Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung

Human Gene Therapy

Volumn 7, Issue 14, 1996, Pages 1701-1717

  Lee, Edward R ^a   Marshall, John ^a   Siegel, Craig S ^a   Jiang, Canwen ^a   Yew, Nelson S ^a   Nichols, Margaret R ^a   Nietupski, Jennifer B ^a   Ziegler, Robin J ^a   Lane, Mathieu B ^a   Wang, Kathryn X ^a   Wan, Nick C ^a   Scheule, Ronald K ^a   Harris, David J ^a   Smith, Alan E ^a   Cheng, Seng H ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMPHOPHILE; CATION; CHLORAMPHENICOL ACETYLTRANSFERASE; CHLORIDE CHANNEL; CHLORIDE ION; CHOLESTEROL; LIPID; PLASMID DNA; SPERMINE; TRANSMEMBRANE CONDUCTANCE REGULATOR;

ADENOVIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CHLORIDE TRANSPORT; CONTROLLED STUDY; CYSTIC FIBROSIS; DNA TRANSFECTION; EXPRESSION VECTOR; FEMALE; FLUID TRANSPORT; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; LUNG; MOUSE; NONHUMAN; RAT;

ADENOVIRIDAE; ANIMALIA; FELIS CATUS; MUS MUSCULUS;

EID: 19244363903     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.14-1701     Document Type: Article

References (55)

