Non-viral gene delivery to the lungs

Current Gene Therapy

Volumn 5, Issue 2, 2005, Pages 181-194

  Kinsey, Berma M ^a   Densmore, Charles L ^a   Orson, Frank M ^a  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

1,2 DIOLEOYL 3 TRIMETHYLAMMONIOPROPANE; CHITOSAN; CHOLESTEROL; DENDRIMER; DEXAMETHASONE; DIPALMITOYLPHOSPHATIDYLCHOLINE; DNA; GAMMA INTERFERON; GENOMIC DNA; INTERLEUKIN 12; INTERLEUKIN 2; KANAMYCIN; LIPID; LIPOFECTIN; LIPOPLEX; LIPOSOME; LYMPHOCYTE ANTIBODY; MACROGOL; MANGANESE SUPEROXIDE DISMUTASE; NAKED DNA; OLIGODEOXYNUCLEOTIDE; PEPTIDE; PLASMID DNA; POLYARGININE; POLYCATION; POLYETHYLENEIMINE; POLYLYSINE; PROTAMINE; PROTEIN P53; UNINDEXED DRUG;

COMPLEX FORMATION; CYSTIC FIBROSIS; DRUG DISTRIBUTION; DRUG FORMULATION; DRUG TARGETING; GENE EXPRESSION; INFLAMMATION; LUNG; LUNG CANCER; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PLASMID; REVIEW; VACCINATION;

ANIMALS; DNA; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LUNG; LUNG DISEASES;

EID: 15944385973     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/1566523053544254     Document Type: Review

References (141)

1. Audouy, S.A., de Leij, L.F.M.H., Hoekstra, D. and Molema, G. (2002). In Vivo Characteristics of Cationic Liposomes as Delivery Vectors for Gene Therapy. Pharm. Res. 19: 1599-1605.
2. Barron, L.G., Gagne, L. and Szoka, F.C.J. (1999). Lipoplex-mediated gene delivery to the lung occurs within 60 minutes of intravenous administration. Human Gene Ther. 10: 1683-1694.
3. Belmont, P., Aissaoui, A., Hauchecorne, M., Oudrhiri, N., Petit, L., Vigneron, J.-P., Lehn, J.-M. and Lehn, P. (2002). Aminoglycoside-derived cationic lipids as efficient vectors for gene transfection in vitro and in vivo. J. Gene Med. 4: 517-526.
4. Blezinger, P., Freimark, B.D., Matar, M., Wilson, E., Singhal, A., Min, W., Nordstrom, J.L. and Pericle, F. (1999). Intratracheal administration of interleukin 12 plasmid-cationic lipid complexes inhibits murine lung metastases. Human Gene Ther. 10 723-731.
5. Boussif, O., Lezoualc'h, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B. and Behr, J.P. (1995). A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo polyethylenimine. PNAS 92: 7297-7301.
6. Bragonzi, A., Boletta, A., Biffi, A., Muggia, A., Sersale, G., Cheng, S.H., Bordignon, C., Assael, B.M. and Conese, M. (1999). Comparison between cationic polymers and lipids in mediating systemic gene delivery to the lungs. Gene Ther. 6: 1995-2004.
7. Bragonzi, A., Dina, G., Villa, A., Calori, G., Biffi, A., Bordignon, C., Assael, B.M. and Conese, M. (2000). Biodistribution and transgene expression with nonviral cationic vector/DNA complexes in the lungs. Gene Ther. 7: 1753-1760.
8. Canonico, A.E., Plitman, J.D., Conary, J.T., Meyrick, B.O. and Brigham, K.L. (1994). No lung toxicity after repeated aerosol or intravenous delivery of plasmid-cationic liposome complexes. 77: 415-419.
9. Chollet, P., Favrot, M.C., Hurbin, A. and Coll, J.-L. (2002). Side-effects of a systemic injection of linear polyethylenimine-DNA complexes. J. Gene Med. 4: 84-91.
10. Cunningham, S., Meng, Q.-H., Klein, N., McAnulty, R.J. and Hart, S.L. (2002). Evaluation of a porcine model for pulmonary gene transfer using a novel synthetic vector. J. Gene Med. 4: 438-446.
11. D'Souza, S., Rosseels, V., Denis, O., Tanghe, A., De Smet, N., Jurion, F., Palfliet, K., Castiglion, N., Vanonckelen, A., Wheeler, C.J. and Huygen, K. (2002). Improved tuberculosis DNA vaccines by formulation in cationic lipids. 70: 3681-3688.
12. Das, A. and Niven, R. (2001). Use of perfluorocarbon (fluorinert) to enhance reporter gene expression following intratracheal instillation into the lungs of Balb/c mice: implications for nebulized delivery of plasmids. 90: 1336-1344.
13. Dash, P.R., Read, M.L., Barrett, L.B., Wolfert, M.A. and Seymour, L.W. (1999). Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery. Gene Ther. 6: 643-650.
14. Dean, D.A., Machado-Aranda, D., Blair-Parks, K., Yeldandi, A.V. and Young, J.L. (2003). Electroporation as a method for high-level nonviral gene transfer to the lung. Gene Ther. 10: 1608-1615.
