Synthesis, activity, and structure-activity relationship studies of novel cationic lipids for DNA transfer

Journal of Medicinal Chemistry

Volumn 41, Issue 2, 1998, Pages 224-235

  Byk, Gerardo ^a   Dubertret, Catherine ^a   Escriou, Virginie ^a   Frederic, Marc ^a   Jaslin, Gabrielle ^a   Rangara, Ravi ^a   Pitard, Bruno ^a   Crouzet, Joel ^b   Wils, Pierre ^a   Schwartz, Bertrand ^a   Scherman, Daniel ^a  

^a SANOFI   (France)

^b Vector Development   (France)

Author keywords

[No Author keywords available]

Indexed keywords

POLYAMINE; QUATERNARY AMMONIUM DERIVATIVE; CATION; DNA; LIPID;

ANIMAL CELL; ARTICLE; CELL STRAIN 3T3; DNA DETERMINATION; DNA TRANSFER; DRUG SYNTHESIS; FIBROBLAST; GENE TARGETING; GENE THERAPY; GENETIC TRANSFECTION; HELA CELL; HUMAN; HUMAN CELL; LIPID ANALYSIS; MOUSE; NONHUMAN; STRUCTURE ACTIVITY RELATION; AGAR GEL ELECTROPHORESIS; ANIMAL; C57BL MOUSE; CANCER TRANSPLANTATION; CELL CULTURE; CHEMICAL MODEL; CHEMISTRY; GENETICS; LEWIS CARCINOMA; LIPID METABOLISM; METABOLISM; METHODOLOGY; SYNTHESIS;

3T3 CELLS; ANIMALS; CARCINOMA, LEWIS LUNG; CATIONS; DNA; ELECTROPHORESIS, AGAR GEL; LIPID METABOLISM; LIPIDS; MICE; MICE, INBRED C57BL; MODELS, CHEMICAL; NEOPLASM TRANSPLANTATION; POLYAMINES; QUATERNARY AMMONIUM COMPOUNDS; STRUCTURE-ACTIVITY RELATIONSHIP; TRANSFECTION; TUMOR CELLS, CULTURED;

EID: 0032518063     PISSN: 00222623     EISSN: None     Source Type: Journal    
DOI: 10.1021/jm9704964     Document Type: Article

References (31)

