The use of CpG-free plasmids to mediate persistent gene expression following repeated aerosol delivery of pDNA/PEI complexes

Biomaterials

Volumn 33, Issue 22, 2012, Pages 5618-5627

  Davies, Lee A ^a,b   Hyde, Stephen C ^a,b   Nunez Alonso, Graciela ^a,b   Bazzani, Reto P ^a,b   Harding Smith, Rebekka ^a,b   Pringle, Ian A ^a,b   Lawton, Anna E ^a,b,c   Abdullah, Syahril ^a,d   Roberts, Thomas C ^a,c   McCormick, Dominique ^a,b,e   Sumner Jones, Stephanie G ^a,b   Gill, Deborah R ^a,b  

^a UNIVERSITY OF OXFORD   (United Kingdom)

^b United Kingdom Cystic Fibrosis Gene Therapy Consortium   (United Kingdom)

^c UNIVERSITY OF OXFORD   (United Kingdom)

^d UNIVERSITY PUTRA MALAYSIA   (Malaysia)

^e UNIVERSITY OF EAST ANGLIA   (United Kingdom)

Author keywords

Aerosol; Cystic fibrosis; Cytokine; Gene therapy; Lung; Polyethylenimine

Indexed keywords

AEROSOL DELIVERY; CHRONIC LUNG DISEASE; CYSTIC FIBROSIS; CYTOKINES; GENE DELIVERY; IMMUNE RESPONSE; LUNG; LUNG EPITHELIAL CELLS; MOUSE STRAIN; NONVIRAL GENE THERAPY; PLASMID DNA (PDNA); POLYETHYLENIMINES; REPORTER GENE; SYNTHETIC POLYMERS; TRANSGENE EXPRESSION;

AEROSOLS; BIOLOGICAL ORGANS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; MAMMALS; PULMONARY DISEASES;

ATMOSPHERIC AEROSOLS;

CXCL1 CHEMOKINE; EOTAXIN; GAMMA INTERFERON; GRANULOCYTE COLONY STIMULATING FACTOR; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; INTERLEUKIN 10; INTERLEUKIN 12P40; INTERLEUKIN 12P70; INTERLEUKIN 13; INTERLEUKIN 17; INTERLEUKIN 1ALPHA; INTERLEUKIN 1BETA; INTERLEUKIN 2; INTERLEUKIN 3; INTERLEUKIN 4; INTERLEUKIN 5; INTERLEUKIN 6; INTERLEUKIN 9; LUCIFERASE; MACROPHAGE INFLAMMATORY PROTEIN 1ALPHA; MACROPHAGE INFLAMMATORY PROTEIN 1BETA; MONOCYTE CHEMOTACTIC PROTEIN 1; PLASMID DNA; POLYETHYLENEIMINE; RANTES; TUMOR NECROSIS FACTOR ALPHA;

AEROSOL; ANIMAL EXPERIMENT; ARTICLE; CHRONIC LUNG DISEASE; CONTROLLED STUDY; CPG ISLAND; CYTOTOXICITY; FEMALE; GENE ACTIVITY; GENE DELIVERY SYSTEM; GENE EXPRESSION; IMMUNE RESPONSE; LUNG LAVAGE; MOUSE; NONHUMAN; PRIORITY JOURNAL; REPORTER GENE; TRANSGENE;

ADMINISTRATION, INHALATION; AEROSOLS; ANIMALS; CPG ISLANDS; DRUG CARRIERS; FEMALE; GENE EXPRESSION REGULATION; IMINES; LUNG; MICE; MICE, INBRED BALB C; MICE, INBRED C57BL; PLASMIDS; POLYETHYLENES; TISSUE DISTRIBUTION;

MUS;

EID: 84861223229     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2012.04.019     Document Type: Article

References (38)

