Cationic lipids with increased DNA binding affinity for nonviral gene transfer in dividing and nondividing cells

Bioconjugate Chemistry

Volumn 16, Issue 1, 2005, Pages 156-168

  Narang, Ajit S ^a   Thoma, Laura ^a   Miller, Duane D ^a   Mahato, Ram I ^a  

^a UNIVERSITY OF TENNESSEE HEALTH SCIENCE CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BINDING ENERGY; DICHROISM; DNA; ELECTROPHORETIC MOBILITY; GENE TRANSFER; HIGH RESOLUTION TRANSMISSION ELECTRON MICROSCOPY; HYDROGEN BONDS; LIPIDS; PARTICLE SIZE; PROTEINS;

CATIONIC LIPIDS; DNA BINDING AFFINITY; HEAD GROUPS; MESYLATES; NON-DIVIDING CELLS; NONVIRAL GENE TRANSFER; PHOSPHATIDYLETHANOLAMINE; QUATERNARY AMINES; TERTIARY AMINE; TRANSFECTION EFFICIENCY;

LIPOSOMES;

AMINE; CHOLESTEROL; DIOLEOYLPHOSPHATIDYLETHANOLAMINE; GREEN FLUORESCENT PROTEIN; HYDROXYL GROUP; LIPID; LIPOFECTAMINE; LIPOSOME; LUCIFERASE; MESYLIC ACID; PLASMID DNA; TERTIARY AMINE;

ANIMAL CELL; AORTA MEDIA; ARTICLE; BINDING AFFINITY; CELL DIVISION; CIRCULAR DICHROISM; COVALENT BOND; DNA BINDING; DNA CONFORMATION; GEL MOBILITY SHIFT ASSAY; GENE TRANSFER; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; HYDROGEN BOND; LASER DIFFRACTION; MOUSE; NONHUMAN; NONVIRAL GENE THERAPY; PANCREAS ISLET CELL; PARTICLE SIZE; POLYMERIZATION; PROTEIN EXPRESSION; RAT; SURFACE PROPERTY; TRANSMISSION ELECTRON MICROSCOPY; ZETA POTENTIAL;

ANIMALIA;

EID: 12344253784     PISSN: 10431802     EISSN: None     Source Type: Journal    
DOI: 10.1021/bc049818q     Document Type: Article

References (59)