1. ALTON, E.W.F.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 PORTEOUS, 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.
2. ARMENTANO, D., SOOKDEO, C.C., HEHIR, K., GREGORY, R.J., ST. GEORGE, J.A., PRINCE, G.A., WADSWORTH, S.C., and SMITH, A.E. (1995). Characterization of an adenovirus gene transfer vector containing an E4 deletion. Hum. Gene Ther. 6, 1343-1353.
3. BEHR, J.P., DEMENEIX, B., LOEFFLER, J.P., and MUTUL, J.P. (1989). Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc. Natl. Acad. Sci. USA 86, 6982-6986.
4. BOAT, T.F., WELSH, M.J., and BEAUDET, A.L. (1989). Cystic fibrosis. In The Molecular Basis of Inherited Disease. C.R. Scriver, A.L. Beaudet, W.S. Sly, and D. Valle, eds. (McGraw-Hill Inc., New York) pp. 2649-2680.
5. BRIGHAM, K.L., MEYRICK, B., CHRISTMAN, B., MAGNUSON, M., KING, G., and BERRY, L.C. (1989). In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298, 278-281.
6. CANONICO, A.E., CONARY, J.T., MEYRICK, B.O., and BRIGHAM, K.L. (1994). Aerosol and intravenous transfection of human a1-antitrypsin gene to lungs of rabbits. Am. J. Respir. Cell Mol. Biol. 10, 24-29.
7. CAPLEN, N.J., ALTON, E.W.F.W., MIDDLETON, P.G., DORIN, J.R., STEVENSON, B.J., GAO, X., DURHAM, S.R., JEFFERY, 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.
8. ENGELHARDT, J.F., YANG, Y., STRATFORD-PERRICAUDET, L.D., ALLEN, E.D., KOZARSKY, K., PERRICAUDET, M., YANKASKAS, J.R., and WILSON, J.M. (1993). Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with E1-deleted adenoviruses. Nature Genet. 4, 27-34.
9. FARHOOD, H., BOTTEGA, R., EPAND, R.M., and HUANG, L. (1992). Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity. Biochim. Biophys. Acta 1111, 239-246.
10. FARHOOD, H., SERBINA, N., and HUANG, L. (1995). The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta 1235, 289-295.
11. FELGNER, P.L., GADEK, T.R., HOLM, M., ROMAN, R., CHAN, H.W., WENZ, M., NORTHROP, J.P., RINGOLD, G.M., and DANIELSON, M. (1987). Lipofection: a highly efficient, lipid-mediated DNA transfection procedure. Proc. Natl. Acad. Sci USA 84, 7413-7417.
12. FELGNER, J.H., KUMAR, R., SRIDHAR, C.N., WHEELER, C.J., TSAI, Y.J., BORDER, R., RAMSEY, P., MARTIN, M., and FELGNER, P.L. (1994). Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269, 2550-2561.
13. GAO, X., and HUANG, L. (1991). A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179, 280-285.
14. GAO, X., and HUANG, L. (1995). Cationic liposome-mediated gene transfer. Gene Ther. 2, 710-722.
15. GAO, X., and HUANG, L. (1996). Potentiation of cationic liposome-mediated gene delivery by polycations. Biochemistry 35, 1027-1036.
16. GORMAN, C.M, MOFFAT, L.F., and HOWARD, B.H. (1982). Recombinant genomes which express chloramphenicol acetyltransferase in mammalian cells. Mol. Cell. Biol. 2, 1044-1051.
17. GRUBB, B.R., PICKLES, R.J., YANKASKAS, J.R., VICK, R.N., ENGELHARDT, J.F., WILSON, J.M., JOHNSON, L.G., and BOUCHER, R.C. (1994). Inefficient gene transfer by adenovirus vector to cystic fibrosis airway epithelia of mice and humans. Nature 371, 802-806.
18. GUY-CAFFEY, J.K., BODEPUDI, V., BISHOP, J.S., JAYARAMAN, K., and CHAUDHARY, N. (1995). Novel polyaminolipids enhance the cellular uptake of oligonucleotides. J. Biol. Chem. 270, 31391-31396.
19. HARMS, J.S., and SPLITTER, G.A. (1995). Interferon-γ inhibits transgene expression driven by SV40 or CMV promoters but augments expression driven by the mammalian MHC I promoter. Hum. Gene Ther. 6, 1291-1297.
20. HASEGAWA, H., SKACK, W., BAKER, O., CALAYAG, M.C., LINGAPPA, V., and VERKMAN, A.S. (1992). A multifunctional aqueous channel formed by CFTR. Science 258, 1477-1479.
21. HAWLEY-NELSON, P., CICCARONE, V., GEBEYEHU, G., and JESSEE, J. (1993). Lipofectamine reagent: A new, higher efficiency polycationic liposome transfection reagent. Focus 15, 73-79.