1. Patton J.S., Brain J.D., Davies L.A., Fiegel J., Gumbleton M., Kim K.J., et al. The particle has landed-characterizing the fate of inhaled pharmaceuticals. J Aerosol Med Pulm Drug Deliv 2010, (23 Suppl. 2):S71-S87.
2. Densmore C.L., Orson F.M., Xu B., Kinsey B.M., Waldrep J.C., Hua P., et al. Aerosol delivery of robust polyethyleneimine-DNA complexes for gene therapy and genetic immunization. Mol Ther 2000, 1:180-188.
3. Rudolph C., Muller R.H., Rosenecker J. Jet nebulization of PEI/DNA polyplexes: physical stability and in vitro gene delivery efficiency. J Gene Med 2002, 4:66-74.
4. McLachlan G., Davidson H., Holder E., Davies L.A., Pringle I.A., Sumner-Jones S.G., et al. Pre-clinical evaluation of three non-viral gene transfer agents for cystic fibrosis after aerosol delivery to the ovine lung. Gene Ther 2011, 18:996-1005.
5. Rudolph C., Schillinger U., Ortiz A., Plank C., Golas M.M., Sander B., et al. Aerosolized nanogram quantities of plasmid DNA mediate highly efficient gene delivery to mouse airway epithelium. Mol Ther 2005, 12:493-501.
6. Davies L.A., McLachlan G., Sumner-Jones S.G., Ferguson D., Baker A., Tennant P., et al. Enhanced lung gene expression after aerosol delivery of concentrated pDNA/PEI complexes. Mol Ther 2008, 16:1283-1290.
7. Rawlins E.L., Hogan B.L. Ciliated epithelial cell lifespan in the mouse trachea and lung. Am J Physiol Lung Cell Mol Physiol 2008, 295:L231-L234.
8. Gill D.R., Bazzani R.P., Hyde S.C. Strategies for long-term expression of transgenes in the respiratory epithelium. Curr Opin Mol Ther 2010, 12:386-393.
9. Jenkins R.G., Herrick S.E., Meng Q.H., Kinnon C., Laurent G.J., McAnulty R.J., et al. An integrin-targeted non-viral vector for pulmonary gene therapy. Gene Ther 2000, 7:393-400.
10. Rose A.C., Goddard C.A., Colledge W.H., Cheng S.H., Gill D.R., Hyde S.C. Optimisation of real-time quantitative RT-PCR for the evaluation of non-viral mediated gene transfer to the airways. Gene Ther 2002, 9:1312-1320.
11. Hyde S.C., Southern K.W., Gileadi U., Fitzjohn E.M., Mofford K.A., Waddell B.E., et al. Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis. Gene Ther 2000, 7:1156-1165.
12. Wiseman J.W., Goddard C.A., McLelland D., Colledge W.H. A comparison of linear and branched polyethylenimine (PEI) with DCChol/DOPE liposomes for gene delivery to epithelial cells in vitro and in vivo. Gene Ther 2003, 10:1654-1662.
13. Dames P., Ortiz A., Schillinger U., Lesina E., Plank C., Rosenecker J., et al. Aerosol gene delivery to the murine lung is mouse strain dependent. J Mol Med (Berl) 2007, 85:371-378.
14. Gill D.R., Pringle I.A., Hyde S.C. Progress and prospects: the design and production of plasmid vectors. Gene Ther 2009, 16:165-171.
15. Herweijer H., Zhang G., Subbotin V.M., Budker V., Williams P., Wolff J.A. Time course of gene expression after plasmid DNA gene transfer to the liver. J Gene Med 2001, 3:280-291.
16. Hemmi H., Takeuchi O., Kawai T., Kaisho T., Sato S., Sanjo H., et al. A Toll-like receptor recognizes bacterial DNA. Nature 2000, 408:740-745.
17. Yew N.S., Zhao H., Wu I.H., Song A., Tousignant J.D., Przybylska M., et al. Reduced inflammatory response to plasmid DNA vectors by elimination and inhibition of immunostimulatory CpG motifs. Mol Ther 2000, 1:255-262.
18. Hyde S.C., Pringle I.A., Abdullah S., Lawton A.E., Davies L.A., Varathalingam A., et al. CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression. Nat Biotechnol 2008, 26:549-551.
19. Davies L.A., Hannavy K., Davies N., Pirrie A., Coffee R.A., Hyde S.C., et al. Electrohydrodynamic comminution: a novel technique for the aerosolisation of plasmid DNA. Pharm Res 2005, 22:1294-1304.
