Micro-minicircle gene therapy: Implications of size on fermentation, complexation, shearing resistance, and expression

Molecular Therapy Nucleic Acids

Volumn 3, Issue , 2014, Pages e140-

  Stenler, Sofia ^a   Wiklander, Oscar P B ^a   Badal Tejedor, Maria ^b   Turunen, Janne ^a   Nordin, Joel Z ^a   Hallengärd, David ^a   Wahren, Britta ^a   Andaloussi, Samir E L ^a,c   Rutland, Mark W ^d   Smith, C I Edvard ^a   Lundin, Karin E ^a   Blomberg, Pontus ^a,e  

^a KAROLINSKA INSTITUTE   (Sweden)

^b INSTITUTE FOR SURFACE CHEMISTRY   (Sweden)

^c UNIVERSITY OF OXFORD   (United Kingdom)

^d ROYAL INSTITUTE OF TECHNOLOGY   (Sweden)

^e KAROLINSKA UNIVERSITY HOSPITAL   (Sweden)

Author keywords

Nano; Pre mRNA; RNA editing; RNA therapeutics

Indexed keywords

PLASMID VECTOR; RNA; SMALL NUCLEAR RNA; SMALL REGULATORY RNA; UNCLASSIFIED DRUG; LUCIFERASE; MICRO MINICIRCLE; MINICIRCLE DNA;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; ATOMIC FORCE MICROSCOPY; COMPLEX FORMATION; CONTROLLED STUDY; ELECTROPORATION; FERMENTATION; GEL ELECTROPHORESIS; GENE EXPRESSION; GENE VECTOR; GENETIC TRANSFECTION; HELA CELL; HUMAN; HUMAN CELL; HYDRODYNAMICS; IN VITRO STUDY; IN VIVO STUDY; MICRO MINICIRCLE GENE VECTOR; MOUSE; NONHUMAN; NONVIRAL GENE THERAPY; PARTICLE SIZE; PRIORITY JOURNAL; DNA SUPERCOILING; DRUG SAFETY; FEMALE; GENE THERAPY; GENETIC RECOMBINATION; MOLECULAR STABILITY; PLASMID;

EID: 84893163206     PISSN: None     EISSN: 21622531     Source Type: Journal    
DOI: None     Document Type: Article

References (36)

