Synthesis and gene transfer activities of novel serum compatible reducible tocopherol-based cationic lipids

Molecular Pharmaceutics

Volumn 9, Issue 5, 2012, Pages 1146-1162

  Kedika, Bhavani ^a   Patri, Srilakshmi V ^a  

^a NATIONAL INSTITUTE OF TECHNOLOGY   (India)

Author keywords

cationic liposome; DNA release; gene delivery; lipofection; lipoplexes; reducible tocopherol based cationic amphiphiles

Indexed keywords

CYSTINE; LIPID; N,N DI TERT BUTYLOXYCARBONYL LEVO CYSTINE MONOTOCOPHEROL ESTER; TOCOPHEROL; UNCLASSIFIED DRUG;

ARTICLE; CANCER CELL CULTURE; CONTROLLED STUDY; GENE DELIVERY SYSTEM; GENE THERAPY; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; PRIORITY JOURNAL; STRUCTURE ACTIVITY RELATION; SYNTHESIS;

ANIMALS; CELL LINE, TUMOR; CHO CELLS; CRICETINAE; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEP G2 CELLS; HUMANS; LIPIDS; LIPOSOMES; MICROSCOPY, FLUORESCENCE; TOCOPHEROLS; TRANSFECTION;

EID: 84860737273     PISSN: 15438384     EISSN: 15438392     Source Type: Journal    
DOI: 10.1021/mp200435y     Document Type: Article

References (59)