1. (a) Bishop, J. M. The Molecular Genetics of Cancer. Science 1987, 235, 305-311.
2. (b) Yang, Y.; Nunes, F. A.; Berencsi, K.; Furth, E. E.; Gonczol, E. and Wilson, J. M. Cellular immunity to viral antigenes limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. U.S.A. 1994, 91, 4407-4411.
3. (a) Feigner, P. L.; Rhodes, G. Gene Therapeutics. Nature 1991, 349, 351-352.
4. (b) Wheeler, C. J.; Feigner, P. L.; Tsai, Y. J.; Marshal, 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. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc. Natl. Acad. Sci. U.S.A. 1996, 93, 11454-11459.
5. Feigner, P. L.; Gadek, T. G.; Holm, M.; Roman, R.; Chan, H. W.; Wenz, M.; Northrop, J. P.; Ringold, G. M.; Danielsen, M. Lipofection: A highly Efficient, Lipid-Mediated DNA-Transfection Procedure. Proc. Natl. Acad. Sci. U.S.A. 1987, 84, 7413-7417.
6. Gao, X.; Huang, L. A Novel Cationic Liposome Reagent for Efficient Transfection of Mammalian Cells. Biochem. Biophys. Res. Commun. 1991, 179, 280-285.
7. Behr, J. P.; Demeneix, B.; Loeffler, J. P.; Perez-Mutul, J. Efficient Gene Transfer into Mammalian Primary Endocrine Cells with Lipopolyamine-Coated DNA. Proc. Natl. Acad. Sci. U.S.A. 1989, 86, 6982-6986.
8. Hawley-Nelson, P. Ciccarone, V.; Gebeyehu, G.; Jesse, J.; Felgner, P. L. Lipofectamine Reagent: A new Higher Efficiency Polycationic Liposome Transfection Reagent. Focus 1993, 15, 73-79.
9. Lee, E. R.; Marshall, J.; Siegel, C. S.; Jiang, C.; Yew, N. S.; Nichols, M. R.; Nietupsky, J. B.; Ziegler, R. J.; Lane, M. B.; Wang, K. X.; Wan, N. C.; Scheule, R. K.; Harris, D. J.; Smith, A. E.; Cheng, S. H. Detailed Analysis of Structures and Formulations of Cationic Lipids for Efficient Gene Transfer to the Lung. Hum. Gene Ther. 1996, 7, 1701-1717.
10. Feigner, J. H.; Kumar, R.; Sridhar, C. N.; Wheeler, C. J.; Tsai, Y. J.; Border, R.; Ramsey, P.; Martin, M.; Feigner, P. L. Enhanced Gene Delivery and Mechanism Studies with a Novel Series of Cationic Lipid Formulations. J. Biol. Chem. 1994, 269, 2550-2561.
11. Lasic, D. D. and Templeton, N. S. Liposomes in Gene Therapy. Adv. Drug Delivery Rev. 1996, 20, 221-266.
12. (a) Ruysschaert, J. M.; El Ouahabi, A.; Willeaume, V.; Huez, G.; Fuks, R.; Vandenbranden, M.; Di Stefano, P. A Novel Cationic Amphiphile for Transfection of Mammalian Cells. Biochem. Biophys. Res. Commun. 1994, 203, 1622-1628.
13. (b) El Ouahabi, A.; Pector, V. Fuks, R.; Vandenbranden, M. and Ruysschaert, J. M. Double Long-Chain Amidine Liposome-Mediated Self-Replicating RNA Transfection. Acta Clin. Belg. 1996, 51-3, 194.
14. (a) Lewis, J. L.; Lin, K.-Y.; Kothavale, A.; Flanagan, W. M.; Mateucci, M. D.; DePrince, R. B.; Mook, R. A., Jr.; Hendren, R. W.; Wagner, R. W. A Serum-Resistant Cytofectin for Cellular Delivery of Antisense Oligodeoxynucleotides and Plasmid DNA. Proc. Natl. Acad. Sci. U.S.A. 1996, 93, 3176-3181.
15. (b) Vigneron, J. P.; Oudrhiri, N.; Fauquet, M.; Vergely, L.; Bradley, J. C.; Bassville, M.; Lehn, P.; Lehn, J. M. Guanidinium-Cholesterol Cationic Lipids: Efficient vectors for the Transfection of Eukariotic Cells. Proc. Natl. Acad. Sci. U.S.A. 1996, 93, 9682-9686.
16. Byk, G.; Dubertret, C.; Schwartz, B.; Scherman, D. Nouveaux Agents de Transfection et Leur Applications Pharmaceutiques. French Patent Application FR9601774, 1995; International Patent Application PCT/FR96/01774, International Publication WO 9718185.
17. (a) Ganem, B. New Chemistry of Naturally Occurring Polyamines. Acc. Chem. Res 1982, 15, 290-298.
18. (b) Bergeron, R. J. Methods for the Selective Modification of Spermidine and its Homologs. Acc. Chem. Res 1986, 19, 105-113.
19. Edwards, M. L.; Stemerick D. M.; Kline S. J. Stereospecific Synthesis of Secondary Amines by The Mitsunobu Reaction. Tetrahedron Lett. 1990, 31, 3417.
20. Blagbrough, I. S.; Moya, E.; Walfort, S. P. Practical, Convergent Total Synthesis of Polyamines Amide Spider Toxin NSTX-3. Tetrahedron Lett. 1996, 37, 551-554.
21. (e) Goulaouic-Dubois, C.; Guggisberg, A.; Hesse, M. Synthesis and Selective Deprotection of the Penta-N-Protected Polyamine 1,16-Diamino-4,8,13-triazahexadecane. Tetrahedron 1995, 51, 12573-12582.
22. Merrifield, R. B. Solid Phase Peptide Synthesis I. The Synthesis of a Tetrapeptide. J. Am. Chem. Soc. 1963, 85, 2149.
23. Byk, G.; Frederic, M.; Scherman, D. One Pot Synthesis of Unsymmetrically Functionalized Polyamines by a Solid Phase Strategy Starting from Their Symmetrical Polyamine-Counter-parts. Tetrahedron Lett. 1997, 38, 3219-3222.
24. Castro, B.; Dormoy, J. R.; Dourtglou, B.; Evin, G.; Selve, C.; Ziegler, J. C. Peptide Coupling Reagents VI. A Novel, Cheaper Preparation of Benzotriazolyltris[dimethylamino] phosphonium Hexafluorophosphate (BOP Reagent). Synthesis 1976, 751-752.
25. Vitiello, L.; Chonn, A.; Wasserman, J. D.; Duff, C.; Worton, R. G. Condensation of Plasmid DNA with Polylysine Improves Liposome-Mediated Gene Transfer into Established abd Primary Muscle Cells. Gene Ther. 1996, 3, 396-404.
26. Wrobel, I.; Collins, D. Fusion of Cationic Liposomes with Mammalian Cells Occurs After Endocytosis. Biochim. Biophys. Acta 1995, 1235, 296-304.
27. Escriou, V.; Ciolina, C.; Lacroix, F.; Byk, G.; Scherman, D.; Wils, P. Catioinic Lipid-Mediated Gene Transfer: Effect of Serum on Cellular Uptake and Intracellular Fate of Lipipopolyamine/DNA Complexes. Biochim. Biophys. Acta 1997, in press.
28. Mayo, J. C. Biologic Characterization of the Subcutaneously Implanted Lewis Lung Tumor. Cancer Chemother. Rep. Part 2 1972, 3, 325-330.
29. Lavelle, F.; Bissery, M.-C.; André, S.; Roquet, F.; Riou, J.-F. Preclinical evaluation of CPT-11 and its active metabolite SN-38. Sem. Oncol. 1996, 23, 11-20.
30. Pitard, B.; Aguerre, O.; Ariau, M.; Lachages, A. M.; Bouknikachvilli, T.; Byk, G.; Dubertret, C.; Daniel, J. C.; Herviou, C.; Scherman, D.; Mayaux, J. F.; Crouzet, J. Submitted 1997. Virus-sized self assembling lamellar complexes between DNA and cationic micelles promote gene transfer. Proc. Natl. Acad. Sci. U.S.A. 1997, in press.
31. Makowski, M.; Rzeszotarska, B.; Smelka, L.; Kubica, Z. Debenzyloxycarbonylation and Detrifluoroacetylation of Dehydroalanine and Dehydrophenylalanine Peptides. Liebigs Ann. Chem. 1985, 1457-1458.