Solvoplex: A new type of synthetic vector for intrapulmonary gene delivery

Human Gene Therapy

Volumn 10, Issue 18, 1999, Pages 2891-2905

  Schughart, K ^a   Bischoff, R ^a,b   Rasmussen, U B ^a   Ali Hadji, D ^a   Perraud, F ^a   Accart, N ^a   Boussif, O ^a   Silvestre, N ^a   Cordier, Y ^a   Pavirani, A ^a   Kolbe, H V J ^a  

^a TRANSGENE S A   (France)

^b Astra Draco AB ^*  (Sweden)

Author keywords

[No Author keywords available]

Indexed keywords

ARTICLE; GENE TARGETING; GENE THERAPY; REPORTER GENE; RESPIRATORY EPITHELIUM;

ANIMALS; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; LUCIFERASES; LUNG; MICE; RATS;

ADENOVIRIDAE;

EID: 0033544903     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430349950016311     Document Type: Article

References (60)

1. ABDALLAH, B., SACHS, L., and DEMENEIX, B.A. (1995). Non-viral gene transfer: Applications in developmental biology and gene therapy. Biol. Cell 85, 1-7.
2. ABERLE, A.M., BENNETT, M.J., MALONE, R.W., and NANTZ, M.H. (1996). The counterion influence on cationic lipid-mediated transfection of plasmid DNA. Biochim. Biophys. Acta 1299, 281-283.
3. ALTON, E.W., MIDDLETON, P.G., CAPLEN, N.J., SMITH, S.N., STEEL, D.M., MUNKONGE, F.M., JEFFERY, P.K., GEDDES, D.M., HART, S.L., WILLIAMSON, R., FASOLD, K.I., MILLER, A.D., DICKINSON, P., STEVENSON, B.J., McLACHLAN, G., DORIN, J.R., and PORTEUS, D.J. (1993). Non-invasive liposome-mediated gene delivery can correct the ion transport defect in cystic fibrosis mutant mice. Nature Genet. 5, 135-142.
4. ALTON, E.W., GEDDES, D.M., GILL, D.R., HIGGINS, C.F., HYDE, S.C., INNES, J.A., and PORTEOUS, D.J. (1998). Towards gene therapy for cystis fibrosis: A clinical progress report. Gene Ther. 5, 291-292.
5. ARMENTANO, D., ZABNER, J., SACKS, C., SOOKDEO, C.C., SMITH, M.P., ST. GEORGE, J.A., WADSWORTH, S.C., SMITH, A.E., and GREGORY, R.J. (1997). Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J. Virol. 71, 2408-2416.
6. BARRON, L.G., MEYER, K.B., and SZOKA, F.C., JR. (1998). Effects of complement depletion on the pharmacokinetics and gene delivery mediated by cationic lipid-DNA complexes. Hum. Gene Ther. 9, 315-323.
7. BELLON, G., MICHEL-CALEMARD, L., THOUVENOT, D., JAGNEAUX, V., POITEVIN, F., MALCUS, C., ACCART, N., LAYANI, M.P., AYMARD, M., BERNON, H., BIENVENU, J., COURTNEY, M., DÖRING, G., GILLY, B., GILLY, R., LAMY, D., LEVREY, H., MOREL, Y., PAULIN, C., PERRAUD, F., RODILLON, L., SENÉ, C., SO, S., TOURAINE-MOULIN, F., SCHATZ, C., and PAVIRANI, A. (1997). Aerosol administration of a recombinant adenovirus expressing CFTR to cystic fibrosis patients: A phase I clinical trial. Hum. Gene Ther. 8, 15-25.
8. BRIGHAM, K.L., MEYRICK, B., CHRISTMAN, B., MAGNUSON, M., KING, G., and BERRY, L.C., JR. (1989). In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298, 278-281.
9. 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. J. Appl. Physiol. 77, 415-419.
10. CAPLEN, N.J., ALTON, E.W., MIDDLETON, P.G., DORIN, J.R., STEVENSON, B.J., GAO, X., DURHAM, S.R., JEFFERY, P.K., HODSON, M.E., COUTELLE, C., HUANG, L., PORTEOUS, D.J., WILLIAMSON, R., and GEDDES, D.M. (1995). Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med. 1, 39-46.
11. CHU, Q., TOUSIGNANT, J.D., FANG, S., JIANG, C., CHEN, L.H., CHENG, S.H., SCHEULE, R.K., and EASTMAN, S.J. (1999). Binding and DNA uptake of cationic lipid:pDNA complexes by polarized airway epithelial cells. Hum. Gene Ther. 10, 25-36.