15. Delépine, P., Guillaume, C., Montier, T., Clément, J.C., Yaouanc, J.J., Des Abbayes, H., Berthou, F., Le Pape, A. and Férec, C. (2003). Biodistribution study of phosphonolipids: a class of non-viral vectors efficient in mice lung-directed gene transfer. J. Gene Med. 5: 600-608.
16. Delépine, P., Montier, T., Guillaume, C., Vaysse, L., Le Pape, A. and Ferec, C. (2002). Visualization of the transgene distribution according to the administration route allows prediction of the transfection efficacy and validation of the results obtained. Gene Ther. 9: 736-739.
17. Densmore, C.L., Giddings, T.H., Waldrep, J.C., Kinsey, B.M. and Knight, V. (1999). Gene transfer by guanidinium-cholesterol: dioleoylphosphatidyl-ethanolamine liposome-DNA complexes in aerosol. J. Gene Med. 1: 251-264.
18. Densmore, C.L., Kleinerman, E.S., Gautam, A., Jia, S.F., Xu, B., Worth, L.L., Waldrep, J.C., Fung, Y.K., T'Ang, A. and Knight, V. (2001). Growth suppression of established human osteosarcoma lung metastases in mice by aerosol gene therapy with PEI-p53 complexes. Cancer Gene Ther. 8: 619-627.
19. Densmore, C.L., Orson, F.M., Xu, B., Kinsey, B.M., Waldrep, J.C., Hua, P.J., Bhogal, B.S. and Knight, V. (2000). Aerosol delivery of robust polyethyleneimine-DNA complexes for gene therapy and genetic immunization. Mol. Ther. 1: 180-188.
20. Densmore, C.L., Xu, B., Gautam, A., Sopata, C., Orson, F., Waldrep, J.C., Kinsey, B. and Knight, V. (2001). Sustained gene expression in the lungs following repeated aerosol delivery of PEI-plasmid DNA despite the presence of a non-CpG-mediated refractory effect. Mol. Ther. 3: S543.
21. Dokka, S., Toledo, D., Shi, X., Castranova, V. and Rojanasakul, Y. (2000). Oxygen radical-mediated pulmonary toxicity induced by some cationic liposomes. Pharm. Res. 17: 521-525.
22. Dow, S.W., Elmslie, R.E., Fradkin, L.G., Liggitt, D.H., Heath, T.D., Willson, A.P. and Potter, T.A. (1999). Intravenous cytokine gene delivery by lipid DNA complexes controls the growth of established lung metastases. Human Gene Ther. 10: 2961-2972.
23. Dow, S.W., Fradkin, L.G., Liggitt, D.H., Willson, A.P., Heath, T.D. and Potter, T.A. (1999). Lipid-DNA complexes induce potent activation of innate immune responses and antitumor activity when administered intravenously. J. Immunol. 163: 1552-1561.
24. Dow, S.W., Schwartze, J., Heath, T.D., Potter, T.A. and Gelfand, E.W. (1999). Systemic and local interferon gamma gene delivery to the lungs for treatment of allergen-induced airway hyperresponsiveness in mice. Human Gene Ther. 10: 1905-1914.
25. Driskell, R. and Engelhardt, J.F. (2003). Current Status of Gene Therapy for Inherited Lung Diseases. Ann. Rev. Physiol. 65 25.1-25.28.
26. Eastman, S.J., Lukason, M.J., Tousignant, J.D., Murray, H., Lane, M.D., St George, J.A., Akita, G.Y., Cherry, M., Cheng, S.H. and Scheule, R.K. (1997). A concentrated and stable aerosol formulation of cationic lipid: DNA complexes giving high-level gene expression in mouse lung. Human Gene Ther. 8: 765-773.
27. Elouahabi, A., Flamand, V., Ozkan, S., Paulart, F., Vandenbranden, M., Goldman, M. and Ruysschaert, J.-M. (2003). Free Cationic Liposomes Inhibit the Inflammatory Response to Cationic Lipid-DNA complex Injected Intravenously and Enhance Its Transfection Efficiency. Mol. Ther. 7: 81-88.
28. Epperly, M.W., Guo, H.L., Jefferson, M., Nie, S., Gretton, J., Bernarding, M., Bar-Sagi, D., Archer, H. and Greenberger, J.S. (2003). Cell phenotype specific kinetics of expression of intratracheally injected manganese superoxide dismutase-plasmid/liposomes (MnSOD-PL) during lung radioprotective gene therapy. Gene Ther. 10: 163-171.
29. Epperly, M.W., Sikora, C.A., DeFilippi, S.J., Gretton, J.E., Bar-Sagi, D., Archer, H., Carlos, T., Guo, H.L. and Greenberger, J.S. (2002). Pulmonary irradiation-induced expression of VCAM-I and ICAM-I is decreased by manganese superoxide dismutase-plasmid/liposome (MnSOD-PL) Gene therapy. 8: 175-187.
30. Fajac, I., Thévenot, G., Bédouet, L., Danel, C., Riquet, M., Merten, M., Figarella, C., Dall' Ava-Santucci, J., Monsigny, M. and Briand, P. (2003). Uptake of plasmid/glycosylated polymer complexes and gene transfer efficiency in differentiated airway epithelial cells. J. Gene Med. 5: 38-48.