1. Luo, D.; Saltzman, W. M. Synthetic DNA delivery systems Nat. Biotechnol. 2000, 18, 33-37
2. El-Aneed, A. An overview of current delivery systems in cancer gene therapy J. Controlled Release 2004, 94, 1-14
3. Simoes, S.; Filipe, A.; Feneca, H.; Mano, M.; Penacho, N.; Duzgunes, N. Cationic liposomes for gene delivery Expert Opin. Drug Delivery 2005, 2, 237-254
4. Unciti-Broceta, A.; Holder, E.; Jones, L. J.; Stevenson, B.; Turner, A. R.; Porteous, D. J.; Boyd, A. C.; Bradley, M. Tripod-like cationic lipids as gene carriers J. Med. Chem. 2008, 51, 4076-4084
5. Lynn, D. M.; Anderson, D. G.; Putman, D.; Langer, R. Accelerated discovery of synthetic transfection vectors: Parallel synthesis and screening of a degradable polymer library J. Am. Chem. Soc. 2001, 123, 8155-8156
6. Choi, J. S.; Joo, D. K.; Kim, C. H.; Kim, K.; Park, J. S. 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. 2000, 122, 474-480
7. Luo, D.; Haverstick, K.; Belcheva, N.; Han, E.; Saltzman, W. M. Poly(ethylene glycol)-conjugated PAMAM dendrimer for biocompatible, high-efficiency DNA delivery Macromolecules 2002, 35, 3456-3462
8. Srinivas, R.; Samanta, S.; Chaudhuri, A. Cationic amphiphiles: promising carriers of genetic materials in gene therapy Chem. Soc. Rev. 2009, 38, 3326-3338
9. Bhattacharya, S.; Bajaj, A. Advances in gene delivery through molecular design of cationic lipids Chem. Commun. 2009, 31, 4632-4656
10. Felgner, P. L.; Gadek, T. R.; Holm, M.; Roman, R.; Fhan, H. W.; Wenz, M.; Nortrop, 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
11. Koynova, R.; Tenchov, B. Recent patents in cationic lipid carriers for delivery of nucleic acids Recent Pat. DNA Gene Seq. 2011, 5 (1) 8-27
12. Koynova, R.; Tenchov, B. Cationic lipids: molecular structure/ transfection activity relationships and interactions with biomembranes Top. Curr. Chem. 2010, 296, 51-93
13. de Jesus, M. B.; Ferreira, C. V.; de Paula, E.; Hoekstra, D.; Zuhorn, I. S. Design of solid lipid nanoparticles for gene delivery into prostate cancer J. Controlled Release 2010, 148 (1) 89-90
14. Hoekstra, D.; Rejman, J.; Wasungu, L.; Shi, F.; Zuhorn, I. Gene delivery by cationic lipids: in and out of an endosome Biochem. Soc. Trans. 2007, 35 (Part 1) 68-71
15. Zuhorn, I. S.; Engberts, J. B.; Hoekstra, D. Gene delivery by cationic lipid vectors: overcoming cellular barriers Eur. Biophys. J. 2007, 36 (4-5) 349-362
16. Wasungu, L.; Hoekstra, D. Cationic lipids, lipoplexes and intracellular delivery of genes J. Controlled Release 2006, 116 (2) 255-264
17. Caracciolo, G.; Pozzi, D.; Caminiti, R.; Marchini, C.; Montani, M.; Amici, A.; Amenitsch, H. Transfection efficiency boost by designer multicomponent lipoplexes Biochim. Biophys. Acta 2007, 1768 (9) 2280-2292
18. Li, W.; Szoka, F. C., Jr. Lipid-based nanoparticles for nucleic acid delivery Pharm. Res. 2007, 24 (3) 438-449
19. Singh, R. S.; Mukherjee, K.; Banerjee, R.; Chaudhuri, A.; Hait, S. K.; Moulik, S.; Ramadas, Y.; Vijayalakshmi, A.; Rao, N. M. Anchor dependency for non-glycerol based cationic lipofectins: mixedbag of regular and anomalous transfection profiles Chem.-Eur. J. 2002, 8, 900-909
20. Koynova, R.; Tenchov, B.; Wang, L.; Macdonald, R. C. Hydrophobic moiety of cationic lipids strongly modulates their transfection activity Mol. Pharm. 2009, 6 (3) 951-958
21. Felgner, J. H.; Kumar, R.; Sridhar, C. N.; Wheeler, C. J.; Tsai, Y. J.; Border, R.; Ramsey, P.; Martin, M.; Felgner, P. L. Enhanced gene delivery and mechanism studies with a series of cationic lipid formulations J. Biol. Chem. 1994, 269, 2550-2561
22. Majeti, B. K.; Singh, R. S.; Yadav, S. K.; Reddy, S. B.; Ramkrishna, S.; Diwan, P. V.; Madhavendra, S. S.; Chaudhuri, A. Enhanced intravenous transgene expression in mouse lung using cyclic-head cationic lipids Chem. Biol. 2004, 11, 427-437
23. Bhavani, K.; Srilakshmi, P. V. Influence of Minor Backbone Structural Variations in Modulating the in Vitro Gene Transfer Efficacies of α-Tocopherol Based Cationic Transfection Lipids Bioconjugate Chem. 2011, 22 (12) 2581-2592
24. Rajesh, M.; Sen, J.; Srujan, M.; Mukherjee, K.; Sridhar, B.; Chaudhuri, A. Dramatic influence of the orientation of linker between hydrophilic and hydrophobic lipid moiety in liposomal gene delivery J. Am. Chem. Soc. 2007, 129, 11408-11420
25. Mukthavaram, R.; Marepally, S.; Mahidhar, Y. V.; Naidu, V. G. M.; Ramakrishna, S.; Chaudhuri, A. Cationic glycolipids with cyclic and open galactose head-groups for the selective targeting of genes to mouse liver Biomaterials 2009, 30, 2369-2384
26. Bhavani, K.; Srilakshmi, P. V. Design, synthesis and in vitro transfection biology of novel tocopherol based monocationic lipids: a structure activity investigation J. Med. Chem. 2011, 54 (2) 548-561
27. Wasungu, L.; Stuart, M. C.; Scarzello, M.; Engberts, J. B.; Hoekstra, D. Lipoplexes formed from sugar-based gemini surfactants undergo a lamellar-to-micellar phase transition at acidic pH. Evidence for a non-inverted membrane-destabilizing hexagonal phase of lipoplexes Biochim. Biophys. Acta 2006, 1758 (10) 1677-1684