12. DEDIEU, J.F., VIGNE, E., TORRENT, C., JULLIEN, C., MAHFOUZ, I., CAILLAUD, J.M., AUBAILLY, N., ORSINI, C., GUILLAUME, J.M., OPOLON, P., DELAERE, P., PERRICAUDET, M., and YEH, P. (1997). Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses. J. Virol. 71, 4626-4637.
13. DEMOLY, P., MATHIEU, M., CURIEL, D.T., GODARD, P., BOUSQUET, J., and MICHEL, F.B. (1997). Gene therapy strategies for asthma. Gene Ther. 4, 507-516.
14. 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. (1997a). A concentrated and stable aerosol formulation of cationic lipid:DNA complexes giving high-level gene expression in mouse lung. Hum. Gene Ther. 8, 765-773.
15. EASTMAN, S.J., TOUSIGNANT, J.D., LUKASON, M.J., MURRAY, H., SIEGEL, C.S., CONSTANTINO, P., HARRIS, D.J., CHENG, S.H., and SCHEULE, R.K. (1997b). Optimization of formulations and conditions for the aerosol delivery of functional cationic lipid:DNA complexes. Hum. Gene Ther. 8, 313-322.
16. EASTMAN, S.J., TOUSIGNANT, J.D., LUKASON, M.J., CHU, Q., CHENG, S.H., and SCHEULE, R.K. (1998). Aerosolization of cationic lipid:pDNA complexes - in vitro optimization of nebulizer parameters for human clinical studies. Hum. Gene Ther. 9, 43-52.
17. FASSENDER, A., MARSHALL, J., MONINGER, T.O., GRUNST, T., CHENG, S., and WELSH, M.J. (1997). Effect of co-lipids in enhancing cationic lipid-mediated gene transfer in vitro and in vivo. Gene Ther. 4, 716-725.
18. FELGNER, P.L., TSAI, Y.L., SUKHU, L., WHEELER, C.J., MANTHROPE, M., MARSHALL, J., and CHENG, S.H. (1995). Improved cationic lipid formulations for in vivo gene therapy. Ann. N.Y. Acad. Sci. 772, 126-139.
19. FERRARI, S., MORO, E., PETTENAZZO, A., BEHR, J.P., ZACCHELLO, F., and SCARPA, M. (1997). ExGen500 is an efficient vector for gene delivery to lung epithelial cells in vitro and in vivo. Gene Ther. 4, 1100-1106.
20. FORTUNATI, E., BOUT, A., ZANTA, M.A., VALERIO, D., and SCARPA, M. (1996). In vitro and in vivo gene transfer to pulmonary cells mediated by cationic liposomes. Biochim. Biophys. Acta 1306, 55-62.
21. FRIZZELL, R.A. (1995). Functions of the cystic fibrosis transmembrane regulator protein. Am. J. Respir. Crit. Care Med. 151, 554-558.
22. GILL, D.R., SOUTHERN, K.W., MOFFORD, K.A., SEDDON, T., HUANG, L., SORGI, F., THOMSON, A., MACVINISH, L.J., RATCLIFF, R., BILTON, D., LANE, D.J., LITTLEWOOD, J.M., WEBB, A.K., MIDDLETON, P.C., COLLEDGE, W.H., CUTHBERT, A.W., EVANS, M.J., HIGGINS, C.F., and HYDE, S.C. (1997). A placebo-controlled study of liposome-mediated gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4, 199-209.
23. GUILLAUME-GABLE, C., FLOCH, V., MERCIER, B., AUDREZET, M., GOBIN, E., LEBLOCH, G., YAOUANC, J., CLEMENT, J., DES ABBAYES, H., LEROY, J., MORIN, V., and FEREC, C. (1998). Cationic phosphonolipids as nonviral gene transfer agents in the lungs of mice. Hum. Gene Ther. 9, 2309-2319.
24. HYDE, S.C., GILL, D.R., HIGGINS, C.F., TREZISE, A.E., MACVINISH, L.J., CUTHBERT, A.W., RATCLIFF, R., EVANS, M.J., and COLLEDGE, W.H. (1993). Correction of the ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature (London) 362, 250-255.
25. JIANG, C., O'CONNOR, S.P., FANG, S.L., WANG, K.X., MARSHALL, J., WILLIAMS, J.L., WILBURN, B., ECHELARD, Y., and CHENG, S.H. (1998). Efficiency of cationic lipid-mediated transfection of polarised and differentiated airway epithelial cells in vitro and in vivo. Hum. Gene Ther. 9, 1531-1542.
26. JOBE, A.H., IKEGAMI, M., YEI, S., WHITSETT, J.A., and TRAPNELL, B. (1996). Surfactant effects on aerosolized and instilled adenoviral-mediated gene transfer. Hum. Gene Ther. 7, 697-704.