31. Ferrari, S., Moro, E., Pettenazzo, A., Behr, J.-P., Zacchello, F. and Scarpa, M. (1997). ExGen 500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther. 4: 1100-6.
32. Ferrari, S., Pettenazzo, A., Garbati, N., Zacchello, F., Behr, J.-P. and Scarpa, M. (1999). Polyethylenimine shows properties of interest of cystic fibrosis gene therapy. 1447: 219-225.
33. Finn, O.J. (2003). Cancer vaccines: Between the Idea and the reality. Nature Rev. Immunol. 3: 630-641.
34. Freimark, B.D., Blezinger, H.P., Florack, V.J., Nordstrom, J.L., Long, S.D., Deshpande, D.S., Nochunson, S. and Petrak, K.L. (1998). Cationic Lipids Enhance Cytokine and Cell Influx Levels in the Lung Following Administration of Plasmid: Cationic Lipid Complexes. J. Immunol. 160: 4580-4586.
35. Gautam, A., Densmore, C.L., Golunski, E., Xu, B. and Waldrep, J.C. (2001). Transgene expression in mouse airway epithelium by aerosol gene therapy with PEI-DNA complexes. Mol. Ther. 3: 551-556.
36. Gautam, A., Densmore, C.L., Melton, S., Golunski, E. and Waldrep, J.C. (2002a). Aerosol delivery of PEI-p53 complexes inhibits B16-F10 lung metastases through regulation of angiogenesis. Cancer Gene Ther. 9: 28-36.
37. Gautam, A., Densmore, C.L. and Waldrep, J.C. (2000). Inhibition of experimental lung metastasis by aerosol delivery of PEI-p53 complexes. Mol. Ther. 2: 318-323.
38. Gautam, A., Densmore, C.L., Xu, B. and Waldrep, J.C. (2000). Enhanced gene expression in mouse lung after PEI-DNA aerosol delivery. Mol. Ther. 2: 63-70.
39. Gautam, A., Waldrep, J.C., Densmore, C.L., Koshkina, N., Melton, S., Roberts, L., Gilbert, B. and Knight, V. (2002b). Growth inhibition of established B16-F10 lung metastases by sequential aerosol delivery of p53 gene and 9-nitrocamptothecin. Gene Ther. 9: 353-357.
40. Gill, D.R., Smyth, S.E., A., G.C., Pringle, I.A., Higgins, C.F., Colledge, W.H. and Hyde, S.C. (2001). Increased persistence of lung gene expression using plasmids containing the ubiquitin C or elongation factor 1alpha promoter. Gene Ther. 8: 1539-1546.
41. Glasspool-Malone, J., Steenland, P.R., McDonald, R.J., Sanchez, R.A., Watts, T.L., Zabner, J. and Malone, R.W. (2002). DNA transfection of macaque and murine respiratory tissue is greatly enhanced by use of a nuclease inhibitor. J. Gene Med. 4: 323-322.
42. Goula, D., Becker, N., Lemkine, G.F., Normandie, P., Rodrigues, J., Mantero, S., Levi, G. and Demeneix, B.A. (2000). Rapid crossing of the pulmonary endothelial barrier by polyethylenimine/DNA complexes. Gene Ther. 7: 499-504.
43. Goula, D., Benoist, C., Mantero, S., Merlo, G., Levi, G. and Demeneix, B.A. (1998). Polyethylenimine-based intravenous delivery of transgenes to mouse lung. Gene Ther. 5: 1291-1295.
44. Griesenbach, U., Chonn, A., Cassady, R., Hamman, V., Ackerly, C., Post, M., Tanswell, A.K., Olek, K., O'Brodovich, H. and Tsui, L.-C. (1998). Comparison between intratracheal and intravenous administration of liposome-DNA complexes for cystic fibrosis lung gene therapy. Gene Ther. 5: 181-188.
45. Gu, Q. and Lee, L.Y. (2001). Hypersensitivity of pulmonary C fibre afferents induced by cationic proteins in the rat. J. Physiol. 537(Pt 3): 887-897.
46. Guillaume, C., Delepine, P., Droal, C., Montier, T., Tymen, G. and Claude, F. (2001). Aerosolization of cationic lipid-DNA complexes: lipoplex characterization and optimization of aerosol delivery conditions. BBA Res. Comm. 286: 464-471.
47. Hofman, C.R., Dileo, J.P., Li, Z., Li, S. and Huang, L. (200 1). Efficient in vivo gene transfer by PCR amplified fragment with recuded inflammatory activity. Gene Ther. 8: 71-74.
48. Huang, L. and Li, S. (1997). Liposomal gene delivery: a complex package. Nat. Biotech. 15: 620-621.
49. Iqbal, M., Lin, W., Jabbal-Gill, I., Davis, S.S., Steward, M.W. and Illum, L. (2003). Nasal delivery of chitosan-DNA plasmid expressing epitopes of respiratory syncytial virus (RSV) induces protective CTL responses in BALB/c mice. Vaccine 21: 1478-1485.