20. Gautam A., Densmore C.L., Golunski E., Xu B., Waldrep J.C. Transgene expression in mouse airway epithelium by aerosol gene therapy with PEI-DNA complexes. Mol Ther 2001, 3:551-556.
21. Gill D.R., Smyth S.E., Goddard C.A., Pringle I.A., Higgins C.F., Colledge W.H., et al. Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter. Gene Ther 2001, 8:1539-1546.
22. Mombaerts P., Iacomini J., Johnson R.S., Herrup K., Tonegawa S., Papaioannou V.E. RAG-1-deficient mice have no mature B and T lymphocytes. Cell 1992, 68:869-877.
23. Gautam A., Densmore C.L., Waldrep J.C. Pulmonary cytokine responses associated with PEI-DNA aerosol gene therapy. Gene Ther 2001, 8:254-257.
24. Lesina E., Dames P., Flemmer A., Hajek K., Kirchner T., Bittmann I., et al. CpG-free plasmid DNA prevents deterioration of pulmonary function in mice. Eur J Pharm Biopharm 2010, 74:427-434.
25. Bazzani R.P., Cai Y., Hebel H.L., Hyde S.C., Gill D.R. The significance of plasmid DNA preparations contaminated with bacterial genomic DNA on inflammatory responses following delivery of lipoplexes to the murine lung. Biomaterials 2011, 32:9854-9865.
26. Wooddell C.I., Subbotin V.M., Sebestyen M.G., Griffin J.B., Zhang G., Schleef M., et al. Muscle damage after delivery of naked plasmid DNA into skeletal muscles is batch dependent. Hum Gene Ther 2011, 22:225-235.
27. Sonawane N.D., Szoka F.C., Verkman A.S. Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes. J Biol Chem 2003, 278:44826-44831.
28. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 1995, 92:7297-7301.
29. Iida T., Mori T., Katayama Y., Niidome T. Overall interaction of cytosolic proteins with the PEI/DNA complex. J Control Release 2007, 118:364-369.
30. Beyerle A., Braun A., Banerjee A., Ercal N., Eickelberg O., Kissel T.H., et al. Inflammatory responses to pulmonary application of PEI-based siRNA nanocarriers in mice. Biomaterials 2011, 32:8694-8701.
31. Thomas M., Lu J.J., Zhang C., Chen J., Klibanov A.M. Identification of novel superior polycationic vectors for gene delivery by high-throughput synthesis and screening of a combinatorial library. Pharm Res 2007, 24:1564-1571.
32. Zhao D., Gong T., Zhu D., Zhang Z., Sun X. Comprehensive comparison of two new biodegradable gene carriers. Int J Pharm 2011, 413:260-270.
33. Davies J.C., Davies G., Gill D., Hyde S., Boyd A.C., Innes J.A., et al. Safety and expression of a single dose of lipid-mediated CFTR gene therapy to the upper and lower airway of patients with CF. Pediatr Pulmonol 2011, 46:281.
34. Gunther M., Lipka J., Malek A., Gutsch D., Kreyling W., Aigner A. Polyethylenimines for RNAi-mediated gene targeting in vivo and siRNA delivery to the lung. Eur J Pharm Biopharm 2011, 77:438-449.
35. Ge Q., Filip L., Bai A., Nguyen T., Eisen H.N., Chen J. Inhibition of influenza virus production in virus-infected mice by RNA interference. Proc Natl Acad Sci USA 2004, 101:8676-8681.
36. Anchordoquy T.J., Koe G.S. Physical stability of nonviral plasmid-based therapeutics. J Pharm Sci 2000, 89:289-296.
37. Davies L., Nunez-Alonso G., Hebel H., Scheule R., Cheng S., Hyde S., et al. A novel mixing device for the reproducible generation of nonviral gene therapy formulations. Biotechniques 2010, 49:666-668.
38. Pfeifer C., Hasenpusch G., Uezguen S., Aneja M.K., Reinhardt D., Kirch J., et al. Dry powder aerosols of polyethylenimine (PEI)-based gene vectors mediate efficient gene delivery to the lung. J Control Release 2011, 154:69-76.