Gene transfer efficacies of novel cationic amphiphiles with alanine, β-alanine, and serine headgroups: A structure-activity investigation

Bioconjugate Chemistry

Volumn 16, Issue 4, 2005, Pages 903-912

  Sen, Joyeeta ^a   Chaudhuri, Arabinda ^a  

^a INDIAN INSTITUTE OF CHEMICAL TECHNOLOGY   (India)

Author keywords

[No Author keywords available]

Indexed keywords

AMPHIPHILES; CELL CULTURE; EFFICIENCY; GENE TRANSFER; GENES; ISOMERS; LIPIDS; LIPOSOMES;

AMINO-ACIDS; CATIONIC AMPHIPHILE; CATIONIC LIPIDS; HEAD GROUPS; HYDROPHOBIC TAILS; IN-VITRO; LIPOFECTAMINE; MCF-7 CELLS; STRUCTURE ACTIVITY; Β-ALANINE;

AMINO ACIDS;

ALANINE; AMPHOPHILE; BETA ALANINE; CATION; CHOLESTEROL; DNA; LIPID; LIPOFECTAMINE; LIPOSOME; MYRISTIC ACID DERIVATIVE; PALMITIC ACID DERIVATIVE; SERINE;

AMINO ACID SUBSTITUTION; ARTICLE; CELL STRAIN COS1; CELL STRAIN HEPG2; CELL STRAIN MCF 7; CHO CELL; DRUG CYTOTOXICITY; GENE DELIVERY SYSTEM; GENE TRANSFER; GENE VECTOR; GENETIC TRANSFECTION; HISTOCHEMISTRY; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; ISOMER; REPORTER GENE; STRUCTURE ACTIVITY RELATION;

MINK CELL FOCUS-FORMING VIRUS;

EID: 22644434652     PISSN: 10431802     EISSN: None     Source Type: Journal    
DOI: 10.1021/bc0500443     Document Type: Article

References (54)