1. Mahato, R. I., Rolland, A., and Tomlinson, E. (1997) Cationic lipid-based gene delivery systems: pharmaceutical perspectives. Pharm. Res. 14, 853-859.
2. 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.
3. 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.
4. Felgner, P. L., Gadek, T. R., Holm, M., Roman, R., Chan, H. W., Wenz, M., Northrop, J. P., Ringold, G. M., and Danielsen, M. (1987) Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc. Natl. Acad. Sci. U.S.A. 84, 7413-7417.
5. Gao, X., and Huang, L. (1991) A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179, 280-285.
6. 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. U.S.A. 86, 6982-6986.
7. Edelstein, M. L., Abedi, M. R., Wixon, J., and Edelstein, R. M. (2004) Gene therapy clinical trials worldwide 1989-2004-an overview. J. Gene. Med. 6, 597-602.
8. Hui, K. M., Ang, P. T., Huang, L., and Tay, S. K. (1997) Phase I study of immunotherapy of cutaneous metastases of human carcinoma using allogeneic and xenogeneic MHC DNA-liposome complexes. Gene Ther. 4, 783-790.
9. Galanis, E., Hersh, E. M., Stopeck, A. T., Gonzalez, R., Burch, P., Spier, C., Akporiaye, E. T., Rinehart, J. J., Edmonson, J., Sobol, R. E., Forscher, C., Sondak, V. K., Lewis, B. D., Unger, E. C., O'driscoll, M., Selk, L., and Rubin, J. (1999) Immunotherapy of advanced malignancy by direct gene transfer of an interleukin-2 DNA/DMRIE/DOPE lipid complex: phase I/II experience. J. Clin. Oncol. 17, 3313-3323.
10. Ruiz, F. E., Clancy, J. P., Perricone, M. A., Bebok, Z., Hong, J. S., Cheng, S. H., Meeker, D. P., Young, K. R., Schoumacher, R. A., Weatherly, M. R., Wing, L., Morris, J. E., Sindel, L., Rosenberg, M., Van Ginkel, F. W., Mcghee, J. R., Kelly, D., Lyrene, R. K., and Sorscher, E. J. (2001) A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis. Hum. Gene Ther. 12, 751-761.
11. Mahato, R. I., Anwer, K., Tagliaferri, F., Meaney, C., Leonard, P., Wadhwa, M. S., Logan, M., French, M., and Rolland, A. (1998) Biodistribution and gene expression of lipid/plasmid complexes after systemic administration. Hum. Gene Ther. 9, 2083-2099.
12. Nomura, T., Nakajima, S., Kawabata, K., Yamashita, F., Takakura, Y., and Hashida, M. (1997) Intratumoral pharmacokinetics and in vivo gene expression of naked plasmid DNA and its cationic liposome complexes after direct gene transfer. Cancer Res. 57, 2681-2686.
13. Nomura, T., Yasuda, K., Yamada, T., Okamoto, S., Mahato, R. I., Watanabe, Y., Takakura, Y., and Hashida, M. (1999) Gene expression and antitumor effects following direct interferon (IFN)-gamma gene transfer with naked plasmid DNA and DC-chol liposome complexes in mice. Gene Ther. 6, 121-129.
14. Wang, D. A., Narang, A. S., Kotb, M., Gaber, A. O., Miller, D. D., Kim, S. W., and Mahato, R. I. (2002) Novel branched poly(ethylenimine)-cholesterol water-soluble lipopolymers for gene delivery. Biomacromolecules 3, 1197-1207.
15. Han, S., Mahato, R. I., Sung, Y. K., and Kim, S. W. (2000) Development of biomaterials for gene therapy. Mol. Ther. 2, 302-317.
16. Mahato, R. I., Henry, J., Narang, A. S., Sabek, O., Fraga, D., Kotb, M., and Gaber, A. O. (2003) Cationic lipid and polymer-based gene delivery to human pancreatic islets. Mol. Ther. 7, 89-100.
17. Narang, A. S., Cheng, K., Henry, J., Zhang, C., Sabek, O., Fraga, D., Kotb, M., Gaber, A. O., and Mahato, R. I. (2004) Vascular endothelial growth factor gene delivery for revascularization in transplanted human islets. Pharm. Res. 21, 15-25.
18. Gaber, A. O., Fraga, D. W., Callicutt, C. S., Gerling, I. C., Sabek, O. M., and Kotb, M. Y. (2001) Improved in vivo pancreatic islet function after prolonged in vitro islet culture. Transplantation 72, 1730-1736.
19. Ferrari, M. E., Rusalov, D., Enas, J., and Wheeler, C. J. (2002) Synergy between cationic lipid and co-lipid determines the macroscopic structure and transfection activity of lipoplexes. Nucleic Acids Res. 30, 1808-1816.
