Cationic lipid-based gene delivery systems: Pharmaceutical perspectives

Pharmaceutical Research

Volumn 14, Issue 7, 1997, Pages 853-859

  Mahato, Ram I ^a   Rolland, Alain ^a   Tomlinson, Eric ^a  

^a Gene Medicine Inc   (United States)

Author keywords

Cationic liposomes; Formulation; Non viral gene delivery; Plasmid; Transfection

Indexed keywords

CATION; GENE PRODUCT; LIPOSOME;

GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; PRIORITY JOURNAL; REVIEW;

ANIMALS; CATIONS; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LIVER; LUNG;

EID: 0030852003     PISSN: 07248741     EISSN: None     Source Type: Journal    
DOI: 10.1023/A:1012187414126     Document Type: Review

References (54)

1. F. D. Ledley. Pharmaceutical approach to somatic gene therapy. Pharm. Res. 13:1595-1614 (1996).
2. E. Tomlinson and A. Rolland. Controllable gene therapy: Pharmaceutics of non-viral gene delivery systems. J. Control. Rel. 39:357-372 (1996).
3. A. P. Rolland. From genes to gene medicines. Crit. Rev. Ther. Drug Carrier Syst. (in press).
4. T. S. Ledley and F. D. Ledley. A multicompartment, numerical model of cellular events in the pharmacokinetics of gene therapies. Hum. Gene Ther. 5:679-91 (1994).
5. P. L. Feigner, T. R. Gadek, M. Holm, R. Roman, H. W. Chan, M. Wenz, J. P. Northrop, G. M. Ringold, and M. Danielsen. Lipofection: a highly efficient lipid-mediated DNA transfection procedure. Proc. Natl. Acad. Sci. USA 84:7413-7417 (1987).
6. D. D. Lasic and N. S. Templeton. Liposomes in gene therapy. Adv. Drug Del. Rev. 20:221-266 (1996).
7. J. P. Behr, B. Demeinex, J. P. Loeffler, and J. Perez-Mutul. Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc. Natl. Acad. Sci. USA 86:6982-6986 (1989).
8. R. I. Mahato, Y. Takakura, and M. Hashida. In non-viral vectors for in vivo gene therapy: Physicochemical and pharmacokinetic considerations. Crit. Rev. Ther. Drug Carrier Syst. 14:133-172 (1997).
9. G. J. Nabel, E. G. Nabel, Z.-Y. Yang, B. A. Fox, G. E. Plautz, X. Gao, L. Huang, S. Shu, D. Gordon, and A. E. Chang. 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 (1993).
10. N. J. Caplen, E. W. F. W. Alton, P. G. Middleton, J. R. Dorin, B. J. Stevenson, X. Gao, S. R. Durham, P. K. Jeffery, M. E. Hodson, C. Coutelle, L. Huang, D. J. Porteous, R. Williamson, and D.M. Geddes. Liposome-mediated CFTR gene transfer to the nasal epithelium of patients with cystic fibrosis. Nature Med. 1:39-46 (1995).
11. M. Krieger. Gene transfer. In: Gene Transfer and Expression: A Laboratory Manual, W.H. Freeman and Company, New York, NY. p 3-81 (1990).
12. H. Farhood, N. Serbina, and L. Huang. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta. 1235:289-295 (1995).
13. S. W. Hui, M. Langer, Y-L. Zhao, P. Ross, E. Hurley and K. Chan. The role of helper lipids in cationic liposome-mediated gene transfer. Biophys. J. 71:590-599 (1996).
14. M. J. Bennett, M. H. Nantz, R. P. Balasubramanian, D. C. Gruenert, and R. W. Malone. Cholesterol enhances cationic liposome-mediated DNA transfection of human respiratory epithelial cells. Bioscience Reports 15:47-53 (1995).
15. R. Leventis and J. R. Silvius. Interactions of mammalian cells with dispersions containing novel metabolizable cationic amphiphiles. Biochim. Biophys. Acta 1023:124-132 (1990).
16. J. K. Rose, L. Buonocore, and M. A. Whitt. A new cationic liposome reagent mediating nearly quantitative transfection of animal cells. Biotechniques 10:520-525 (1991).
17. G. McLachlan, H. Davidson, D. Davison, P. Dickinson, J. Dorin, and D. Porteous. DOTAP as a vehicle for efficient gene delivery in vitro and in vivo. Biochemistry 11:19-21 (1994).