22. HAY, J.G., MCELVANEY, N.G., HERENA, J., and CRYSTAL, R.G. (1995). Modification of nasal epithelial potential differences of individuals with cystic fibrosis consequent to local administration of a normal CFTR cDNA adenovirus gene transfer vector. Hum. Gene Ther. 6, 1487-1496.
23. HYDE, S.C., GILL, D.R., HIGGINS, C.F., TRESIZE, A.E.O., MACVINISH, L.J., CUTHBERT, A.W., RATCLIFF, R., REVANS, M.J., and COLLEDGE, W.H. (1993). Correction of ion transport defect in cystic fibrosis transgenic mice by gene therapy. Nature 362, 250-255.
24. JIANG, C., O'CONNOR, S.P., ARMENTANO, D., BERTHELETTE, P.B., SCHIAVI, S.C., JEFFERSON, D.M., SMITH, A.E., WADSWORTH, S.C., and CHENG, S.H. (1996). Ability of adenovirus vectors containing different CFTR transcriptional cassettes to correct ion transport defects in CF cells. Am. J. Physiol. (in press).
25. KAMEJI, R., RANNELS, S.R., PEGG, A.E., and RANNELS, E. (1989). Spermidine uptake by type II pulmonary epithelial cells in primary culture. Am. J. Physiol. 256, C160-C167.
26. KNOWLES, M.R., HOHNEKER, K.W., ZHOU, Z., OLSEN, J.C., NOAH, T.L., HU, P.C., LEIGH, M.W., ENGELHARDT, J.F., EDWARDS, L.J., JONES, K.R., GROSSMAN, M., WILSON, J.M., JOHNSON, L.G., and BOUCHER, R.C. (1995). A controlled study of adenoviral-vector-mediated gene transfer in the nasal epithelium of patients with cystic fibrosis. N. Engl. J. Med. 333, 823-831.
27. LEVENTIS, R., and SILVIUS, J.R. (1990). Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles. Biochim. Biophys. Acta 1023, 124-132.
28. LIU, Y., LIGGITT, D., ZHONG, W., TU, G., GAENSLER, K., and DEBS, R. (1995). Cationic liposome-mediated intravenous gene delivery. J. Biol. Chem. 270, 24864-24870.
29. LOGAN, J.L., BEBOK, Z., WALKER, L.C., PENG, S., FELGNER, P.L., SIEGAL, G., FRIZZELL, R.A., DONG, J., HOWARD, M., MATALON, S., LINDSEY, J.R., DUVALL, M., and SORSCHER, E.J. (1994). Cationic lipid for reporter gene and CFTR transfer to rat pulmonary epithelium. Gene Ther. 2, 38-49.
30. McLACHLAN, G., DAVIDSON, D.J., STEVENSON, B.J., DICKINSON, P., DAVIDSON-SMITH, H., DORIN, J., 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.
31. MEYER, K.B., THOMPSON, M.M., LEVY, M.Y., BARRON, L.G., and SZOKA, F.C. (1995). Intratracheal gene delivery to the mouse airway: Characterization of plasmid DNA expression and pharmacokinetics. Gene Ther. 2, 450-460.
32. NABEL, G.J., NABEL, E.G., YANG, Z.Y., FOX, B.A., PLAUTZ, G.E., GAO, X., HUANG, L., SHU, S., GORDON, D., and CHANG, A.E. (1993). Direct gene transfer with DNA-liposome complexes in melanoma: expression, biologic activity, and lack of toxicity in humans. Proc. Natl. Acad. Sci. USA 90, 11307-11311.
33. NABEL, G.J., CHANG, A.E., NABEL, E.G., PLAUTZ, G.E., ENSMINGER, W., FOX, B.A., FELGNER, P., SHU, S., and CHO, K. (1994). Immunotherapy for cancer by direct gene transfer into tumors. Hum. Gene Ther. 5, 57-77.
34. REMY, J.S., SIRLIN, C., VIERLINING, P., and BEHR, J.P. (1994). Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. 5, 1812-1815.
35. REMY, J.S., KICHLER, A., MORDVINOV, V., SCHUBER, F., and BEHR, J.P. (1995). Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial viruses. Proc. Natl. Acad. Sci. USA 92, 1744-1748.
36. RICH, D.P., COUTURE, L.A., CARDOZA, L.M., GUIGGIO, V.M., ARMENTANO, D., ESPINO, P.C., HEHIR, K., WELSH, M.J., SMITH, A.E., and GREGORY, R.J. (1993). Development and analysis of recombinant adenovirus for gene therapy of cystic fibrosis. Hum. Gene Ther. 4, 461-476.
37. ROSENFELD, M.A., CHU, C.S., SETH, P., DANEL, C., BANKS, T., YONEYAMA, K., YOSHIMURA, K., and CRYSTAL, R.G. (1994). Gene transfer to freshly isolated human respiratory epithelial cells in vitro using a replication-defective adenovirus containing the human cystic fibrosis transmembrane conductance regulator cDNA. Hum. Gene Ther. 5, 331-342.