20. Matulis, D., Rouzina, I., and Bloomfield, V. A. (2002) Thermodynamics of cationic lipid binding to DNA and DNA condensation: roles of electrostatics and hydrophobicity. J. Am. Chem. Soc. 124, 7331-7342.
21. Floch, V., Le Bolc'h, G., Audrezet, M. P., Yaouanc, J. J., Clement, J. C., Des Abbayes, H., Mercier, B., Abgrall, J. F., and Ferec, C. (1997) Cationic phosphonolipids as non viral vectors for DNA transfection in hematopoietic cell lines and CD34+ cells. Blood Cells Mol. Dis. 23, 69-87.
22. Mahato, R. I., Kawabata, K., Nomura, T., Takakura, Y., and Hashida, M. (1995) Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes. J. Pharm. Sci. 84, 1267-1271.
23. Mahato, R. I., Lee, M., Han, S., Maheshwari, A., and Kim, S. W. (2001) Intratumoral delivery of p2CMVmIL-12 using water-soluble lipopolymers. Mol. Ther. 4, 130-138.
24. Benhamou, P. Y., Moriscot, C., Prevost, P., Rolland, E., Halimi, S., and Chroboczek, J. (1997) Standardization of procedure for efficient ex vivo gene transfer into porcine pancreatic islets with cationic liposomes. Transplantation 63, 1798-1803.
25. Saldeen, J., Sandler, S., Bendtzen, K., and Welsh, N. (2000) Liposome-mediated transfer of IL-1 receptor antagonist gene to dispersed islet cells does not prevent recurrence of disease in syngeneically transplanted NOD mice. Cytokine 12, 405-408.
26. Saldeen, J., Curiel, D. T., Eizirik, D. L., Andersson, A., Strandell, E., Buschard, K., and Welsh, N. (1996) Efficient gene transfer to dispersed human pancreatic islet cells in vitro using adenovirus-polylysine/DNA complexes or polycationic liposomes. Diabetes 45, 1197-1203.
27. 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. Nat. Biotechnol. 15, 167-173.
28. Byk, G., Dubertret, C., Escriou, V., Frederic, M., Jaslin, G., Rangara, R., Pitard, B., Crouzet, J., Wils, P., Schwartz, B., and Scherman, D. (1998) Synthesis, activity, and structure-activity relationship studies of novel cationic lipids for DNA transfer. J. Med. Chem. 41, 229-235.
29. Banerjee, R., Mahidhar, Y. V., Chaudhuri, A., Gopal, V., and Rao, N. M. (2001) Design, synthesis, and transfection biology of novel cationic glycolipids for use in liposomal gene delivery. J. Med. Chem. 44, 4176-4185.
30. Regelin, A. E., Fankhaenel, S., Gurtesch, L., Prinz, C., Von Kiedrowski, G., and Massing, U. (2000) Biophysical and lipofection studies of DOTAP analogues. Biochim. Biophys. Acta 1464, 151-164.
31. Venkata Srilakshmi, G., Sen, J., Chaudhuri, A., Ramadas, Y., and Madhusudhana Rao, N. (2002) Anchor-dependent lipofection with nonglycerol based cytofectins containing single 2-hydroxyethyl headgroups. Biochim. Biophys. Acta 1559, 87-95.
32. Heyes, J. A., Niculescu-Duvaz, D., Cooper, R. G., and Springer, C. J. (2002) Synthesis of novel cationic lipids: effect of structural modification on the efficiency of gene transfer. J. Med. Chem. 45, 99-114.
33. Vigneron, J. P., Oudrhiri, N., Fauquet, M., Vergely, L., Bradley, J. C., Basseville, M., Lehn, P., and Lehn, J. M. (1996) Guanidinium-cholesterol cationic lipids: efficient vectors for the transfection of eukaryotic cells. Proc. Natl. Acad. Sci. U.S.A. 93, 9682-9686.
34. Ren, T., Song, Y. K., Zhang, G., and Liu, D. (2000) Structural basis of DOTMA for its high intravenous transfection activity in mouse. Gene Ther. 7, 764-768.
35. Gaucheron, J., Wong, T., Wong, K. F., Maurer, N., and Cullis, P. R. (2002) Synthesis and properties of novel tetraalkyl cationic lipids. Bioconjugate Chem. 13, 671-675.
36. Miller, A. D. (1998) Cationic Liposomes for Gene Therapy. Angew Chem., Int. Ed. 37, 1768-1765.
37. Niculescu-Duvaz, D., Heyes, J., and Springer, C. J. (2003) Structure-activity relationship in cationic lipid mediated gene transfection. Curr. Med. Chem. 10, 1233-1261.
38. Liu, D., Ren, T., and Gao, X. (2003) Cationic Transfection Lipids. Curr. Med. Chem. 10, 1005-1013.
39. Nicolazzi, C., Garinot, M., Mignet, N., Scherman, D., and Bessodes, M. (2003) Cationic lipids for transfection. Curr. Med. Chem. 10, 1263-1277.