18. P. Hawley-Nelson, V. Ciccarone, G. Gebeyehu, J. Jessee, and P. L. Felgner. Lipofectamine reagent: A new, higher efficiency polycationic liposome transfection reagent. Focus 15:73-79 (1993).
19. J. H. Felgner, R. Kumar, C. N. Shridhar, C. J. Wheeler, Y. J. Tsai, R. Border, P. Ramsey, M. Martin, and P. L. Felgner. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269:2550-2561 (1994).
20. X. A. Gao and L. Huang. A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179:280-285 (1991).
21. I. Solodin, C. S. Brown, M. S. Bruno, C.-Y. Chow, E.-H. Jang, R. J. Debs, and T. D. Heath. A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery. Biochemistry 34:13537-13544 (1995).
22. C. J. Wheeler, P. L. Felgner, Y. J. Tsai, J. Marshall, L. Shukhu, S. G. Doh, J. Hartikka, J. Nietupski, M. Manthorpe, M. Nichols, M. Plewe, X. Liang, J. Norman, A. Smith, and S. H. Cheng. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc. Natl. Acad. Sci. USA 93:11454-11459 (1996).
23. E. R. Lee, J. Marshall, C. S. Siegel, C. Jiang, N. S. Yew, M. R. Nichols, J. B. Nietupski, R. J. Ziegler, M. B. Lane, K. X. Wang, N. C. Wan, R. K. Scheule, D. J. Harris, A. E. Smith, and S. H. Cheng. Detailed analysis of structures and formulations of cationic lipids for efficient gene transfer to the lung. Hum. Gene Ther. 7:1701-1717 (1996).
24. P. L. Felgner, Y. J. Tasai, L. Sukhu, C. J. Wheeler, and M. Manthorpe. Improved cationic lipid formulations for in vivo gene therapy. Ann. N.Y. Acad Sci. 772:126-139 (1995).
25. M. J. Bennett, A. M. Aberle, R. P. Balasubramanian, J. G. Malone, M. H. Nantz, and R. W. Malone. Considerations for the design of improved cationic amphiphile based transfection reagents. J. Liposome Res. 6:545-565 (1996).
26. M. S. Bruno, A. Singhal, R. J. Mumper, M. K. Barron, F. Nicol, F. C. Szoka, Jr., and A. P. Rolland. Cryoprotection of cationic lipid-DNA complexes for gene delivery. Pharm. Res. 12:5-79 (1995).
27. H. Gershon, R. Ghirlando, S. B. Guttman, and A. Minsky. Mode of formation and structures of DNA-cationic liposome complexes used for transfection. Biochemistry 32:7143-7151 (1993).
28. B. Sternberg, F. L. Sorgi, and L. Huang. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy. FEBS Lett. 356:361-366 (1996).
29. J. S. Remy, C. Sirlin, P. Vierling, and J. P. Behr. Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. 5:647-654 (1994).
30. C. Plank, K. Mechtler, F. C. Szoka, Jr., and E. Wagner. Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum. Gene Ther. 7:1437-1446 (1996).
31. E. Tomlinson. Theory and practice of site-specific drug delivery. Adv. Drug Del. Rev. 1:87-198 (1987).
32. J.-S. Remy, A. Kichler, V. Mordvinov, F. Schuber, and J.-P. Behr. 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 (1995).
33. P.-W. Cheng. Receptor ligand-facilitated gene transfer: Enhancement of liposome-mediated gene transfer and expression by transferrin. Hum. Gene Ther. 7:275-282 (1996).
34. R. I. Mahato, K. Kawabata, Y. Takakura, and M. Hashida. In vivo disposition characteristics of plasmid DNA complexed with cationic liposomes. J. Drug Target. 3:149-157 (1995).
35. D. Lew, S. E. Parker, T. Latimer, A. M. Kuwahara-Rundell, S. G. Doh, Z.-Y. Yang, D. Laface, S. H. Gramkowski, G. J. Nabel, M. Manthorpe, and J. Norman. Cancer gene therapy using plasmid DNA: Pharmacokinetic study of DNA following injection in mice. Hum. Gene Ther. 6:553-564 (1995).
36. R. I. Mahato, K. Kawabata, T. Nomura, Y. Takakura, and M. Hashida. Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes. J. Pharm. Sci. 84:1267-1271 (1995).