50. Ito, I., Began, G., Mohiuddin, I., Saeki, T., Saito, Y., Branch, C.D., Vaporciyan, A., Stephens, L.C., Yen, N., Roth, J.A. and Ramesh, R. (2003). Increased uptake of liposomal-DNA complexes by lung metastases following intravenous administration. Mol. Ther. 7 409-418.
51. Ito I, Saeki T, Mohuiddin I, Saito Y, Branch CD, Vaporciyan A, Roth JA, Ramesh R. (2004) Persistent transgene expression following intravenous administration of a liposomal complex: role of interleukin-10-mediated immune suppression. Mol. Ther.. 318-327.
52. Jenkins, R.G., Meng, Q.-H., Hodges, R.J., Lee, L.K., Bottoms, S.E.W., Laurent, G.J., Willis, D., Ayazi Shamlou, P., McAnulty, R.J. and Hart, S.L. (2003). Formation of LID vector complexes in water alters physicochemical properties and enhances pulmonary gene expression in vivo. Gene Ther. 10: 1026-1034.
53. Jia, S.F., Worth, L.L., Densmore, C.L., Xu, B., Duan, X. and Kleinerman, E.S. (2003). Aerosol gene therapy with PEI: IL-12 eradicates osteosarcoma lung metastases. Clin Cancer Res. 15: 3462-3468.
54. Jia, S.F., Worth, L.L., Densmore, C.L., Xu, B., Zhou, Z. and Kleinerman, E.S. (2002). Eradication of osteosarcoma lung metastases following intranasal interleukin-12 gene therapy using a nonviral polyethylenimine vector. Cancer Gene Ther. 9: 260-266.
55. Kichler, A., Chillon, M., Leborgne, C., Danos, O. and Frisch, B. (2002). Intranasal gene delivery with a polyethylenimine-PEG conjugate. 81: 379-388.
56. Kircheis, R., Wightman, L. and Wagner, E. (2001). Design and gene delivery activity of modified polyethylenimines. Adv. Drug Deliv. Rev. 53: 341-358.
57. Klavinskis, L.S., Barnfield, C., Gao, L. and Parker, S. (1999). Intranasal immunization with plasmid DNA-lipid complexes elicits mucosal immunity in the female genital and rectal tracts. J. Immunol. 162: 254-262.
58. Koping-Hoggard, M., Mel'nikova, Y.S., Varum, K.M., Lindman, B. and Artursson, P. (2003). Relationship between the physical shape and the efficiency of oligomeric chitosan as a gene delivery system in vitro and in vivo. J. Gene Med. 5: 130-141.
59. Koshkina, N.V., Agoulnik, I.Y., Melton, S.L., Densmore, C.L. and Knight, V. (2003). Biodistribution and pharmacokinetics of aerosol and intravenously administered DNA-polyethyleneimine complexes: optimization of pulmonary delivery and retention. Mol. Ther. 8 249-254.
60. Lee, E.R., Marshall, J., Siegel, C.S., Jiang, C., Yew, N.S., Nichols, M.R., Nietupski, J.B., Ziegler, R.J., Lane, M.B., Wang, K.X., Wan, N.C., Scheule, R.K., Harris, D.J., Smith, A.E. and Cheng, S.H. (1996). Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Human Gene Ther. 7: 1701-1717.
61. Lemkine, G.F. and Demeneix, B.A. (2001). Polyethylenimines for in vivo gene delivery. Curr. Opin. Gene Ther. 3: 178-182.
62. Li, S. and Huang, L. (1997). In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther. 4: 891-900.
63. Li, S., Rizzo, M., Bhattacharya, S. and Huang, L. (1998). Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther. 5: 930-937.
64. Li, S., Tan, Y., Viroonchatapan, E., Pitt, B.R. and Huang, L. (2000). Targeted gene delivery to pulmonary endothelium by anti-PECAM antibody. Am. J. Physiol. Lung. Cell. Mol. Physiol. 278: L504-L511.
65. Li, S., Tseng, W.-C., Stolz, D.B., Wu, S.-P., Watkins, S.C. and Huang, L. (1999). Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection. Gene Ther. 6: 585-594.
66. Li, S., Wu, S.-P., Whitmore, M., Loeffert, E.J., Wang, L., Watkins, S.C., Pitt, B.R. and Huang, L. (1999). Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors. Am. J. Physiol. 276: L796-L804.
67. Li, X., Fu, G.-F., Fan, Y.-R., Shi, C.-F., Liu, X.-J., Xu, G.-X. and Wang, J.-J. (2003). Potent inhibition of angiogenesis and liver tumor growth by administration of an aerosol containing a transferrin-liposome-endostatin complex. World J. Gastroentrol. 9: 262-266.
68. Liu, Y., Thor, A., Shtivelman, E., Cao, Y., Tu, G., Heath, T.D. and Debs, R.J. (1999). Systemic gene delivery expands the repertoire of effective antiangiogenic agents. J. Biol. Chem. 274: 13338-13344.
69. Loisel, S., Le Gall, C., Doucet, L., C., F. and Floch, V. (2001). Contribution of plasmid DNA to hepatotoxicity after systemic administration of lipoplexes. Human Gene Ther. 12: 685-696.
