Cationic lipid-DNA complexes for gene therapy: Understanding the relationship between complex structure and gene delivery pathways at the molecular level

Current Medicinal Chemistry

Volumn 11, Issue 2, 2004, Pages 133-149

  Ewert, Kai ^a   Slack, Nelle L ^a   Ahmad, Ayesha ^a   Evans, Heather M ^a   Lin, Alison J ^a   Samuel, Charles E ^b   Safinya, Cyrus R ^a  

^a University of California   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Cationic liposomes; Gene delivery; Gene therapy; Lipofection; Multivalent; Nonviral; Small angle x ray scattering

Indexed keywords

1,2 DIOLEOYL 3 TRIMETHYLAMMONIOPROPANE; 2,3 DI(MYRISTYLOXY)PROPYL(2 HYDROXYETHYL)DIMETHYLAMMONIUM BROMIDE; 2,3 DIOLEYLOXY N [2 (SPERMINECARBOXAMIDO)ETHYL] N,N DIMETHYL 1 PROPYLAMMONIUM CHLORIDE; AMMONIUM DERIVATIVE; CANCER VACCINE; CATION; CHLOROQUINE; DIOLEOYLPHOSPHATIDYLCHOLINE; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; DNA; DNA VACCINE; LIPID; LIPOSOME; LUCIFERASE; MACROGOL; ORNITHINE DERIVATIVE; PEPTIDE; PLASMID DNA; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL CELL; CELL INTERACTION; CHEMICAL STRUCTURE; CLINICAL TRIAL; COMPLEX FORMATION; CONFOCAL LASER MICROSCOPY; EX VIVO STUDY; GENE EXPRESSION; GENE VECTOR; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HUMAN; IN VIVO STUDY; INTERMETHOD COMPARISON; LIPID MEMBRANE; MOLECULAR GENETICS; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PROCESS OPTIMIZATION; REPORTER GENE; REVIEW; STRUCTURE ANALYSIS; SYNCHROTRON; X RAY DIFFRACTION;

ANIMALS; CATIONS; DNA; GENE THERAPY; HUMANS; LIPIDS; TRANSFECTION;

EID: 1642503835     PISSN: 09298673     EISSN: None     Source Type: Journal    
DOI: 10.2174/0929867043456160     Document Type: Review

References (54)

