Nuclear-targeted minicircle to enhance gene transfer with non-viral vectors in vitro and in vivo

Journal of Gene Medicine

Volumn 8, Issue 6, 2006, Pages 754-763

  Vaysse, Laurence ^a   Gregory, Lisa G ^a   Harbottle, Richard P ^a   Perouzel, Eric ^b   Tolmachov, Oleg ^a   Coutelle, Charles ^a  

^a IMPERIAL COLLEGE LONDON   (United Kingdom)

^b IC Vec Ltd   (United Kingdom)

Author keywords

DNA binding protein; Gene transfer; Minicircle; Nuclear localisation signal; Tat peptide

Indexed keywords

1,2 DIOLEOYL 3 TRIMETHYLAMMONIOPROPANE; BETA GALACTOSIDASE; CHOLESTEROL; HYBRID PROTEIN; LIPOSOME; PLASMID DNA; REPRESSOR PROTEIN; SIGNAL PEPTIDE; TETRACYCLINE; TRANSACTIVATOR PROTEIN;

AMINO ACID SEQUENCE; ANIMAL EXPERIMENT; ARTICLE; BINDING AFFINITY; CELL CYCLE ARREST; CONTROLLED STUDY; DNA BINDING; DNA SEQUENCE; ENZYME ACTIVITY; FEMALE; GENE VECTOR; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NUCLEAR IMPORT; NUCLEAR LOCALIZATION SIGNAL; PRIORITY JOURNAL; PROTEIN EXPRESSION; SIMIAN VIRUS 40;

ANIMALS; BETA-GALACTOSIDASE; CELL NUCLEUS; DNA, CIRCULAR; FEMALE; GENE EXPRESSION; GENETIC VECTORS; MICE; NIH 3T3 CELLS; TETRACYCLINE; TRANSFECTION; TRANSGENES;

HUMAN IMMUNODEFICIENCY VIRUS 1;

EID: 33745275571     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.883     Document Type: Article

References (36)