70. Luo, X., Belcastro, R., Cabacungan, J., Hannam, V., Negus, A., Wen, Y., Plumb, J., Hu, J., Steer, B., Koehler, D.R., Downey, G.P. and Tanswell, A.K. (2003). Transfection of lung cells in vitro and in vivo: effect of antioxidants and intraliposomal bFGF. Am. J. Physiol. Lung Cell. Mol. Physiol. January 3, 2003 [epub ahead of print].
71. Ma, Z., Zhang, J., Alber, S., Dileo, J.P., Negishi, Y., Stolz, D.B., Watkins, S.C., Huang, L., Pitt, B.R. and Li, S. (2002). Lipid-mediated delivery of oligonucleotide to pulmonary endothelium. Am. J. Respir. Cell. Mol. Biol. 27: 151-159.
72. Maruyama, K. (2002). PEG-immunoliposomes. Biosci. Rep. 22: 251-266.
73. Matsuura, M., Yamazaki, Y., Sugiyama, M., Kondo, M., Ori, H., Nango, M. and Oku, N. (2003). Polycation liposome-mediated gene transfer in vivo. Biochim. Biophys. Acta 1612: 136-143.
74. McCluskie, M.J., Chu, Y., Xia, J.L., Jessee, J., Gebyehu, G. and Davis, H.L. (1998). Direct gene transfer to the respiratory tract of mice with pure plasmid and lipid-formulated DNA. Antisense Nucleic Acid Drug Dev. 8: 401-414.
75. McDonald, R.J., Liggitt, H.D., Roche, L., Nguyen, H.T., Pearlman, R., Raabe, O.G., Bussey, L.B. and Gorman, C.M. (1998). Aerosol delivery of lipid: DNA complexes to lungs of rhesus monkeys. Pharm. Res. 15: 671-679.
76. McLachlan, G., Stevenson, B.J., Davidson, D.J. and Porteous, D.J. (2000). Bacterial DNA is implicated in the inflammatory response to delivery of DNA/DATAP to mouse lungs. Gene Ther. 7: 384-392.
77. Merdan, T., Kopecek, J. and Kissel, T. (2002). Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv. Drug Deliv. Rev. 54: 715-758.
78. Nakashima, Y., Yano, M., Kobayashi, Y., Moriyama, S., Sasaki, H., Toyama, T., Yamashita, H., Fukai, I., Iwase, H., Yamakawa, Y. and Fujii, Y. (2003). Endostatin gene therapy on murine lung metastases model utilizing cationic vector-mediated intravenous gene delivery. Gene Ther. 10: 123-130.
79. Nicolazzi, C., Mignet, N., de la Figuera, N., Cadet, M., Ibad, R.T., Seguin, J., Scherman, D. and Bessodes, M. (2003). Anionic polyethyleneglycol lipids added to cationic lipoplexes increase their plasmatic circulation time. J. Control. Release 88: 429-443.
80. Niven, R.W., Pearlman, R., Wedeking, T., Mackeigan, J., Noker, P., Simpson-Herren, L. and Smith, J.G. (1998). Biodistribution of radiolabeled lipid-DNA complexes and DNA in mice. J. Pharm. Sci. 87: 1292-1299.
81. Ochietti, B., Guerin, N., Vinogradov, S.V., St-Pierre, Y., Lemieux, P., Kabanov, A.V. and Alakhov, V.Y. (2002). Altered organ accumulation of oligonucleotides using polyethyleneimine grafted with poly(ethylene oxide) or pluronic as carriers. J. Drug Targ. 10 113-1121.
82. Oh, Y.-K., Kim, J.-P., Yoon, H., Kim, J.M., Yang, J.-S. and Kim, C.-K. (2001). Prolonged organ retention and safety of plasmid DNA administered in polyethylenimine complexes. Gene Ther. 8: 1587-1592.
83. Okamoto, H., Nishida, S., Todo, H., Sakakura, Y., Iida, K. and Danjo, K. (2003). Pulmonary gene delivery by chitosan-pDNA complex powder prepared by a supercritical carbon dioxide process. J. Pharm. Sci. 92: 371-380.
84. Orson, F.M., Kinsey, B.M., Hua, P.J., Bhogal, B.S., Densmore, C.L. and Barry, M.A. (2000). Genetic immunization with lung-targeting macro-aggregated polyethyleneimine-albumin conjugates elicits combined systemic and mucosal immune responses. J. Immunol. 164: 6313-6321.
85. Orson, F.M., Song, L., Gautam, A., Densmore, C.L., Bhogal, B.S. and Kinsey, B.M. (2002). Gene delivery to the lung using protein/polyethylenimine/plasmid complexes. Gene Ther. 9: 463-471.
86. Pillai, R., Petrak, K., Blezinger, P., Deshpande, D., Florack, V., Freimark, B.D., Padmabandu, G. and Rolland, A. (1998). Ultrasonic nebulization of cationic lipid-based gene delivery systems for airway administration. Pharm. Res. 15: 1743-1747.