1. Verma, I. M., and Somina, M. (1997) Gene Therapy-Promises, Problems and Prospects. Nature 389, 239-242.
2. Anderson, W. F. (1998) Human Gene Therapy. Nature 392, 25-30.
3. Yla-Herttuala, S., and Martin, J. F. (2000) Cardiovascular Gene Therapy. Lancet 355, 213-222.
4. Yang, Y., Nunes, F. A., Berencsi, K., Furth, E. E., Gonczol, E., and Wilson, J. M. (1994) Cellular Immunity to Viral Antigens Limits E1-Deleted Adenoviruses for Gene Therapy. Proc. Natl. Acad. Sci. U.S.A. 91, 4407-4411.
5. Knowles, M. R., Hohneker, K. W., Zhou, Z., Olsen, J. C., Noah, T. L., Hu, P. C., Leigh, M. W., Engelhardt, J. F., Edwards, L. J., Jones, K. R., and Boucher, R. A. (1995) Controlled Study of Adenoviral-Vector mediated Gene Transfer in the Nasal Epithelium of Patients with Cystic Fibrosis. New Engl. J. Med. 333, 823-831.
6. Crystal, R. G., McElvaney, N. G., Rosenfeld, M. A., Chu, C. S., Mastrangeli, A., Hay, J. G., Brody, S. L., Jaffe, H. A., Eissa, N. T., and Danel, C. (1994) Administration of an Adenovirus containing the Human CFTR cDNA to the Respiratory Tract of Individuals with Cystic Fibrosis. Nat. Genet. 8, 42-51.
7. Yang, Y., Nunes, F. A., Berencsi, K., Gonczol, E., Engelhardt, J. F., and Wilson, J. (1994) Inactivation of E2a in Recombinant Adenoviruses Improves the Prospect for Gene Therapy in Cystic Fibrosis. Nat. Genet. 7, 362-369.
8. Lehrman, S. (1999) Virus Treatment Questioned after Gene Therapy Death. Nature 401, 517-518.
9. McGregor, C., Perrin, C., Monck, M., Camilleri, P., Anthony, and Kirby, J. (2001) Rational Approaches to the Design of Cationic Gemini Surfactants for Gene Delivery. J. Am. Chem. Soc. 123, 6215-6220.
10. Prata, C. A. H., Zhao, Y., Barthelemy, P., Li, Y., Luo, D., McIntosh, T. J., Lee, S. J, and Grinstaff, M. W. (2004) Charge-Reversal Amphiphiles for Gene Delivery. J. Am. Chem. Soc. 126, 12196-12197.
11. Bell, P. C., Bergsma, I. P., Bras, W., Stuart, M. C. A., Rowan, M. C., Feiters, M. C., and Engberts, J. B. F. N. (2003) Transfection mediated by Gemini Surfactants: Engineered Escape from Endosomal Compartment. J. Am. Chem. Soc. 125, 1551-1558.
12. Majeti, B. K., Singh, R. S., Yadav, S. K., Reddy, S. B., Ramkrishna, S., Diwan, P. V., Madhavendra, S. S., and Chaudhuri, A. (2004) Enhanced Intravenous Transgene Expression in Mouse Lung using Cyclic-head Cationic Lipids. Chem. Biol. 11, 427-437.
13. 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 and the references therein.
14. Zhu, J., Munn, R. J., and Nantz, M. H. (2000) Self-cleaving Ortho Ester Lipids: A Class of pH-Vulnerable Ampliphiles. J. Am. Chem. Soc. 122, 2645-2646.
15. Guenin, E., Herve, A. C., Floch, V., Loisel, S., Yaounac, J. J., Clement, J. C., Ferec, C., and des Abbayas, H. (2000) Cationic Phosphonolipids containing Quaternary Phosphonium and Arsonium Groups for DNA Transfection with Good Efficiency and Low Cellular Toxicity. Angew. Chem., Int. Ed. 39, 629-631.
16. Mahidhar, Y. V., Rajesh, M., and Chaudhuri, A. (2004) Strikingly Spacer-Arm Modulated Gene Delivery Efficacies of Novel Cationic Glycolipids: Design, Synthesis and In Vitro Transfection Biology. J. Med. Chem. 47, 3938-3948.
17. Karmali, P. P., Kumar, V. V., and Chaudhuri, A. (2004) Design, Syntheses and InVitro Gene Delivery efficacies of Novel Mono-, Di-, and Tri-lysinated Cationic Lipids: A Structure Activity Investigation. J. Med. Chem. 47, 2123-2132.
18. Miller, A. D. (1998) Cationic Liposomes in Gene Therapy. Angew. Chem., Int. Ed. 37, 1768-1785 and the references therein.