1. Nishikawa M, Huang L. Nonviral vectors in the new millennium: delivery barriers in gene transfer. Hum Gene Ther 2001; 12: 861-870.
2. Li S, Ma Z. Nonviral gene therapy. Curr Gene Ther 2001; 1: 201-226.
3. Morris MC, Chaloin L, Heitz F, Divita G. Translocating peptides and proteins and their use for gene delivery. Curr Opin Biotechnol 2000; 11: 461-466.
4. Vaysse L, Harbottle R, Bigger B, et al. Development of a self-assembling nuclear targeting vector system based on the tetracycline repressor protein. J Biol Chem 2004; 279: 5555-5564.
5. Vives E, Brodin P, Lebleu B. A truncated HIV-1 Tat protein basic domain rapidly translocates through the plasma membrane and accumulates in the cell nucleus. J Biol Chem 1997; 272: 16010-16017.
6. Nagahara H, Vocero-Akbani AM, Snyder EL, et al. Transduction of full-length TAT fusion proteins into mammalian cells: TAT-p27Kip1 induces cell migration. Nat Med 1998; 4: 1449-1452.
7. Futaki S, Suzuki T, Ohashi W, et al. Arginine-rich peptides. An abundant source of membrane-permeable peptides having potential as carriers for intracellular protein delivery. J Biol Chem 2001; 276: 5836-5840.
8. Efthymiadis A, Briggs LJ, Jans DA. The HIV-1 Tat nuclear localization sequence confers novel nuclear import properties. J Biol Chem 1998; 273: 1623-1628.
9. Truant R, Cullen BR. The arginine-rich domains present in human immunodeficiency virus type 1 Tat and Rev function as direct importin beta-dependent nuclear localization signals. Mol Cell Biol 1999; 19: 1210-1217.
10. Herweijer H, Zhang G, Subbotin VM, et al. Time course of gene expression after plasmid DNA gene transfer to the liver. J Gene Med 2001; 3: 280-291.
11. Tan Y, Li S, Pitt BR, Huang L. The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivo. Hum Gene Ther 1999; 10: 2153-2161.
12. Li S, Wu SP, Whitmore M, et al. Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors. Am J Physiol 1999; 276: L796-804.
13. Yew NS, Zhao H, Wu IH, et al. Reduced inflammatory response to plasmid DNA vectors by elimination and inhibition of immunostimulatory CpG motifs. Mol Ther 2000; 1: 255-262.
14. Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther 2004; 11: 856-864.
15. Bigger BW, Tolmachov O, Collombet JM, et al. An araC-controlled bacterial cre expression system to produce DNA minicircle vectors for nuclear and mitochondrial gene therapy. J Biol Chem 2001; 276: 23018-23027.
16. Darquet AM, Rangara R, Kreiss P, et al. Minicircle: an improved DNA molecule for in vitro and in vivo gene transfer. Gene Ther 1999; 6: 209-218.
17. Chen ZY, He CY, Ehrhardt A, Kay MA. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 2003; 8: 495-500.
18. Gossen M, Freundlieb S, Bender G, et al. Transcriptional activation by tetracyclines in mammalian cells. Science 1995; 268: 1766-1769.
19. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999; 6: 1258-1266.
20. Tan Y, Liu F, Li Z, et al. Sequential injection of cationic liposome and plasmid DNA effectively transfects the lung with minimal inflammatory toxicity. Mol Ther 2001; 3: 673-682.
21. Gossen M, Bujard H. Tight control of gene expression in mammalian cells by tetracycline-responsive promoters. Proc Natl Acad Sci U S A 1992; 89: 5547-5551.
22. Song YK, Liu F, Liu D. Enhanced gene expression in mouse lung by prolonging the retention time of intravenously injected plasmid DNA. Gene Ther 1998; 5: 1531-1537.
23. Li J, Ma Z, Tang ZL, et al. CpG DNA-mediated immune response in pulmonary endothelial cells. Am J Physiol Lung Cell Mol Physiol 2004; 287: L552-558.
24. Reyes-Sandoval A, Ertl HC. CpG methylation of a plasmid vector results in extended transgene product expression by circumventing induction of immune responses. Mol Ther 2004; 9: 249-261.
25. Ludtke JJ, Zhang G, Sebestyen MG, Wolff JA. A nuclear localization signal can enhance both the nuclear transport and expression of 1 kb DNA. J Cell Sci 1999; 112: 2033-2041.
26. Zanta MA, Belguise-Valladier P, Behr JP. Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc Natl Acad Sci U S A 1999; 96: 91-96.
27. Pollard H, Remy JS, Loussouarn G, et al. Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells. J Biol Chem 1998; 273: 7507-7511.
28. Lechardeur D, Sohn KJ, Haardt M, et al. Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer. Gene Ther 1999; 6: 482-497.
29. Leifert JA, Harkins S, Whitton JL. Full-length proteins attached to the HIV tat protein transfuction domain are neither transduced between cells, nor exhibit enhanced immunogenicity. Gene Ther 2002; 9: 1422-1428.
30. Ferrari A, Pellegrini V, Arcangeli C, et al. Caveolae-mediated internalization of extracellular HIV-1 tat fusion proteins visualized in real time. Mol Ther 2003; 8: 284-294.
31. Richard JP, Melikov K, Vives E, et al. Cell-penetrating peptides. A reevaluation of the mechanism of cellular uptake. J Biol Chem 2003; 278: 585-590.
32. Vives E. Cellular uptake of the Tat peptide: an endocytosis mechanism following ionic interactions. J Mol Recognit 2003; 16: 265-271.
33. Caron NJ, Quenneville SP, Tremblay JP. Endosome disruption enhances the functional nuclear delivery of Tat-fusion proteins. Biochem Biophys Res Commun 2004; 319: 12-20.
34. Rudolph C, Plank C, Lausier J, et al. Oligomers of the arginine-rich motif of the HIV-1 TAT protein are capable of transferring plasmid DNA into cells. J Biol Chem 2003; 278: 11411-11418.
35. Hyndman L, Lemoine JL, Huang L, et al. HIV-1 Tat protein transduction domain peptide facilitates gene transfer in combination with cationic liposomes. J Control Release 2004; 99: 435-444.
36. Conti E, Izaurralde E. Nucleocytoplasmic transport enters the atomic age. Curr Opin Cell Biol 2001; 13: 310-319.