87. Pitard, B., Oudrhiri, N., Lambert, O., Vivien, E., Masson, C., Wetzer, B., Hauchecorne, M., Scherman, D., Rigaud, J.-L., Vigneron, J.-P., Lehn, J.-M. and Lehn, P. (2001). Sterically stabilized BGTC-based lipoplexes: structural features and gene transfection into the mouse airways in vivo. J. Gene Med. 3: 478-487.
88. Pollard, H., Remy, J.S., Loussouarn, G., Demolombe, S., Behr, J.P. and Escande, D. (1998). Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells. J. Biol. Chem. 273: 7507-7511.
89. Raczka, E., Kukowska-Latallo, J.F., Rymaszewski, M., Chen, C. and Baker, J.R.J. (1998). The effect of synthetic surfactant Exosurf on gene transfer in mouse lung in vivo. Gene Ther. 5: 1333-1339.
90. Ramesh, R., Saeki, T., Templeton, N.S., Ji, L., Stephens, L.C., Ito, I., Wilson, D.R., Wu, Z., Branch, C.D., Minna, J.D. and Roth, J.A. (2001). Successful Treatment of Primary and Disseminated Human Lung Cancers by Systemic Delivery of Tumor Suppressor Genes Using an Improved Liposome Vector. Mol. Ther. 3: 337-350.
91. Ren, T., Song, Y.K., Zhang, G. and Liu, D. (2000). Structural basis of DOTMA for its high intravenous transfection activity in mouse. Gene Ther. 7: 764-768.
92. Rittner, K., Benavente, A., Bompard-Sorlet, A., Heitz, F., Divita, G., Brasseur, R. and Jacobs, E. (2002). New basic membrane-destabilizing peptides for plasmid-based gene delivery in vitro and in vivo. Mol. Ther. 5: 104-114.
93. Rochat, T. and Morris, M.A. (2002). Gene therapy for cystic fibrosis by means of aerosol. J. Aerosol Med. 15: 229-235.
94. Rosenecker, J., Naundorf, S., Gersting, S.W., Hauck, R.W., Gessner, A., Nicklaus, P., Muller, R.H. and Rudolph, C. (2003). Interaction of bronchoalveolar lavage fluid with polyplexes and lipoplexes: analysing the role of proteins and glycoproteins. J. Gene Med. 5: 49-60.
95. Rudolph, C., Lausier, J., Naundorf, S., Muller, R.H. and Rosenecker, J. (2000). In vivo gene delivery to the lung using polyethylenimine and fractured polyamidoamine dendrimers. J. Gene Med. 2: 269-278.
96. Rudolph, C., Plank, C., Lausier, J., Schillinger, U., Muller, R.H. and Rosenecker, J. (2003). Oligomers of the arginine-rich motif of the HIV-1 TAT protein are capable of transferring plasmid DNA into cells, J. Biol. Chem. 278: 11411-11418.
97. Rudolph, C., Schillinger, U., Plank, C., Gessner, A., Nicklaus, P., Muller, R.H. and Rosenecker, J. (2002). Nonviral gene delivery to the lung with copolymer-protected and transferrin-modified polyethylenimine. Biochim. Biophys. Acta 1573: 75-83.
98. Ruiz, F.E., Clancy, J.P., Perricone, M.A., Bebok, Z., Hong, J.S., Cheng, S.H., Meeker, D.P., Young, K.R., Schoumacher, R.A., Weatherly, M.R., Wing, L., Morris, J.E., Sindel, L., Rosenberg, M., van Ginkel, F.W., McGhee, J.R., Kelly, D., Lyrene, R.K. and Sorscher, E.J. (2001). A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis. Human Gene Ther. 12 751-761.
99. Sakurai, F., Nishioka, T., Yamashita, F., Takakura, Y. and Hashida, M. (2001). Effects of erythrocytes and serum proteins on lung accumulation of lipoplexes containing cholesterol or DOPE as helper lipid in the single-pass rat lung perfusion system. Eur. J. Pharm. Biopharm. 52: 165-72.
100. Sakurai, F., Terada, T., Yasuda, K., Yamashita, F., Takakura, Y. and Hashida, M. (2002). The role of tissue macrophages in the induction of proinflammatory cytokine production following intravenous injection of lipoplexes. Gene Ther. 9: 1120-1126.
101. Sawa, T., Miyazaki, H., Pittet, J.F., Widdicombe, J.H., Gropper, M.A., Hashimoto, S., Conrad, D.J., Folkesson, H.G., Debs, R., Forsayeth, J.R., Fox, B. and Weiner-Kronish, J.P. (1996). Intraluminal water increases expression of plasmid DNA in rat lung. Hum. Gene Ther. 7: 933-941.
102. Scheule, R.K., St George, J.A., Bagley, R.G., Marshall, J., Kaplan, J.M., Akita, G.Y., Wang, K.X., Lee, E.R., Harris, D.J., Jiang, C., Yew, N.S., Smith, A.E. and Cheng, S.H. (1997). Basis of pulmonary toxicity associated with cationic lipid-mediated gene transfer to the mammalian lung. Human Gene Ther. 8: 689-707.