37. Y. Liu, L. C. Mounkes, H. D. Liggitt, C. S. Brown, I. Solodin, T. D. Heath, and R. J. Debs. Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nature Biotechnol. 15:167-173 (1997).
38. K. L. Brigham, B. Meyrick, B. Christman, M. Magnuson, G. King, and L. C. Berry. Rapid communication: In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298:278-281 (1989).
39. J. J. Logan, Z. Bebok, L. C. Walker, S. Peng, P. L. Feigner, G. P. Siegel, R. A. Frizzell, J. Dong, M. Howard, S. Matalon, J. R. Lindsey, M. DuVall, and E. J. Sorscher. Cationic lipids for reporter gene and CFTR transfer to rat pulmonary epithelium. Gene Ther. 2:38-49 (1995).
40. A. E. Canonico, J. T. Conary, B. O. Meyrick, and K. L. Brigham. Aerosol and intravenous transfection of human alpha-1 antitrypsin gene to lungs of rabbits. Am. J. Respir. Cell Mol. Biol. 10:24-29 (1994).
41. K. L. Brigham, B. Meyrick, B. Christman, J. T. Conary, G. King, L. C. Berry, Jr., and M. A. Magnuson. Expression of human growth hormone fusion genes in cultured lung endothelial cells and in the lungs of mice. Am. J. Respir. Cell. Mol. Biol. 8:209-213 (1993).
42. K. B. Meyer, M. M. Thompson, M. Y. Levy, L. G. Barron, and F. C. Szoka, Jr. Intratracheal gene delivery to the mouse airway: characterization of plasmid DNA expression and pharmacokinetics. Gene Ther. 2:450-460 (1995).
43. R. Stribling, E. Brunetter, D. Liggitt, K. Gannsler, and R. Debs. Aerosol gene delivery in vivo. Proc. Natl. Acad. Sci. USA 89:11277-11281 (1992).
44. F. Labat-Moleur, A.-M. Steffan, C. Brisson, H. Perron, O. Feugeas, P. Fursteberger, F. Oberling, E. Brambilla, and J.-P. Behr. An electron microscopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther. 3:1010-1017 (1996).
45. R. K. Scheule and S. H. Cheng. Gene transfer into mammalian cells using synthetic cationic lipids. In: Artificial Self-Assembling Systems for Gene Delivery (P. L. Felgner, M. J. Heller, P. Lehr, J. P. Behr, and F. C. Szoka, Jr., eds.). ACS Books, Washington, p. 177-190 (1996).
46. S. Li and L. Huang. Lipidic supramolecular assemblies for gene transfer. J. Liposome Res. 6:589-608 (1996).
47. X. Zhou and L. Huang. DNA transfection mediated by cationic liposomes containing lipopolylysines: characterization and mechanism of action. Biochim. Biophys. Acta. 1189:195-203 (1994).
48. D. S. Friend, D. Papahadjopoulos and RJ. Debs. Endocytosis and intracellular processing accompanying transfection mediated by cationic liposomes. Biochim. Biophys. Acta. 1278:41-50 (1996).
49. Y. Xu and F. C. Szoka, Jr. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 35:5616-5623 (1996).
50. M. R. Capecchi. High efficiency transformation by direct microinjection of DNA into cultured mammalian cells. Cells 22:479-(1980).
51. J. Zabner, A. J. Fasbender, T. Monsinger, K. A. Poellinger, and M. J. Welsh. Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270:18997-19007 (1995).
52. X. Gao and L. Huang. Cytoplasmic expression of a reporter gene by co-delivery of T7 RNA polymerase and T7 promoter sequence with cationic liposomes. Nucleic Acids Res. 21:2867-2872 (1993).
53. C. Gauther-Rouviere, M. Vandromme, N. Lautredou, Q. Q. Cai, F. Girard, A. Fernandez, and N. Lamb. The serum response factor nuclear localization signal: General implications for cyclic AMP-dependent protein kinase activity in control of nuclear translocation. Mol. Cell Biol. 15:433-444 (1995).
54. M. Miller, M. K. Park, and J. A. Hanover. Nuclear pore complex: Structure, function, and regulation. Physiol. Rev. 71:909-949 (1991).