19. Lynn, D. M., Anderson, D. G., Putman, D., and Langer, R. (2001) Accelerated Discovery of Synthetic Transfection Vectors: Parallel Synthesis and Screening of a Degradable Polymer Library. J. Am. Chem. Soc. 123, 8155-8156.
20. Choi, J. S., Joo, D. K., Kim, C. H., Kim, K., and Park, J. S. (2000) Synthesis of a Barbell-like Triblock Copolymer, Poly-(L-lysine) Dendrimer-block-Poly(ethylene glycol)-block-Poly-(L-lysine) Dendrimer, and Its Self-Assembly with Plasmid DNA. J. Am. Chem. Soc. 122, 474-480.
21. Tang, M. X., Redemann, C. T., and Szoka, F. C. (1996) In Vitro Gene Delivery by Degraded Polyamidoamine Dendrimers. Bioconjugate Chem. 7, 703-714.
22. Kukowska-Latallo, J. F., Bielinska, A. U., Johnson, J., Spinder, R., Tomalia, D. A., and Baker, J. R. (1996) Efficient transfer of genetic material into mammalian cells using Starburst polyamidoamine dendrimers. Proc. Natl. Acad. Sci. U.S.A. 93, 4897-4902.
23. Luo, D., Haverstick, K., Belcheva, N., Han, E., and Saltzman, W. M. (2002) Poly(ethylene glycol)-Conjugated PAMAM Dendrimer for Biocompatible, High-Efficiency DNA Delivery. Macromolecules 35, 3456-3462.
24. Ilies, M. A., Seitz, W. A., Ghiviriga, I., Johnson, B. H., Miller, A., Thompson, E. B., and Balaban, A. T. (2004) Pyridinium Cationic Lipids in Gene Delivery: A Structure-Activity Correlation Study. J. Med. Chem. 15, 3744-3754.
25. Lee, Y., Koo, H., Lim, Y. B., Lee, Y., Mo, H., and Park, J. S. (2004) New Cationic Lipids for Gene Transfer with High Efficiency and Low Toxicity: T-shape Cholesterol Ester Derivatives. Bioorg. Med. Chem. Lett. 14, 2637-2641.
26. Wang, L., and MacDonald, R. C. (2004) New Strategy for Transfection: Mixtures of Medium-Chain and Long Chain Cationic Lipids Synergistically Enhance Transfection. Gene Ther. 11, 1358-1362.
27. 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.
28. Kirby, A. J., Carmilleri, P., Engberts, J. B. F. N., Feiters, M. C., Nolte, R. J. M., Soderman, O., Bergsma, M., Bell, P. C., Fielden, M. L., Garcia Rodriguez, C. L., Guedat, P., Kremer, A., McGregor, C., Perrin, C., Ronsin, G., and Van Eijik, M. C. P. (2003) Gemini Surfactants: New Synthetic Vectors for Gene Transfection. Angew. Chem., Int. Ed. 42, 1448-1457.
29. Leclercq, F., Cohen-Ohana, M., Mignet, N., Sbarbati, A., Herscovici, J., Scherman, D., and Byk, G. (2003) Design, Synthesis and Evaluation of Gadolinium Cationic Lipids as Tools for Biodistribution Studies of Gene Delivery Complexes. Bioconjugate Chem. 14, 112-119.
30. Fabio, K., Gaucheron, J., Giorgio, C. D., and Vierling, P. (2003) Novel Galactosylated Polyamine Bolaamphiphiles for Gene Delivery. Bioconjugate Chem. 14, 358-367.
31. Heyes, J. A., Duvaz, D. N., 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.
32. Ewert, K., Ahmed, A., Evans, H. M., Schmidt, H. W., and Safinya, C. R. (2002) Efficient Synthesis and Cell-transfection Properties of a New Multivalent Cationic Lipid for Non-Viral Gene Delivery. J. Med. Chem. 45, 5023-5029.
33. Floch, V., Loisel, S., Guenin, E., Herve, A. C., Clement, J. C., Yaouanc, J. J., des Abbayes, H., and Ferec, C. (2000) Cation Substitution in Cationic Phosphono lipids: A New Concept to Improve Transfection Activity and Decrease Cellular Toxicity. J. Med. Chem. 43, 4617-4628.
34. Fiebert, T., Regelin, A., and Massing, U. (2000) Synthesis and Transfection Properties of Novel Nontoxic Monocationic Lipids: Variations of Lipid Anchor, Spacer and Headgroup Structure. Bioorg. Med. Chem. Lett. 10, 787-791.
35. Kawakami, S., Sato, A., Nishikawa, M., Yamashita, F., and Hashida, M. (2000) Mannose Receptor Mediated Gene Tranfer into Macrophases using Novel Mannosylated Cationic Liposomes. Gene. Ther. 7, 292-299.
