Synthesis of cholesteryl polyamine carbamates: pK(a) studies and condensation of calf thymus DNA

Bioconjugate Chemistry

Volumn 11, Issue 3, 2000, Pages 314-326

  Geall, Andrew J ^a   Taylor, Richard J ^b   Earll, Mark E ^b   Eaton, Michael A W ^b   Blagbrough, Ian S ^a  

^a University of Bath   (United Kingdom)

^b UCB PHARMA   (Belgium)

Author keywords

[No Author keywords available]

Indexed keywords

BINDING KINETICS; CHOLESTEROL; CHOLESTERYL POLYAMINE CARBAMATE; DNA; GENE THERAPY; GENE TRANSFER; LIPOFECTION; PARTICLE; POTENTIOMETRY; SYNTHESIS; THYMUS;

AMINES; BINDING ENERGY; CONDENSATION; FLUORESCENCE QUENCHING; GENE THERAPY; GENE TRANSFER; LIGHT SCATTERING; LIPOSOMES; THYMUS;

AMINO FUNCTIONAL GROUP; BINDING AFFINITIES; CALF THYMUS; ETHIDIUM BROMIDE; METHYLENE CHAINS; POLYAMINES; POSITIVE CHARGES; PROTECTING GROUP; PROTECTION STRATEGY; SPECTROSCOPIC DATA;

DNA;

MAMMALIA;

EID: 0034030753     PISSN: 10431802     EISSN: None     Source Type: Journal    
DOI: 10.1021/bc990115w     Document Type: Article

References (54)

