252–260.10.1007/s12033-008-9082-3
83. E.Riu,, Z.Y.Chen,, H.Xu,, C.Y.He,, & M.A.Kay, (2007). Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Molecular Therapy, 15, 1348–1355.10.1038/sj.mt.6300177
84. R.C.Roberts,, A.R.Ström,, & D.R.Helinski, (1994). The parDE operon of the broad-host-range plasmid RK2 specifies growth inhibition associated with plasmid loss. Journal of Molecular Biology, 237, 35–51.10.1006/jmbi.1994.1207
85. F.Schakowski,, M.Gorschlüter,, P.Buttgereit,, A.Märten,, M.V.Lilienfeld-Toal,, C.Junghans,, … I.G.Schmidt-Wolf, (2007). Minimal size MIDGE vectors improve transgene expression in vivo. In Vivo, 21, 17–23.
86. R.Schirmbeck,, S.A.König-Merediz,, P.Riedl,, M.Kwissa,, F.Sack,, & M.Schroff,, … B.Wittig, (2001). Priming of immune responses to hepatitis B surface antigen with minimal DNA expression constructs modified with a nuclear localization signal peptide. Journal of Molecular Medicine, 79, 343–350.10.1007/s001090100227
87. N.Sharma,, Y.Cai,, R.O.Bak,, M.R.Jakobsen,, L.D.Schrøder,, & J.G.Mikkelsen, (2013). Efficient sleeping beauty DNA transposition from DNA minicircles. Molecular Therapy — Nucleic Acids, 2, e74.10.1038/mtna.2013.1
88. S.Stenler,, A.Andersson,, O.E.Simonson,, K.E.Lundin,, Z.Y.Chen,, M.A.Kay,, … P.Blomberg, (2009). Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. Journal of Cardiovascular Pharmacology, 53, 18–23.10.1097/FJC.0b013e318194234e
89. M.Suzuki,, K.Kasai,, & Y.Saeki, (2006). Plasmid DNA sequences present in conventional herpes simplex virus amplicon vectors cause rapid transgene silencing by forming inactive chromatin. Journal of Virology, 80, 3293–3300.10.1128/JVI.80.7.3293-3300.2006
90. T.Tagami,, K.Nakamura,, T.Shimizu,, N.Yamazaki,, T.Ishida,, & H.Kiwada, (2010). CpG motifs in pDNA-sequences increase anti-PEG IgM production induced by PEG-coated pDNA-lipoplexes. Journal of Controlled Release, 142, 160–166.10.1016/j.jconrel.2009.10.017
91. Y.Tan,, S.Li,, B.R.Pitt,, & L.Huang, (1999). The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivo. Human Gene Therapy, 10, 2153–2161.10.1089/10430349950017149
92. A.Valera,, J.C.Perales,, M.Hatzoglou,, & F.Bosch, (1994). Expression of the neomycin-resistance (neo) gene induces alterations in gene expression and metabolism. Human Gene Therapy, 5, 449–456.10.1089/hum.1994.5.4-449
93. H.Weintraub,, P.F.Cheng,, & K.Conrad, (1986). Expression of transfected DNA depends on DNA topology. Cell, 46, 115–122.10.1016/0092-8674(86)90865-2
94. D.A.Williams, (2008). Sleeping beauty vector system moves toward human trials in the United States. Molecular Therapy, 16, 1515–1516.10.1038/mt.2008.169
95. J.A.Williams,, A.E.Carnes,, & C.P.Hodgson, (2009). Plasmid DNA vaccine vector design: Impact on efficacy, safety and upstream production. Biotechnology Advances, 27, 353–370.10.1016/j.biotechadv.2009.02.003
96. B.Wittig,, A.Märten,, T.Dorbic,, S.Weineck,, H.Min,, S.Niemitz,, … I.G.Schmidt-Wolf, (2001). Therapeutic vaccination against metastatic carcinoma by expression-modulated and immunomodified autologous tumor cells: A first clinical phase I/II trial. Human Gene Therapy, 12, 267–278.10.1089/10430340150218404
97. J.A.Wolff,, R.W.Malone,, P.Williams,, W.Chong,, G.Acsadi,, A.Jani,, & P.L.Felgner, (1990). Direct gene transfer into mouse muscle in vivo. Science, 247, 1465–1468.10.1126/science.1690918
98. J.X.Wu,, X.Xiao,, P.Zhao,, G.Xue,, Y.H.Zhu,, & X.F.Zhu,, … W.Huang, (2006). Minicircle-IFN induces antiproliferative and antitumoral effects in human nasopharyngeal carcinoma. Clinical Cancer Research, 12, 4702–4713.10.1158/1078-0432.CCR-06-0520
99. N.S.Yew,, M.Przybylska,, R.J.Ziegler,, D.Liu,, & S.H.Cheng, (2001). High and sustained transgene expression in vivo from plasmid vectors containing a hybrid ubiquitin promoter. Molecular Therapy, 4, 75–82.10.1006/mthe.2001.0415
100. J.L.Young,, J.N.Benoit,, & D.A.Dean, (2003). Effect of a DNA nuclear targeting sequence on gene transfer and expression of plasmids in the intact vasculature. Gene Therapy, 10, 1465–1470.10.1038/sj.gt.3302021
101. X.C.Zhang,, M.W.Epperly,, M.A.Kay,, Z.Y.Chen,, T.Dixon,, D.Franicola,, … J.S.Greenberger, (2008). Radioprotection in vitro and in vivo by minicircle plasmid carrying the human manganese superoxide dismutase transgene. Human Gene Therapy, 19, 820–826.10.1089/hum.2007.141
102. H.Zhou,, D.Liu,, & C.Liang, (2004). Challenges and strategies: The immune responses in gene therapy. Medicinal Research Reviews, 24, 748–761.10.1002/(ISSN)1098-1128