Cationic lipid structure and formulation considerations for optimal gene transfection of the lung

Journal of Drug Targeting

Volumn 7, Issue 6, 2000, Pages 453-469

  Marshall, John ^a   Nietupski, Jennifer B ^a   Lee, Edward R ^a   Siegel, Craig S ^a   Rafter, Patrick W ^a   Rudginsky, Samantha A ^a   Chang, Chau D ^a   Eastman, Simon J ^a   Harris, David J ^a   Scheule, Ronald K ^a   Cheng, Seng H ^a  

^a GENZYME CORPORATION   (United States)

Author keywords

Cationic lipids; Formulation; Gene transfer; Neutral co lipids; Plasmid DNA; Pulmonary transfection

Indexed keywords

CATION; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; LIPID; PHOSPHATIDYLETHANOLAMINE; PLASMID DNA; PROTON;

ACYLATION; ANIMAL EXPERIMENT; ARTICLE; CHEMICAL STRUCTURE; CONTROLLED STUDY; DNA BINDING; DRUG FORMULATION; FEMALE; GENETIC TRANSFECTION; LUNG; MOUSE; NONHUMAN; PRIORITY JOURNAL;

EID: 0034076070     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.3109/10611860009102219     Document Type: Article

References (45)

1. Alton, E.W.F.W., Stern, F., Farley, R., Jaffe, A., Chadwick, S.L., Phillips, J., Davies, J., Smith, S.N., Browning, J., Hodson, M.E., Durham, S., Li, D., Jeffery, P.K., Scallan, M., Balfour, R., Eastman, S.J., Cheng, S.H., Smith, A.E., Meeker, D. and Geddes, D.M. (1999) Cationic lipid-mediatcd CFTR gene transfer to the lungs and nose of patients with cystic fibrosis: a double-blind placebo-controlled trial. Lancet 353, 947 954.
2. Behr, J.P., Demeneix, B., Loeffler, J.P. and Perez-Mutul, J. (1989) Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc. Natl. Acad. Sci. USA 86, 6982 6986.
3. Bischoff, R., Cordier, Y., Perraud, F., Thioudellett, C., Braun, S. and Pavirani, A. (1997) Transfection of myoblasts in primary culture with isomeric cationic cholesterol derivatives. Anal. Biochem. 254, 69 81.
4. Boussif, R., Zanta, M.A. and Behr, J.P. (1996) Optimized galenics improve in vitro gene transfer with cationic molecules up to 1000 fold. Gene Ther. 3, 1074-1080.
5. Byk, G., Dubertret, C., Escriou, V., Frederic, M., Jaslin, G., Rangara, R., Pitard, B., Crouzet, J., Wils, O., Schwartz, B. and Scherman, D. (1998) Synthesis, activity, and structure-activity relationship studies of novel cationic lipids for DNA transfer. J. Med. Chem. 41, 224 235.
6. 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., Porteus, 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.
7. Chang, C.-D. and Harris, D.J. (1998) Reversed-phase high performance liquid chromatography analysis of cationic lipid-based gene transfer agents. J. Liq. Chrom. Rel. Technol. 21, 1119 1136.
8. Cheng, S.H. and Scheule, R.K. (1998) Airway delivery of cationic lipid: DNA complexes for cystic fibrosis. Adv. Drug Del. Rev. 30, 173 184.
9. Cheng, S.H., Marshall, J., Scheule, R.K. and Smith, A.E. (1998) Cationic lipid formulations for intracellular gene delivery of cystic fibrosis transmembrane conductance regulator to airway epithelia. Meth. Enzymol. 292, 697 717.
10. Colin, M., Harbottle, R.P., Knight, A., Kornprobst, M., Cooper, R.G., Miller, A.D., Trugnan, G., Capeau, J., Coutelle, C. and Brahimi-Horn, M.C. (1998) Liposomes enhance delivery and expression of an RGD-oligolysine gene transfer vector in human tracheal cells. Gene Ther. 5, 1488-1498.
11. 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.
12. Fasbender, A., Marshall, J., Moniger, T.O., Grunst, T., Cheng, S.H. 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.
13. Felgner, P.L., Gadek, T.R., Holm, M., Roman, R., Chan, C., 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.
14. 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.
15. Gao, X. and Huang, L. (1991) A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179, 280-285.
16. 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.G., 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.
17. 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, 31 391-31 396.
18. 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 polarized and differentiated airway epithelial cells in vitro and in vivo. Hum. Gene Ther. 9, 1531-1542.
19. Kichler, A., Zauner, W., Ogris, M. and Wagner, E. (1998) Influence of the DNA complexation medium on the transfection of lipospermine/DNA particles. Gene Ther. 5, 855-860.
20. Lee, E.R., Marshall, J., Siegel. C.S., Jiang, C., Yew, N.S., Nichols, M.R., Nietupski, J.B., Ziegler, R.J., Lane, M., 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.
21. Liu, Y., Mounkes, L.C., Liggitt, H.D., Brown, C.S., Solodin, I., Heath, T.D. and Debs, R.J. (1997) Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nature Biotech. 15, 167-173.