1. Ganem, B. (1982) New chemistry of naturally occurring polyamines. Acc. Chem. Res. 15, 290-298.
2. Bergeron, R. J. (1986) Methods for selective modification of spermidine and its homologues. Acc. Chem. Res. 19, 105-113.
3. Bloomfield, V. A. (1996) DNA condensation. Curr. Opin. Struct. Biol. 6, 334-341.
4. Blagbrough, I. S., Carrington, S., and Geall, A. J. (1997) Polyamines and polyamine amides as potent selective receptor probes, novel therapeutic lead compounds, and synthetic vectors in gene therapy. Pharm. Sci. 3, 223-233.
5. Behr, J.-P. (1986) DNA strongly binds to micelles and vesicles containing lipopolyamines or lipointercalants. Tetrahedron Lett. 27, 5861-5864.
6. Behr, J.-P. (1993) Synthetic gene-transfer vectors. Acc. Chem. Res. 26, 274-278.
7. Behr, J.-P. (1994) Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconjugate Chem. 5, 382-389.
8. Felgner, P. L., Barenholz, Y., Behr, J.-P., Cheng, S. H., Cullis, P., Huang, L., Jessee, J. A., Seymour, L., Szoka, F., Thierry, A. R., Wagner, E., and Wu, G. (1997) Nomenclature for synthetic gene delivery systems. Hum. Gene Ther. 8, 511-512.
9. Crystal, R. G. (1995) Transfer of genes to humans. Early lessons and obstacles to success. Science 270, 404-410.
10. Feigner, P. L. (1997) Nonviral strategies for gene therapy. Sci. Am. 276, 86-90.
11. O'Driscoll, C. (1997) Gene geniuses. Chem. Br. 33, 66-69.
12. Mahato, R. I., Rolland, A., and Tomlinson, E. (1997) Cationic lipid-based gene delivery systems: pharmaceutical perspectives. Pharm. Res. 14, 853-859.
13. Verma, I. M., and Somia, N. (1997) Gene therapy -promises, problems and prospects. Nature 389, 239-242.
14. 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.
15. 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.
16. Moradpour, D., Schauer, J. I., Zurawski, V. R., Jr., Wands, J. R., and Boutin, R. H. (1996) Efficient gene transfer into mammalian cells with cholesteryl-spermidine. Biochem. Biophys. Res. Commun. 221, 82-88.
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, 31391-31396.
18. Walker, S., Sofia, M. J., Kakarla, R., Kogan, N. A., Wierichs, L., Longley, C. B., Bruker, K., Axelrod, H. R., Midha, S., Babu, S., and Kahne, D. (1996) Cationic facial amphiphiles: a promising class of transfection agents. Proc. Natl. Acad. Sci. U.S.A. 93, 1585-1590.
19. Hsieh, H.-P., Muller, J. G., and Burrows, C. J. (1994) Structural effects in novel steroidal polyamine-DNA binding. J. Am. Chem. Soc. 116, 12077-12078.
20. Geall, A. J., and Blagbrough, I. S. (1998) Homologation of polyamines in the synthesis of lipo-spermine conjugates and related lipoplexes. Tetrahedron Lett. 39, 443-446.
21. Blagbrough, I. S., and Geall, A. J. (1998) Practical synthesis of unsymmetrical polyamine amides. Tetrahedron Lett. 39, 439-442.
22. a studies and condensation of calf thymus DNA. Chem. Commun. 1403-1404 and 1607.
23. Geall, A. J., Al-Hadithi, D., and Blagbrough, I. S. (1998) Spermine and thermine conjugates of cholic acid condense DNA, but lithocholic acid polyamine conjugates do so more efficiently. Chem. Commun. 2035-2036.
24. Blagbrough, I. S., Al-Hadithi, D., and Geall, A. J. (1999) DNA condensation by bile acid conjugates of thermine and spermine. Pharm. Pharmacol. Commun. 5, 139-144.
25. Geall, A. J., and Blagbrough, I. S. (1999) DNA condensation by cholesterol polyamine carbamates. Pharm. Pharmacol. Commun. 5, 145-150.
26. Bischoff, R., Cordier, Y., Perraud, F., Thioudellet, C., Braun, S., and Pavirani, A. (1997) Transfection of myoblasts in primary culture with isomeric cationic cholesterol derivatives. Anal. Biochem. 254, 69-81.
27. Cooper, R. G., Etheridge, C. J., Stewart, L., Marshall, J., Rudginsky, S., Cheng, S. H., and Miller, A. D. (1998) Polyamine analogues of 3β-[N-(N′,N′-dimethylaminoethane)-carbamoyl]cholesterol (DC-Chol) as agents for gene delivery. Chem. Eur. J. 4, 137-153.
28. Cain, B. F., Baguley, B. C., and Denny, W. A. (1978) Potential antitumor agents. 28. Deoxyribonucleic acid polyintercalating agents. J. Med. Chem. 21, 658-668.
29. 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, 224-235.
30. a values and total proton distribution pattern of spermidine by carbon-13 nuclear magnetic resonance titrations. Anal. Chem. 53, 789-793.