103. Siders, W.M., Vergillis, K., Johnson, C., Scheule, R.K. and Kaplan, J.M. (2002). Tumor Treatment with Complexes of Cationic Lipid and Noncoding Plasmid DNA results in the Induction of Cytotoxic T Cells and Systemic Tumor Elimination. Mol. Ther. 6: 519-527.
104. Simoes, S., Slepushkin, V., Pires, P., Gaspar, R., Pedroso de Lima, M.C. and Duzgunes, N. (2000). Human serum albumin enhances DNA transfection by lipoplexes and confers resistance to inhibition by serum. Biochim. Biophys. Acta 1463: 459-469.
105. Smith, J.G., Wedeking, T., Vernachio, J.H., Way, H. and Niven, R.W. (1998). Characterization and in vivo testing of a heterogeneous cationic lipid-DNA formulation. Pharm. Res. 15: 1356-1363.
106. Song, L.Y., Ahkong, Q.F., Rong, Q., Wang, Z., Ansell, S., Hope, M.J. and Mui, B. (2002). Characterization of the inhibitory effect of PEG-lipid conjugates on the intracellular delivery of plasmid and antisense DNA mediated by cationic lipid liposomes. Biochim. Biophys. Acta 1558: 1-13.
107. Song, Y.K., Liu, F. and Liu, D. (1998). Enhanced gene expression in mouse lung by prolonging the retention time of intravenously injected plasmid DNA. Gene Ther. 5: 1531-1537.
108. Stammberger, U., Uduehi, A.N., Kubisa, B., Roth, T. and Schmid, R.A. (2002). Non-viral gene delivery to atelectatic and ventilated lungs. Ann. Thoracic Surg. 73: 432-437.
109. Stern, M., Ulrich, K., Geddes, D.M. and Alton, E.W. (2003). Poly (D, L-lactide-co-glycolide)/DNA microspheres to facilitate prolonged transgene expression in airway epithelium in vitro, ex vivo and in vivo. Gene Ther. 10: 1282-1288.
110. Stribling, R., Brunette, E., Liggitt, D.H., Gaensler, K. and Debs, R. (1992). Aerosol gene delivery in vivo. PNAS 89: 11277-11281.
111. Suh, J., Wirtz, D. and Hanes, J. (2003). Efficient active transport of gene nanocarriers to the cell nucleus. PNAS 100: 3878-3882.
112. Sweeney, P., Karashima, T., Ishikura, H., Wiehle, S., Yamashita, M., Benedict, W.F., Cristiano, R.J. and Dinney, C.P. (2003). Efficient therapeutic gene delivery after systemic administration of a novel polyethylenimine/DNA vector in an orthotopic bladder cancer model. Cancer Res. 63: 4017-4020.
113. Tagawa, T., Manvell, M., Brown, N., Keller, M., Perouzel, E., Murray, K.D., Harbottle, R.P., Tecle, M., Booy, F., Brahimi-Horn, M.C., Coutelle, C., Lemoine, N.R., Alton, E.W. and Miller, A.D. (2002). Characterisation of LMD virus-like nanoparticles self-assembled from cationic liposomes, adenovirus core peptide mu and plasmid DNA. Gene Ther. 9: 564-576.
114. Tan, Y. and Huang, L. (2002). Overcoming the inflammatory toxicity of cationic gene vectors. J. Drug Targ. 10: 153-160.
115. Tan, Y., Li, S., Pitt, B.R. and Huang, L. (1999). The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivo. Human Gene Ther. 10: 2153-2161.
116. Tan, Y., Liu, F., Li, Z., Li, S. and Huang, L. (2001). Sequential Injection of Cationic Liposome and Plasmid DNA Effectively Transfects the Lung with Minimal Inflammatory Toxicity. Mol. Ther. 3: 673-682.
117. Tan, Y., Zhang, J.S. and Huang, L. (2002). Codelivery of NF-kappaB Decoy-Related Oligodeoxynucleotide Improves LPD-Mediated Systemic Gene Transfer. Mol. Ther. 6: 804-812.
118. Templeton, N.S., Lasic, D.D., Frederik, P.M., Strey, H.H., Roberts, D.D. and Pavlakis, G.N. (1997). Improved DNA:liposome complexes for increased systemic delivery and gene expression. Nat. Biotech. 15: 647-652.
119. Thomas, M. and Klibanov, A.M. (2003). Non-viral gene therapy: polycation-mediated DNA delivery. Appl. Microbiol. Biotechnol. 62: 27-34.
120. Tousignant, J.D., Gates, A.L., Ingram, L.A., Johnson, C.L., Nietupski, J.B., Cheng, S.H., Eastman, S.J. and Scheule, R.K. (2000). Comprehensive Analysis of the Acute Toxicities Induced by Systemic Administration of Cationic Lipid: Plasmid DNA Complexes in Mice. Human Gene Ther. 11: 2493 - 2513.
121. Tousignant, J.D., Zhao, H., Yew, N.S., Cheng, S.H., Eastman, S.J. and Scheule, R.K. (2003). DNA Sequences in Cationic Lipid: pDNA-Mediated Systemic Toxicities. Human Gene Ther. 14: 203-214.