22. Marshall, J., Yew, N.S., Eastman, S.J., Jiang, C., Scheule, R.K. and Cheng, S.H. (1999) Cationic lipid-mediated gene delivery to the airways. In Huang, L., Hung, M.C. and Wagner, E. (Eds.), Non-viral Vectors for Gene Therapy (Academic Press), pp. 39-68.
23. Matsui, H., Johnson, L.G., Randell, S.H. and Boucher, R.C. (1997) Loss of binding and entry of liposome DNA complexes decreases transfection efficiency in differentiated airway epithelial cells. J. Biol. Chem. 272, 1117-1126.
24. 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.
25. Moradpour, D., Schauer, J.I., Zurawski Jr., V.R., Wands, J.R. and Boutin, R.H. (1996) Efficient gene transfer into mammalian cells with cholesteryl-spermidine. Biochem. Biophys. Res. Comm. 221, 82-88.
26. Nabel, G.J., Gordon, D., Bishop, D.K., Nickoloff, B.J., Yang, Z.Y., Aruga, A., Cameron, M.J., Nabel, E.G. and Chang, A.E. (1996) Immune response in human melanoma after transfer of an allogenic class I major histocompatibility complex gene with DNA-liposome complexes. Proc. Natl. Acad. Sci. USA 93, 15 388-15 393.
27. Oudrhiri, N., Virgeron, 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. USA 94, 1652-1656.
28. Porteus, D.J., Dorin, J.R., McLachlan, G., Davidson-Smith, 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 the 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.
29. Remy, J.S., Sirlin, C., Vierling, P. and Behr, J.P. (1994) Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. 5, 647 654.
30. Scheule, R.K. and Cheng, S.H. (1996) Gene transfer into mammalian cells using synthetic cationic lipids. In Felgner, P.L., Heller, M.J., Lehn, P., Behr, J.P., and Szoka, F.C. (Eds.), Artificial Self-assembling Systems for Gene Delivery (American Chemical Society), pp. 177-190.
31. 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. Hum. Gene Ther. 8, 689-707.
32. Song, Y.K., Liu, F., Chu, S.Y. and Liu, D. (1997) Characterization of cationic liposome-mediated gene transfer in vivo by intravenous administration. Hum. Gene Ther 8, 1585-1594.
33. 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.
34. Stopeck, A.T., Hersh, E.M., Akporiaye, E.T., Harris, D.T., Grogan, T., Unger, E., Warneke, J., Schluter, S.F. and Stahl, S. (1997) Phase I study of direct gene transfer of an allogenic histocompatibility antigen, HLA-B7, in patients with metastatic melanoma. J. Clin. Oncol. 15, 341-349.
35. Templeton, N.S., Lasic, D.D., Frederick, P.M., Strey, H.H., Roberts, D.D. and Pavlakis, G.N. (1997) Improved DNA: liposome complexes for increased systemic delivery and gene expression. Nature Biotech. 15, 647-652.
36. Wang, J.K., Guo, X., Xu, Y.H., Barron, L. and Szoka, F.C. (1998) Synthesis and characterization of long chain alkylacylcarnithine esters. Potentially biodegradable cationic lipids for use in gene delivery. J. Med. Chem. 41, 2207-2215.
37. 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. (1996a) A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc. Natl. Acad. Sci. USA 93, 11 454-11 459.
38. Wheeler, C.J., Sukhu, L., Yang, G., Tsai, Y.J., Bustamente, C., Felgner, P.L., Norman, J. and Manthorpe, M. (1996b) Converting an alcohol to an amine in a cationic lipid dramatically alters the co-lipid requirement, cellular transfection activity and the ultrastructure of DNA-cytofectin complexes. Biochim. Biophys. Acta 1280, 1-11.
39. Xing, X., Zhang, S., Chang, J., Tucker, S.D., Chen, H., Huang, L. and Hung, M.C. (1998) Safety study and characterization of Ela-liposome complex gene-delivery protocol in an ovarian cancer model. Gene Ther. 5, 1538-1544
40. Yew, N.S., Wysokenski, D.M., Wang, K.X., Ziegler, R.J., Marshall, J., McNeilly, D., Cherry, M., Osburn, W. and Cheng, S.H. (1997) Optimization of plasmid vectors for high-level expression in lung epithelial cells. Hum. Gene Ther. 8, 575-584.
41. Zabner, J. (1997) Cationic lipids used in gene transfer. Adv. Drug Del. Rev. 27, 17-28.
42. Zabner, J., Cheng, S.H., Meeker, D., Launspach, J., Balfour, R., Perricone, M.A., Morris, J.E., Marshall, J., Fasbender, 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.
43. Zabner, J., Fasbender, A.J., Moninger, T., Poellinger, K.A. and Welsh, M.J. (1995) Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270, 18 997 19 007.
44. Zanta, M.A., Belguise-Valladier, P. and Behr, J.P. (1999) Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl. Acad. Sci. USA 96, 91 96.
45. Zhu, N., Liggit, D., Liu, Y. and Debs, R. (1993) Systemic gene expression after intravenous DNA delivery into adult mice. Science 261, 209-211.