38. SANES, J.R., RUBENSTEIN, L.R., and NICOLA, J.F. (1986). Use of a recombinant retrovirus to study post-implantation cell lineage in mouse embryos. EMBO J. 5, 3133-3142.
39. SCHIAVI, S.C., ABDELKADER, N., REBER, S., PENNINGTON, S., NARAYANA, R., McPHERSON, J.M., SMITH, A.E., HOPPE, H., and CHENG, S.H. (1996). Biosynthetic and growth abnormalities are associated with high-level expression of CFTR in heterologous cells. Am. J. Physiol. 270, C341-C351.
40. SHARMA, S.K., MILLER, M.J., and PAYNE, S.M. (1989). Spermexatin and spermexatol: new synthetic spermidine-based siderophore analogues. J. Med. Chem. 32, 357-367.
41. SHEPPARD, D.N., CARSON, M.R., OSTEDGAARD, L.S., DENNING, G.M., and WELSH, M.J. (1994). Expression of cystic fibrosis transmembrane conductance regulator in a model epithelium. Am. J. Physiol. 266, L405-L413.
42. SMITH, J.J., and WELSH, M.J. (1993). Fluid and electrolyte transport by cultured human airway epithelia. J. Clin. Invest. 91, 1590-1597.
43. SOLODIN, I., BROWN, C.S., BRUNO, M.S., CHOW, C.Y., JANG, E., DEBS, R.J., and HEATH, T.D. (1995). A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery. Biochemistry 34, 13537-13544.
44. SORSCHER, E.J., LOGAN, J.J., FRIZZELL, R.A., LYRENE, R.K., BEBOK, Z., DONG, J.Y., DUVALL, M.D., FELGNER, P.L., MATALON, S., WALKER, L., and WIATRAK, B.J. (1994). Gene therapy for cystic fibrosis using cationic liposome mediated gene transfer: a phase I trial of safety and efficacy in the nasal airway. Hum. Gene Ther. 5, 1259-1277.
45. ST. GEORGE, J.A., PENNINGTON, S.E., KAPLAN, J.M., PETERSON, P.A., KLEINE, L.J., SMITH, A.E., and WADSWORTH, S.C. (1996). Biological response of nonhuman primates to long-term repeated lung exposure to Ad2/CFTR-2. Gene Ther. 3, 103-116.
46. STRIBLING, G., BRUNETTE, E., LIGGITT, D., GAENSLER, K., and DEBS, R. (1992). Aerosol gene delivery in vivo. Proc. Natl. Acad. Sci. USA 89, 11277-11281.
47. TSUKAMOTO, M., OCHIYA, T., YOSHIDA, S., SUGIMURA, T., and TERADA, M. (1995). Gene transfer and expression in progeny after intravenous DNA injection into pregnant mice. Nature Genet. 9, 243-248.
48. WOLFF, J.A., MALONE, R.W., WILLIAMS, P., CHONG, W., ACSADI, G., JANI, A., and FELGNER, P.L. (1990). Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468.
49. YANKASKAS, J.R., HAIZLIP, J.E., CONRAD, M., KOVAL, D., LAZAROWSKI, E., PARADISO, A.M., RINEHART, C.A., SARKADI, B., SCHLEGEL, R., and BOUCHER, R.C. (1993). Papilloma virus immortalized tracheal epithelial cells retain a well-differentiated phenotype. Am. J. Physiol. 264, C1219-C1230.
50. YEI, S., MITTEREDER, N., WERT, S., WHITSETT, J.A., WILMOTT, R.W., and TRAPNELL, B.C. (1994). In vivo evaluation of the safety of adenoviras-mediated transfer of the human cystic fibrosis transmembrane conductance regulator cDNA to the lung. Hum. Gene Ther. 5, 731-744.
51. YOSHIMURA, K., ROSENFELD, M.A., NAKAMURA, H., SCHERER, E.M., PAVIRANI, A., LECOCQ, J.P., and CRYSTAL, R.G. (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.
52. ZABNER, J., COUTURE, L.A., GREGORY, R.J., GRAHAM, S.M., SMITH, A.E., and WELSH, M.J. (1993). Adenoviras-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75, 207-216.
53. ZABNER, J., COUTURE, L.A., SMITH, A.E., and WELSH, M.J. (1994). Correction of cAMP-stimulated fluid secretion in cystic fibrosis airway epithelia: Efficiency of adenovirus-mediated gene transfer in vitro. Hum. Gene Ther. 5, 585-593.
54. ZHU, N., LIGGITT, D., LIU, Y., and DEBS, R. (1993). Systemic gene expression after intravenous DNA delivery into adult mice. Science 261, 209-211.
55. ZURZOLO, C., LISANTI, M.P., CARAS, I.W., NITSCH, L., and RODRIGUEZ-BOULAN, E. (1993). Glycosylphosphatidylinositol-anchored proteins are preferentially targeted to the basolateral surface in Fischer rat thyroid epithelial cells. J. Cell Biol. 121, 1031-1039.