122. Trubetskoy, V., Wong, S.C., Subbotin, V., Budker, V.G., Loomis, A., Hagstrom, J.E. and Wolff, J.A. (2003). Recharging cationic DNA complexes with highly charged polyanions for in vitro and in vivo gene delivery. Gene Ther. 10: 261-271.
123. Tsan, M.F., Tsan, G.L. and White, J.E. (1997). Surfactant inhibits cationic liposome-mediated gene transfer. Human Gene Ther. 8: 817-825.
124. Tyler, R.C., Fagan, K.A., Unfer, R.C., Gorman, C., McClarrion, M., Bullock, C. and Rodman, D.M. (1999). Vascular inflammation inhibits gene transfer to the pulmonary circulation in vivo. Am. J. Physiol. 277: L1199-L1204.
125. Uduehi, A.N., Stammberger, U., Kubisa, B., Gugger, M., Buehler, T.A. and Schmid, R.A. (2001). Effects of linear polyethylenimine and polyethylenimine/DNA on lung function after airway instillation to rat lungs. Mol. Ther. 4.
126. Uyechi, L.S., Gagne, L., Thurston, G. and Szoka, F.C.J. (2001). Mechanism of lipoplex gene delivery in mouse lung: binding and internalization of fluorescent lipid and DNA components. Gene Ther. 8: 828-836.
127. Vaysse, L., Guillaume, C., Burgelin, I., Gorry, P., Ferec, C. and Arveiler, B. (2002). Proteolipidic vectors for gene transfer to the lung. Biochem. Biophys. Res. Comm. 290: 1489-1498.
128. Ward, C.M., Read, M.L. and Seymour, L.W. (2001). Systemic circulation of poly(L-lysine)/DNA vectors is influenced by polycation molecular weight and type of DNA: differential circulation in mice and rats and the implications for human gene therapy. Blood 97: 2221-2229.
129. Weiss, D.J. (2002). Delivery of Gene Transfer Vectors to Lung: Obstacles and the Role of Adjunct Techniques for Airway Administration. Mol. Ther. 6: 148-142.
130. Wheeler, C.J., Felgner, P.L., Tsai, Y.J., Marshall, J., Sukhu, L., Doh, S.G., Hartikka, J., Nietupski, J., Manthorpe, M., Nichols, M., Plewe, M., Liang, X., Norman, J., Smith, A. and Cheng, S.H. (1996). A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. PNAS 93: 11454-11459.
131. Whitmore, M.M., Li, S. and Falo, L.J. (2001). Systemic administration of LPD prepared with CpG oligonucleotides inhibits the growth of established pulmonary metastases by stimulating innate and acquired antitumor immune responses. Cancer Immunol. Immunother. 50: 503-514.
132. Wightman, L., Kircheis, R., Rossler, V., Carotta, S., Ruzicka, R., Kursa, M. and Wagner, E. (2001). Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J. Gene Med. 3: 362-372.
133. Wiseman, J.W., Goddard, C.A. and College, W.H. (2001). Steroid hormone enhancement of gene delivery to a human airway epithelial cell line in vitro and mouse airways in vivo. Gene Ther. 8 1562-1571.
134. Wiseman, J.W., Goddard, C.A., McLelland, D. and Colledge, W.H. (2003). 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. 10: 1654-1662.
135. Yew, N.S., Wang, K.X., Przybylska, M., Bagley, R.G., Stedman, M., Marshall, J., Scheule, R.K. and Cheng, S.H. (1999). Contribution of Plasmid DNA to Inflammation in the Lung after Administration of Cationic Lipid: pDNA Complexes. Human Gene Ther. 10: 223-234.
136. Yew, N.S., Zhao, H., Przybylska, M., Wu, I.H., Tousignant, J.D., Scheule, R.K. and Cheng, S.H. (2002). CpG-depleted plasmid DNA vectors with enhanced safety and long-term gene expression in vivo. Mol. Ther. 5: 731-738.
137. Zhang, J., Wilson, A., Alber, S., Ma, Z., Tang, Z.L., Satoh, E., Mazda, O., Watkins, S., Huang, L., Pitt, B. and Li, S. (2003). Prolonged gene expression in mouse lung endothelial cells following transfection with Epstein-Barr virus-based episomal plasmid. Gene Ther. 10: 822-826.
138. Ziady, A.G., Ferkol, T., Dawson, D.V., Perlmutter, D.H. and Davis, P.B. (1999). Chain length of the polylysine in receptor-targeted gene transfer complexes affects duration of reporter gene expression both in vitro and in vivo. J. Biol. Chem. 274: 4908-4916.
139. Zinselmeyer, B.H., Beggbie, N., Uchegbu, I.F. and Schatzlein, A.G. (2003). Quantification of beta-galactosidase activity after non-viral transfection in vivo. J. Control. Release 91: 201-208.
140. Zou, S.M., Erbacher, P., Remy, J.S. and Behr, J.P. (2000). Systemic linear polyethylenimine (L-PEI)-mediated gene delivery in the mouse. J. Gene Med. 2: 128-134.
141. Zou, Y., Zong, G., Ling, Y.H. and Perez-Soler, R. (2000). Development of cationic liposome formulations for intratracheal gene therapy of early lung cancer. Cancer Gene Ther. 7: 683-696.