27. JOHNSON, L.G., OLSEN, J.C., SARKADI, B., MOORE, K.L., SWANSTROM, R., and BOUCHER, R.C. (1992). Efficiency of gene transfer for restoration of normal airway epithelial function in cystic fibrosis. Nature Genet. 2, 21-25.
28. KABANOV, A.V., FELGNER, P.L., and SEYMOUR, L.W. (1998). Self-Assembling Complexes for Gene Delivery. John Wiley & Sons, Chichester, UK.
29. KATKIN, J.P., HUSSER, R.C., LANGSTON, C., and WELTY, S.E. (1997). Exogenous surfactant enhances the delivery of recombinant adenoviral vectors to the lung. Hum. Gene Ther. 8, 171-176.
30. KORST, R.J., McELVANEY, N.G., CHU, C.S., ROSENFELD, M.A., MASTRANGELI, J.H., BRODY, S.L., EISSA, N.T., DANEL, C., JAFFE, H.A., and CRYSTAL, R.G. (1995). Gene therapy for the respiratory manifestations of cystic fibrosis. Am. J. Respir. Crit. Care Med. 151, S75-S87.
31. 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. Hum. Gene Ther. 7, 1701-1717.
32. LEE, R.J., and HUANG, L. (1997). Lipidic vector systems for gene transfer. Crit. Rev. Ther. Drug Carrier Syst. 14, 173-206.
33. LISBY, D.A., BALLARD, P.L., FOX, W.W., WOLFSON, M.R., SHAFFER, T.H., and GONZALES, L.W. (1997). Enhanced distribution of adenovirus-mediated gene transfer to lung parenchyma by perfluorochemical liquid. Hum. Gene Ther. 8, 919-928.
34. LOGAN, J.J., BEBOK, Z., WALKER, L.C., PENG, S., FELGNER, P.L., SIEGAL, G.P., FRIZZELL, R.A., DONG, J., HOWARD, M., MATALON, S., LINDSEY, J.R., DUVALL, M., and SORSCHER, E.J. (1995). Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium. Gene Ther. 2, 38-49.
35. LUSKY, M., CHRIST, M., RITTNER, K., DIETERLE, A., DREYER, D., MOUROT, B., SCHULTZ, H., STOECKEL, F., PAVIRANI, A., and MEHTALI, M. (1998). In vitro and in vivo biology of recombinant adenovirus vectors with E1, E1/E2A, or E1/E4 deleted. J. Virol. 72, 2022-2032.
36. MAHATO, R.I., ROLLAND, A., and TOMLINSON, E. (1997). Cationic lipid-based gene delivery systems: Pharmaceutical perspectives. Pharm. Res. 14, 853-859.
37. McLACHLAN, G., DAVIDSON, D.J., STEVENSON, BJ., DICKINSON, P., DAVIDSON-SMITH, H., DORIN, J.R., and PORTEOUS, D.J. (1995). Evaluation in vitro and in vivo of cationic liposome-expression construct complexes for cystic fibrosis gene therapy. Gene Ther. 2, 614-622.
38. McLACHLAN, G., HO, L.P., DAVIDSON-SMITH, H., SAMWAYS, J., DAVIDSON, H., STEVENSON, B.J., CAROTHERS, A.D., ALTON, E.W., MIDDLETON, P.G., SMITH, S.N., KALLMEYER, G., MICHAELIS, U., SEEBER, S., NAUJOKS, K., GREENING, A.P., INNES, J.A., DORIN, J.R., and PORTEOUS, D.J. (1996). Laboratory and clinical studies in support of cystic fibrosis gene therapy using pCMV-CFTR-DOTAP. Gene Ther. 3, 1113-1123.
39. MEYER, K.B., THOMPSON, M.M., LEVY, M.Y., BARRON, L.G., and SZOKA, F.C. (1995). Intratracheal gene delivery to the mouse airway: Characterisation of plasmid DNA expression and pharmacokinetics. Gene Ther. 2, 450-460.
40. OUDRHIRI, N., VIGNERON, J.P., PEUCHMAUR, M., LECLERC, T., LEHN, J.M., and LEHN, P. (1997). Gene transfer by guanidinium-cholesterol cationic lipids into airway epithelial cells in vitro and in vivo. Proc. Natl. Acad. Sci. U.S.A. 94, 1651-1656.
41. PARSONS, D.W., GRUBB, B.R., JOHNSON, L.G., and BOUCHER, R.C. (1998). Enhanced in vivo airway gene transfer via transient modification of host barrier properties with a surface-active agent. Hum. Gene Ther. 9, 2661-2672.
42. PORTEOUS, D.J., DORIN, J.R., MCLACHLAN, G., DAVIDSON-SMITH, H., DAVIDSON, H., STEVENSON, B.J., CAROTHERS, A.D., WALLACE, W.A., MORALEE, S., HOENES, C., KALLMEYER, G., MICHAELIS, U., NAUJOKS, K., HO, L.P., SAMWAYS, J.M., IMRIE, M., GREENING, A.P., and INNES, J.A. (1997). Evidence for safety and efficacy of DOTAP cationic liposome mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Gene Ther. 4, 210-218.