40. Kumar, V. V., Singh, R. S., and Chaudhuri, A. (2003) Cationic transfection lipids in gene therapy: successes, setbacks, challenges and promises. Curr. Med. Chem. 10, 1297-1306.
41. Miller, A. D. (2003) The problem with cationic liposome/micelle-based nonviral vector systems for gene therapy. Curr. Med. Chem. 10, 1195-1211.
42. Behr, J. P. (1994) Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconjugate Chem. 5, 382-389.
43. Bartsch, H., Terracini, B., Malaveille, C., Tomatis, L., Wahrendorf, J., Brun, G., and Dodet, B. (1983) Quantitative comparison of carcinogenicity, mutagenicity and electrophilicity of 10 direct-acting alkylating agents and of the initial O6: 7-alkylguanine ratio in DNA with carcinogenic potency in rodents. Mutat. Res. 110, 181-219.
44. Eder, E., Kutt, W., and Deininger, C. (2001) On the role of alkylating mechanisms, O-alkylation and DNA-repair in genotoxicity and mutagenicity of alkylating methanesulfonates of widely varying structures in bacterial systems. Chem. Biol. Interact. 137, 89-99.
45. Peterson, A. R. (1980) DNA synthesis, mutagenesis, DNA damage, and cytotoxicity in cultured mammalian cells treated with alkylating agents. Cancer Res. 40, 682-688.
46. Platzek, T., and Bochert, G. (2000) DNA alkylation studies of combined treatment with methylnitrosourea and ethyl-methanesulfonate in mice. Teratog., Carcinog., Mutagen 20, 27-34.
47. Snyder, R. D., and Bhatt, S. (1993) Alterations in repair of alkylating agent-induced DNA damage in polyamine-depleted human cells. Cancer Lett 72, 83-90.
48. Hudson, R. F., and Withey, R. J. (1964) Nucleophilic reactivity. V. The reaction between amines and ethyl methanesulfonate. J. Chem. Soc. 3513-3517.
49. Ross, P. C., and Hui, S. W. (1999) Lipoplex size is a major determinant of in vitro lipofection efficiency. Gene Ther. 6, 651-659.
50. Zelphati, O., Nguyen, C., Ferrari, M., Felgner, J., Tsai, Y., and Felgner, P. L. (1998) Stable and monodisperse lipoplex formulations for gene delivery. Gene Ther. 5, 1272-1282.
51. Gray, D. M., Johnson, K. H., Vaughan, M. R., Morris, P. A., Sutherland, J. C., and Ratliff, R. L. (1990) The vacuum UV CD bands of repeating DNA sequences are dependent on sequence and conformation. Biopolymers 29, 317-323.
52. Allen, F. S., Gray, D. M., and Ratliff, R. L. (1984) On the first-neighbor analysis of nucleic acid CD spectra: the definitive T matrix and considerations of various methods. Biopolymers 23, 2639-2659.
53. Gustafsson, J., Arvidson, G., Karlsson, G., and Almgren, M. (1995) Complexes between cationic liposomes and DNA visualized by cryo-TEM. Biochim. Biophys. Acta 1235, 305-312.
54. Pitard, B., Aguerre, O., Airiau, M., Lachages, A. M., Boukhnikachvili, T., Byk, G., Dubertret, C., Herviou, C., Scherman, D., Mayaux, J. F., and Crouzet, J. (1997) Virus-sized self-assembling lamellar complexes between plasmid DNA and cationic micelles promote gene transfer. Proc. Natl. Acad. Sci. U.S.A. 94, 14412-14417.
55. Rackstraw, B. J., Stolnik, S., Davis, S. S., Bignotti, F., and Garnett, M. C. (2002) Development of multicomponent DNA delivery systems based upon poly(amidoamine)-PEG copolymers. Biochim. Biophys. Acta 1576, 269-286.
56. Thomas, M., and Klibanov, A. M. (2003) Conjugation to gold nanoparticles enhances polyethylenimine's transfer of plasmid DNA into mammalian cells. Proc. Natl. Acad. Sci. U.S.A. 100, 9138-9143.
57. Bennett, M. J., Aberle, A. M., Balasubramaniam, R. P., Malone, J. G., Malone, R. W., and Nantz, M. H. (1997) Cationic lipid-mediated gene delivery to murine lung: correlation of lipid hydration with in vivo transfection activity. J. Med. Chem. 40, 4069-4078.
58. Bloomfield, V. A. (1996) DNA condensation. Curr. Opin. Struct. Biol. 6, 334-341.
59. Plank, C., Mechtler, K., Szoka, F. C., Jr., and Wagner, E. (1996) Activation of the complement system by synthetic DNA complexes: a potential barrier for intravenous gene delivery. Hum. Gene Ther. 7, 1437-1446.