1. Viola, JR, El-Andaloussi, S, Oprea, II and Smith, CI (2010). Non-viral nanovectors for gene delivery: factors that govern successful therapeutics. Expert Opin Drug Deliv 7: 721-735.
2. Gill, DR, Pringle, IA and Hyde, SC (2009). Progress and prospects: the design and production of plasmid vectors. Gene Ther 16: 165-171.
3. Vandermeulen, G, Marie, C, Scherman, D and Préat, V (2011). New generation of plasmid backbones devoid of antibiotic resistance marker for gene therapy trials. Mol Ther 19: 1942-1949.
4. Darquet, AM, Cameron, B, Wils, P, Scherman, D and Crouzet, J (1997). A new DNA vehicle for nonviral gene delivery: supercoiled minicircle. Gene Ther 4: 1341-1349. (Pubitemid 28004593)
5. Bigger, BW, Tolmachov, O, Collombet, JM, Fragkos, M, Palaszewski, I and Coutelle, C (2001). An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy. J Biol Chem 276: 23018-23027.
6. Chen, ZY, He, CY, Ehrhardt, A and Kay, MA (2003). Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8: 495-500. (Pubitemid 37161471)
7. Mayrhofer, P, Blaesen, M, Schleef, M and Jechlinger, W (2008). Minicircle-DNA production by site specific recombination and protein-DNA interaction chromatography. J Gene Med 10: 1253-1269.
8. Kay, MA, He, CY and Chen, ZY (2010). A robust system for production of minicircle DNA vectors. Nat Biotechnol 28: 1287-1289.
9. Zhao, N, Fogg, JM, Zechiedrich, L and Zu, Y (2011). Transfection of shRNA-encoding Minivector DNA of a few hundred base pairs to regulate gene expression in lymphoma cells. Gene Ther 18: 220-224.
10. Liu, X, Yang, Z and Feng, T (2012). [A minicircle DNA vector-mediated siRNA to stably suppress hepatitis B virus replication and expression]. Wei Sheng Wu Xue Bao 52: 191-197.
11. Vafabakhsh, R and Ha, T (2012). Extreme bendability of DNA less than 100 base pairs long revealed by single-molecule cyclization. Science 337: 1097-1101.
12. Bates, AD and Maxwell, A (1989). DNA gyrase can supercoil DNA circles as small as 174 base pairs. EMBO J 8: 1861-1866. (Pubitemid 19274391)
13. Fogg, JM, Kolmakova, N, Rees, I, Magonov, S, Hansma, H, Perona, JJ et al. (2006). Exploring writhe in supercoiled minicircle DNA. J Phys Condens Matter 18: S145-S159.
14. Kano, Y, Miyashita, T, Nakamura, H, Kuroki, K, Nagata, A and Imamoto, F (1981). In vivo correlation between DNA supercoiling and transcription. Gene 13: 173-184. (Pubitemid 11031840)
15. Sekiguchi, JM and Kmiec, EB (1989). DNA superhelicity enhances the assembly of transcriptionally active chromatin in vitro. Mol Gen Genet 220: 73-80. (Pubitemid 20019530)
16. Kang, SH, Cho, MJ and Kole, R (1998). Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry 37: 6235-6239. (Pubitemid 28213640)
17. Goyenvalle, A, Vulin, A, Fougerousse, F, Leturcq, F, Kaplan, JC, Garcia, L et al. (2004). Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306: 1796-1799. (Pubitemid 39601401)
18. Gorman, L, Suter, D, Emerick, V, Schümperli, D and Kole, R (1998). Stable alteration of pre-mRNA splicing patterns by modified U7 small nuclear RNAs. Proc Natl Acad Sci USA 95: 4929-4934. (Pubitemid 28208527)
19. Vahlsing, HL, Yankauckas, MA, Sawdey, M, Gromkowski, SH and Manthorpe, M (1994). Immunization with plasmid DNA using a pneumatic gun. J Immunol Methods 175: 11-22. (Pubitemid 24305227)
20. Suda, T and Liu, D (2007). Hydrodynamic gene delivery: its principles and applications. Mol Ther 15: 2063-2069. (Pubitemid 350148920)
21. Stenler, S, Andersson, A, Simonson, OE, Lundin, KE, Chen, ZY, Kay, MA et al. (2009). Gene transfer to mouse heart and skeletal muscles using a minicircle expressing human vascular endothelial growth factor. J Cardiovasc Pharmacol 53: 18-23.
22. Leahy, P, Carmichael, GG and Rossomando, EF (1997). Transcription from plasmid expression vectors is increased up to 14-fold when plasmids are transfected as concatemers. Nucleic Acids Res 25: 449-450. (Pubitemid 27303342)
23. Voss, C, Schmidt, T, Schleef, M, Friehs, K and Flaschel, E (2003). Production of supercoiled multimeric plasmid DNA for biopharmaceutical application. J Biotechnol 105: 205-213. (Pubitemid 37315763)
24. Catanese, DJ Jr, Fogg, JM, Schrock, DE 2nd, Gilbert, BE and Zechiedrich, L (2012). Supercoiled Minivector DNA resists shear forces associated with gene therapy delivery. Gene Ther 19: 94-100.
25. Wang, R, Epstein, J, Baraceros, FM, Gorak, EJ, Charoenvit, Y, Carucci, DJ et al. (2001). Induction of CD4(+) T cell-dependent CD8(+) type 1 responses in humans by a malaria DNA vaccine. Proc Natl Acad Sci USA 98: 10817-10822. (Pubitemid 32878703)
26. Snoep, JL, van der Weijden, CC, Andersen, HW, Westerhoff, HV and Jensen, PR (2002). DNA supercoiling in Escherichia coli is under tight and subtle homeostatic control, involving gene-expression and metabolic regulation of both topoisomerase I and DNA gyrase. Eur J Biochem 269: 1662-1669. (Pubitemid 34304465)
27. Reyes-Domínguez, Y, Contreras-Ferrat, G, Ramírez-Santos, J, Membrillo-Hernández, J and Gómez-Eichelmann, MC (2003). Plasmid DNA supercoiling and gyrase activity in Escherichia coli wild-Type and rpoS stationary-phase cells. J Bacteriol 185: 1097-1100. (Pubitemid 36139469)
28. Nishikawa, J, Amano, M, Fukue, Y, Tanaka, S, Kishi, H, Hirota, Y et al. (2003). Lefthandedly curved DNA regulates accessibility to cis-DNA elements in chromatin. Nucleic Acids Res 31: 6651-6662. (Pubitemid 37508772)
29. Parvin, JD, McCormick, RJ, Sharp, PA and Fisher, DE (1995). Pre-bending of a promoter sequence enhances affinity for the TATA-binding factor. Nature 373: 724-727.
30. Rajendra, Y, Kiseljak, D, Manoli, S, Baldi, L, Hacker, DL and Wurm, FM (2012). Role of non-specific DNA in reducing coding DNA requirement for transient gene expression with CHO and HEK-293E cells. Biotechnol Bioeng 109: 2271-2278.
31. Chen, ZY, Riu, E, He, CY, Xu, H and Kay, MA (2008). Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther 16: 548-556. (Pubitemid 351314995)
32. Riu, E, Chen, ZY, Xu, H, He, CY and Kay, MA (2007). Histone modifications are associated with the persistence or silencing of vector-mediated transgene expression in vivo. Mol Ther 15: 1348-1355. (Pubitemid 46965331)
33. Schüttrumpf, J, Milanov, P, Abriss, D, Roth, S, Tonn, T and Seifried, E (2011). Transgene loss and changes in the promoter methylation status as determinants for expression duration in nonviral gene transfer for factor IX. Hum Gene Ther 22: 101-106.
34. Lundin, KE, Hasan, M, Moreno, PM, Törnquist, E, Oprea, I, Svahn, MG et al. (2005). Increased stability and specificity through combined hybridization of peptide nucleic acid (PNA) and locked nucleic acid (LNA) to supercoiled plasmids for PNA-Anchored "Bioplex" formation. Biomol Eng 22: 185-192. (Pubitemid 41682013)
35. Svahn, MG, Hasan, M, Sigot, V, Valle-Delgado, JJ, Rutland, MW, Lundin, KE et al. (2007). Self-Assembling supramolecular complexes by single-stranded extension from plasmid DNA. Oligonucleotides 17: 80-94. (Pubitemid 46651430)
36. Verbeeren, J, Niemelä, EH, Turunen, JJ, Will, CL, Ravantti, JJ, Lührmann, R et al. (2010). An ancient mechanism for splicing control: U11 snRNP as an activator of alternative splicing. Mol Cell 37: 821-833.