36. Choi, J. S., Lee, E. J., Jang, H. S., and Sang, J. P. (2001) New Cationic Liposomes for Gene Transfer into Mammalian Cells with High Efficiency and Low Toxicity. Bioconjugate Chem. 12, 108-113.
37. Byk, G., Wetzer, B., Frederic, M., Dubertret, C., Pitard, B., Jaslin, G., and Scherman, D. (2000) Reduction-Sensitive Lipopolyamines as a Novel Nonviral Gene Delivery System For Modulated Release of DNA with Improved Transgene Expression. J. Med. Chem. 43, 4377-4387.
38. 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.
39. Wang, J., Guo, X., Xu, Y., Barron, L., and Szoka, F. C., Jr. (1998) Synthesis and Characterization of Long Chain Alkyl Acyl Carnitine Esters. Potentially Biodegradable Cationic Lipids for Use in Gene Delivery. J. Med. Chem. 41, 2207-2215.
40. Ghosh, Y. K., Visweswariah, S. S., and Bhattacharya, S. (2000) Nature of linkage between the cationic headgroup and cholesterol skeleton controls gene transfection efficiency. FEBS. Lett. 473, 341-344.
41. 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. 289, 2550-2561.
42. Zabner, J., Fasbender, Al, J., Moninger, T., Poellinger, K. A., and Welsh, M. J. (1995) Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270, 18997-19007.
43. Floch, V., Bolćh, G. L., Gable-Guillaume, C., Bris, N. L., Yaouanc, J-J., Abbayes, H., Ferec, C., and Clement, J.-C. (1998) Phosphonolipids as Non-Viral Vectors for Gene Therapy. Eur. J. Med. Chem. 33, 923-934.
44. Gao, H., and Hui, K. M. (2001) Synthesis of Novel series of Cationic Lipids that can act as Efficient Gene Delivery Vehicles through Systematic Heterocyclic Substitution of Cholesterol Derivatives. Gene Ther. 8, 855-863.
45. Ghosh, Y. K., Visweswariah, S. S., and Bhattacharya, S. (2002) Advantage of the Ether Linkage between the Positive Charge and the Cholesteryl Skeleton in Cholesterol- based Amphiphilkes as vectors for Gene Delivery. Bioconjugate Chem. 13, 378-384.
46. 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.
47. Singh, R. S., and Chaudhuri, A. (2004) Single Additional Methylene Group in the Headgroup Region Imparts High Gene Transfer Efficacy to a Transfection-Incompetent Cationic Lipid. FEBS Lett. 556, 86-90.
48. Kumar, V. V., Pichon, C., Refregiers, M., Guerin, B., Midoux, P., and Chaudhuri, A. (2003) Single Histidine Residue in Headgroup Region is Sufficient to Impart Remarkable Gene Transfection Properties to Cationic Lipids: Evidence for Histidine-mediated Membrane Fusion at Acidic pH. Gene. Ther. 10, 1206-1215.
49. Singh, R. S., Mukherjee, K., Banerjee, R., Chaudhuri, A., Hait, S. K., Moulik, S., Ramadas, Y., Vijayalakshmi, A., and Rao, N. M. (2002) Anchor-dependency for Nonglycerol based Cationic Lipofectins: Mixed Bag of Regular and Anomalous Transfection Profiles. Chem. Eur. J. 8, 900-909.
50. Srilakshmi, G. V., Sen, J., Chaudhuri, A., Ramdas, Y., and Rao, N. M. (2002) Anchor-dependent Lipofection with Nonglycerol based Cytofectins containing Single 2-Hydroxyethyl Head groups. Biochim. Biophys. Acta 1559, 87-95.
51. 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.
52. Banerjee, R., Das, P. K., Srilakshmi, G. V., Chaudhuri, A., and Rao, N. M. (1999) A Novel Series of Nonglycerol based Cationic Transfection Lipids for Use in Liposomal Gene Delivery. J. Med. Chem. 42, 4292-4299.
53. Bodanszky, M., and Bodanszky, A. (1984) The Practice of Peptide Synthesis, p 20, Springer-Verlag, Berlin, Heidelberg.
54. Sambrook, J., Fritsch, E. F., and Maniatis, T. (1989) Molecular Cloning: A Laboratory Manual, 2nd ed.; Cold Springer Harbor Laboratory Press, Cold Spring Harbor, NY.