43. RACZKA, E., KUKOWSKA-LATALLO, J.F., RYMASZEWSKI, M., CHEN, C., and BAKER, J.R., JR. (1998). The effect of synthetic surfactant Exosurf on gene transfer in mouse lung in vivo. Gene Ther. 5, 1333-1339.
44. ROLLAND, A.P. (1998). From genes to gene medicines: Recent advances in nonviral gene delivery. Crit. Rev. Ther. Drug Carrier Syst. 15, 143-198.
45. ROSENFELD, M.A., and COLLINS, F.S. (1996). Gene therapy for cystis fibrosis. Chest 109, 241-252.
46. SAWA, T., MIYAZAKI, H., PITTET, J.F., WIDDICOMBE, J.H., CROPPER, M.A., HASHIMOTO, S., CONRAD, D.J., FOLKESSON, H.G., DEBS, R., FORSAYETH, J.R., FOX, B., and WIENER-KRONISH, J.P. (1996). Intraluminal water increases expression of plasmid DNA in rat lung. Hum. Gene Ther. 7, 933-941.
47. SCHERMAN, D., BESSODES, M., CAMERON, B., HERSCOVICI, J., HOFLAND, H., PITARD, B., SOUBRIER, F., WILS, P., and CROUZET, J. (1998). Application of lipids and plasmid design for gene delivery to mammalian cells. Curr. Opin. Biotechnol. 9, 480-485.
48. SCHEULE, R.K., 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. Hum. Gene Ther. 8, 689-707.
49. STRATFORD-PERRICAUDET, L.D., MAKEH, I., PERRICAUDET, M., and BRIAND, P. (1992). Widespread long-term gene transfer to mouse skeletal muscles and heart. J. Clin. Invest. 90, 626-630.
50. STRIBLING, R., BRUNETTE, E., LIGGITT, D., GAENSLER, K., and DEBS, R. (1992). Aerosol gene delivery in vivo. Proc. Natl. Acad. Sci. U.S.A. 89, 11277-11281.
51. TATKEN, R.L., and LEWIS, S.R., JR. (1983). Registry of Toxic Effects of Chemical Substances. Tractor Jitco, Rockville, MD.
52. TRAPNELL, B.C., CHU, C.S., PAAKKO, P.K., BANKS, T.C., YOSHIMURA, K., FERRANS, V.J., CHERNICK, M.S., and CRYSTAL, R.G. (1991). Expression of the cystic fibrosis transmembrane conductance regulator protein in the respiratory tract of normal individuals and individuals with cystic fibrosis. Proc. Natl. Acad. Sci. U.S.A. 88, 6565-6569.
53. TSAN, M.F., TSAN, G.L., and WHITE, J.E. (1997). Surfactant inhibits cationic liposome-mediated gene transfer. Hum. Gene Ther. 8, 817-825.
54. TSUI, L. (1992). The spectrum of cystic fibrosis mutations. Trends Genet. 8, 392-398.
55. 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 the mouse lung. Proc. Natl. Acad. Sci. U.S.A. 93, 11454-11459.
56. YEW, N.S., WANG, K.X., PRZYBYLSKA, M., BAGLEY, R.G., STEDMAN, M., MARSHALL, J., SCHEULE, R., and CHENG, S.H. (1999). Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:DNA complexes. Hum. Gene Ther. 10, 233-234.
57. YOSHIMURA, K., ROSENFELD, M., NAKAMURA, H., SCHERER, E., PAVIRANI, A., LECOCQ, J., and CRYSTAL, R. (1992). Expression of the human cystic fibrosis transmembrane conductance regulator gene in the mouse lung after in vivo intratracheal plasmid-mediated gene transfer. Nucleic Acids Res. 20, 3233-3240.
58. ZABNER, J., CHENG, S.H., MEEKER, D., LANSPACH, J., BALFOUR, R., PERRICONE, M.A., MORRIS, J.E., MARSCHALL, J., FASSENDER, A., SMITH, A.E., and WELSH, M.J. (1997). Comparison of DNA-lipid complexes and DNA alone for gene transfer to cystic fibrosis airway epithelia in vivo. J. Clin. Invest. 100, 1529-1537.
59. ZANTA, M.A., BOUSSIF, O., ADIB, A., and BEHR, J.P. (1997). In vitro gene delivery to hepatocytes with galactosylated polyethylenimine. Bioconj. Chem. 8, 839-844.
60. ZHANG, Y., JIANG, Q., DUDUS, L., YANKASKAS, J.R., and ENGELHARDT, J.F. (1998). Vector-specific complementation profiles of two independent primary defects in cystic fibrosis airways. Hum. Gene Ther. 9, 635-648.