31. Aikens, D. A., Bunce, S. C., Onasch, O. F., Schwartz, H. M., and Hurwitz, C. (1983) Two-dimensional N. M. R. investigation of the protonation sequence in spermidine. J. Chem. Soc., Chem. Commun. 43-45.
32. Tables of Spectral Data for Structure Determination of Organic Compounds (1989) 2nd ed., pp C5-C177 and H15-H80, Springer-Verlag, Berlin.
33. Frassineti, C., Ghelli, S., Gans, P., Sabatini, A., Moruzzi, M. S., and Vacca, A. (1995) Nuclear magnetic resonance as a tool for determining protonation constants of natural polyprotic bases in solution. Anal. Biochem. 231, 374-382.
34. Basu, H. S., Schwietert, H. C. A., Feuerstein, B. G., and Marton, L. J. (1990) Effects of variation in the structure of spermine on the association with DNA and the induction of DNA conformational changes. Biochem. J. 269, 329-334.
35. Pouton, C. W., Lucas, P., Thomas, B. J., Uduehi, A. N., Milroy, D. A., and Moss, S. H. (1998) Polycation-DNA complexes for gene delivery: a comparison of the biopharmaceutical properties of cationic polypeptides and cationic lipids. J. Controlled Release 53, 289-299.
36. Tikchonenko, T. I., Glushakova, S. E., Kislina, O. S., Grodnitskaya, N. A., Manykin, A. A., and Naroditsky, B. S. (1988) Transfer of condensed viral DNA into eukaryotic cells using proteoliposomes. Gene 63, 321-330.
37. Basu, H. S., and Marton, L. J. (1987) The interaction of spermine and pentamines with DNA. Biochem. J. 144, 243-246.
38. Wilson, R. W., and Bloomfield, V. A. (1979) Counterion-induced condensation of deoxyribonucleic acid. A light-scattering study. Biochemistry 18, 2192-2196.
39. 6. J. Mol. Biol. 144, 431-453.
40. Böttcher, C., Endisch, C., Fuhrhop, J.-H., Catterall, C., and Eaton, M. (1998) High-yield preparation of oligomeric C-type DNA toroids and their characterization by cryoelectron microscopy. J. Am. Chem. Soc. 120, 12-17.
41. Anderegg, G., and Bläuenstein, P. (1982) Polyamines XIV. Protonation equilibria of linear tetramines containing two ethylenediamines residues. Helv. Chim. Acta 65, 162-169.
42. 13C nuclear magnetic resonance spectroscopy. Eur. J. Biochem. 130, 383-389.
43. Aikens, D., Bunce, S., Onasch, F., Parker, R., Hurwitz, C., and Clemans, S. (1983) The interaction between nucleic acids and polyamines. Biophys. Chem. 17, 67-74.
44. Bergeron, R. J., McManis, J. S., Weimar, W. R., Schreier, K. M., Gao, F., Wu, Q., Ortiz-Ocasio, J., Luchetta, G. R., Porter, C., and Vinson J. R. T. (1995) The role of charge in polyamine analogue recognition. J. Med. Chem. 38, 2278-2285.
45. Gao, X., and Huang, L. (1991) A novel cationic liposome reagent for efficient transfection of mammalian cells. Biochem. Biophys. Res. Commun. 179, 280-285.
46. 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.
47. Pitard, B., Oudrhiri, N., Vigneron, J.-P., Huachecorne, M., Aguerre, O., Toury, R., Airiau, M., Ramasawmy, R., Scherman, D., Crouzet, J., Lehn, J.-M., and Lehn, P. (1999) Structural characteristics of supramolecular assemblies formed by guanidinium-cholesterol for gene transfection. Proc. Natl. Acad. Sci U.S.A. 96, 2621-2626.
48. Hsieh, H.-P., Muller, J. G., and Burrows, C. J. (1995) Synthesis and DNA binding properties of C3-, C12-, and C24-substituted amino-steroids derived from bile acids. Bioorg. Med. Chem. 3, 823-835.
49. 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.
50. Ren, T., and Liu, D. (1999) Synthesis of targetable cationic amphiphiles. Tetrahedron Lett. 40, 7621-7625.
51. Manning, G. S. (1978) The molecular theory of polyelectrolyte solutions with applications to the electrostatic properties of polynucleotides. Q. Rev. Biophys. 11, 179-246.
52. Labat-Moleur, F., Steffan, A.-M., Brisson, C., Perron, H., Feugeas, O., Furstenberger, P., Oberling, F., Brambilla., and Behr, J.-P. (1996) An electron microcopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther. 3, 1010-1017.
53. Hebling-Leclerc, A., Scherman, D., and Wils, P. (1999) Cellular uptake of cationic lipid/DNA complexes by cultured myoblasts and myotubes. Biochim. Biophys. Acta 1418, 165-175.
54. Geall, A. J., Eaton, M. A. W., Baker, T., Catterall, C., and Blagbrough, I. S. (1999) The regiochemical distribution of positive charges along cholesterol polyamine carbamates plays significant roles in modulating DNA binding affinity and lipofection. FEBS Lett. 459, 337-342.