1. Henry, C.M. Chem. Eng. News, 2001, 79(48), 35.
2. Ferber, D. Science, 2001, 294, 1638.
3. Alper, J. Science, 2002, 296, 838.
4. Chesnoy, S.; Huang, L. Annu. Rev. Biophys. Biomol. Struct., 2000, 29, 27.
5. Friedmann, T. Sci. Am., 1997, 276, 96.
6. Clark, P.R.; Hersh, E.M. Curr. Opin. Mol. Ther., 1999, 1, 158.
7. Miller, A. D. Angew. Chem., Int. Ed., 1998, 37, 1768.
8. Felgner, P.L.; Gadek, T.R.; Holm, M., Roman, R.; Chan, H.W.; Wenz, M.; Northrop, J.P.; Ringold, G.M.; Danielsen, M. Proc. Natl. Acad. Sci. USA, 1987, 84, 7413.
9. Wolff, J. A. Ed. Gene Therapeutics: Methods and Applications of Direct Gene Transfer, Birkhäuser, Boston, 1994.
10. Nabel, G.; Nabel, E.; Yang, Z.; Fox, B.; Plautz, G.; Gao, X.; Huang, L.; Shu, S., Gordon, D.; Chang, A. Proc. Natl. Acad. Sci. USA, 1993, 90, 11307.
11. Extensive and current information on clinical trials in the field of gene therapy can be found on the internet at http://www.wiley.co.uk/genetherapy/clinical/.
12. Marshall, E. Science, 2000, 288, 951.
13. Marshall, E. Science, 2002, 298, 510.
14. Radler, J.O.; Koltover, L; Salditt, T.; Safinya, C.R. Science, 1997, 275, 810.
15. Koltover, I.; Salditt, T.; Rädler, J.O.; Safinya, C.R. Science, 1998, 281, 78.
16. Lasic, D.D.; Strey, H.; Stuart, M.C.A.; Podgornik, R.; Frederik, P.M. J. Am. Chem. Soc., 1997, 119, 832.
17. Harrington, J.J; van Bokkelen, G.; Mays, R.W.; Gustashaw, K.; Willard, H.F. Nat. Genet., 1997, 15, 345.
18. Willard, H.F. Science, 2000, 290, 1308.
19. Koltover, I.; Salditt, T.; Safinya, C.R. Biophys. J., 1999, 77, 915.
20. Salditt, T.; Koltover, I.; Rädler, J.O.; Safinya, C.R. Phys. Rev. Lett., 1997, 79, 2582.
21. Koltover, I.; Wagner, K.; Safinya, C.R. Proc. Natl. Acad. Sci. USA, 2000, 97, 14046.
22. Safinya, C.R. Curr. Opin. Struc. Biol., 2001, 11, 440.
23. Schulze, U.; Schmidt, H.-W.; Safinya, C.R. Bioconjugate Chem., 1999, 10, 548.
24. Ewert, K., Ahmad, A.; Evans, H.M.; Schmidt, H.-W.; Safinya, C.R. J. Med. Chem., 2002, 45, 5023.
25. Rinehart, J.; Hersh, E.; Issell, B.; Triozzi, P.; Buhles, W.; Neidhart, J. Cancer Invest., 1997, 15, 403.
26. Stopeck, A.T.; Hersh, E.M.; Brailey, J.L.; Clark, P.R.; Norman, J.; Parker, S.E. Cancer Gene Ther., 1998, 5, 119.
27. Ewert, K.; Safinya, C.R. manuscript in preparation.
28. Farhood, H.; Serbina, N.; Huang, L. Biochim. Biophys. Acta, 1995, 1235, 289.
29. Hui, S.; Langner, M.; Zhao, Y.; Ross, P.; Hurley, E.; Chan, K. Biophys. J., 1996, 71, 590.
30. Lin, A.J.; Slack, N.L.; Ahmad, A.; Koltover, I.; George, C.X.; Samuel, C.E.; Safinya, C.R. J. Drug Targeting, 2000, 8, 13.
31. Wrobel, I.; Collins, D. Biochim. Biophys. Acta, 1995, 1235, 296.
32. Zabner, J.; Fasbender, A.J.; Moninger, T.; Poellinger, K.A.; Welsh, M.A. J. Biol. Chem., 1995, 270, 18997.
33. Xu, Y.H.; Szoka, F.C. Biochemistry, 1996, 35, 5616.
34. Tristram-Nagle, S.; Petrache, H.I.; Nagle, J.F. Biophys. J., 1998, 75, 917.
35. Felgner, P.L. Adv. Drug Deliv. Rev., 1990, 5, 163.
36. Voet, D.; Voet, J. Biochemistry, Wiley, New York, 1995.
37. Bloomfield, V.A. Biopolymers, 1991, 31, 1471.
38. Lin, A.J.; Slack, N.L.; Ahmad, A., George, C.X.; Samuel, C.E.; Safinya, C.R. Biophys. J., 2003, 84, 1.
39. Mislick, K.A.; Baldeschwieler, J.D. Proc. Natl. Acad. Sci. USA, 1996, 93, 12349.
40. Helfrich, W. Z. Naturforsch., 1973, 28c, 693.
41. Seddon, J. M. Biochim. Biophys. Acta, 1990, 1031, 1.
42. Janiak, M.J.; Small, D.M.; Shipley, G.G. J. Biol. Chem., 1979, 254, 6068.
43. Gompper, G.; Goos, J. J. Phys. II (France), 1995, 5, 621.
44. Wong, G.C.L.; Tang, J.X.; Lin, A.; Li, Y.; Janmey, P.A.; Safinya, C.R. Science, 2000, 288, 2035.
45. Remy, J.-S.; Sirlin, C.; Vierling, P.; Behr, J.-P. Bioconjugate Chem., 1994, 5, 647.
46. Kalderon, D.; Roberts, B.L.; Richardson, W.D.; Smith, A.E. Cell, 1984, 39, 499.
47. Dworetzky, S.I.; Lanford, R.E.; Feldherr, C.M. J. Cell Biol., 1988, 107, 1279.
48. Feldherr C. M.; Akin, D. J. Cell Biol., 1990, 111, 1.
49. Smyth Templeton, N.; Lasic, D.D.; Frederik, P.M.; Strey, H.H.; Roberts, D.D.; Pavlakis, G.N. Nat. Biotechnol., 1997, 15, 647.
50. Plank, C.; Mechtler, K.; Szoka, F.C.; Wagner, E. Human Gene Ther. 1996, 7, 1437.
51. Bradley, A.J.; Devine, D.V.; Ansell, S.M.; Janzen, J.; Brooks, D.E. Arch. Biochem. Biophys., 1998, 357, 185.
52. Lasic, D.D.; Martin, F.J. Eds. Stealth liposomes, CRC Press, Boca Raton, 1995.
53. Allen T. M. Trends Pharmacol. Sci., 1994, 15, 215.
54. Martin-Herranz, A.; Ahmad, A.; Evans, H.M.; Ewert, K.; Schulze, U.; Schmidt, H.-W.; Safinya, C.R. Biophys. J., accepted.