28. Dauty, E.; Remy, J.-S.; Blessing, T.; Behr, J.-P. Dimerizable cationic detergents with a low cmc condense plasmid DNA into nanometreic particles and transfect cells in cluture J. Am. Chem. Soc. 2001, 123, 9227-9234
29. Zuber, G.; Zammut-Italiano, L.; Dauty, E.; Behr, J.-P. Targeted gene delivery to cancer cells: Directed assembly of nanometric DNA particles coated with folic acid Angew. Chem., Int. Ed. 2003, 42, 2666-2669
30. Wetzer, B.; Byk, G.; Frederic, M.; Airiau, M.; Blanche, F.; Pitard, B.; Scherman, D. Reducible cationic lipids for gene transfer Biochem. J. 2001, 356, 747-756
31. Byk., G.; Wetzer, B.; Frederic, M.; Dubertret, C.; Pitard, B.; Jalsin, G.; Scherman, D. Reduction-sensitive lipoplyamines as a novel nonviral gene delivery system for modulated release of DNA with improved transgene expression J. Med. Chem. 2000, 43, 4377-4387
32. Sambrook, J.; Fritsch, E. F.; Maniatis, T. Molecular Cloning: A Laboratory Manual, 2 nd ed.; Cold Spring Harbor Laboratory Press: Cold Spring Harbor, NY, 1989.
33. Hansen, M.-B.; Neilson, S. E.; Berg, K. Re-examination and further development of a precise and rapid dye method for measuring cell growth/cell kill J. Immunol. Methods 1989, 119, 203-210
34. Bajaj, A.; Kondiah, P.; Bhattacharya, S. Design, synthesis and in vitro gene transfection efficacies of cholesterol based gemini lipids and their serum compatibility: a structure activity relationship J. Med. Chem. 2007, 50, 2432-2442
35. Bajaj, A.; Kondaiah, P.; Bhattacharya, S. Synthesis and gene transfer activities of novel serum compatible cholesterol based gemini lipids possessing oxy-ethylene type spacers Bioconjugate Chem. 2007, 18, 1537-1546
36. Matsui, K.; Sando, S.; Sera, T.; Aoyama, Y.; Sasaki, Y.; Komatsu, T.; Terashima, T.; Kikuchi, J.-I. Cerasome as an infusible, cell-friendly, and serum-compartible transfection agent in a viral size J. Am. Chem. Soc. 2006, 128, 3114-3115
37. Karmali, P. P.; Majeti, B. K.; Sreedhar, B.; Chaudhuri, A. In vitro gene transfer efficacies and serum compatibility profiles of novel mono-, di-, and tri-histidinylated cationic transfection lipids: a structure-activity investigation Bioconjugate Chem. 2006, 17, 159-171
38. Scriou, V.; Ciolina, C.; Lacroix, F.; Byk, G.; Scherman, D.; Wils, P. Cationic lipid-mediated gene transfer: effect of serum on cellular uptake and intracellular fate of lipopolyamine/DNA complexes Biochim. Biophys. Acta 1998, 1368, 276-288
39. Vitiello, L.; Bockhold, K.; Joshi, P. B.; Worton, R. G. Transfection of cultured myoblasts in high serum concentration with DODAC: DOPE liposomes Gene Ther. 1998, 5, 1306-1313
40. Turek, J.; Dubertret, C.; Jaslin, G.; Antonakis, K.; Scherman, D.; Pitard, B. Formulations which increase the size of lipoplexes prevent serum-associated inhibition of transfection J. Gene Med. 2000, 2, 32-40
41. Thierry, A. R.; Rabinovich, P.; Peng, B.; Mahan, L. C.; Bryant, J. L.; Gallo, R. C. Characterization of liposome-mediated gene delivery: expression, stability and pharmacokinetics of plasmid DNA Gene Ther. 1997, 4, 226-237
42. Zelphati, O.; Uyechi, L. S.; Barron, L. G.; Szoka, F. C., Jr. Effect of serum components on the physico-chemical properties of cationic lipid/oligonucleotide complexes and on their interactions with cells Biochim. Biophys. Acta 1998, 1390, 119-133
43. Yang, J. P.; Huang, L. Time-dependent maturation of cationic liposome-DNA complex for serum resistance Gene Ther. 1998, 5, 380-387
44. Yang, J. P.; Huang, L. Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA Gene Ther. 1997, 4, 950-960
45. Li, S.; Tseng, W. C.; Stolz, D. B.; Wu, S. P.; Watkins, S. C.; Huang, L. Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection Gene Ther. 1999, 6, 585-594
46. Jones, D. P.; Kurtz, J. C.; Samiec, P. S.; Sternberg, P.; Reed, R. L.; Brown, L. A. S. GSH measurement in human plasma. Evaluation of sample collection storage and derivatization conditions for analysis of dansyl derivatives by HPLC Clin. Chem. Acta 1998, 275, 175-184
47. Anderson, M. E. Glutathione: anoverview of biosynthesis and modulation Chem. Biol. Interact. 1998, 111-112, 1-14
48. Friesen, C.; Kiess, Y.; Debatin, K.-M. A critical role of glutathione in determining apoptosis sensitivity and resistance in leukemia cells Cell Death Diff. 2004, 11, S73-S85
49. Koltover, I.; Salditt, T.; Radler, J. O.; Safinya, C. R. An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery Science 1998, 281, 78-81
50. Mok, K. W.; Cullis, P. R. Structural and fusogenic properties of cationic liposomes in the presence of plasmid DNA Biophys. J. 1997, 73, 2534-2545
51. Pires, P.; Simoes, S.; Nir, S.; Gaspar, R.; Duzgunes, N.; Pedroso de Lima, M. C. Interaction of cationic liposomes and their DNA complexes with monocyticleukemia cells Biochim. Biophys. Acta 1999, 1418, 71-84
52. Stegmann, T.; Legendre, J.-Y. Gene transfer mediated by cationic lipids:lack of a correlation between lipid mixing and transfection Biochim. Biophys. Acta 1997, 1325, 71-79
53. Hui, S. W.; Langer, M.; Zhao, Y. L.; Ross, P.; Hurley, E.; Chan, K. Role of Helper Lipids in Cationic Liposome-Mediated Gene Transfer Biophys. J. 1996, 71, 590-599
54. Fasbender, A.; Marshall, J.; Moninger, T. O.; Grunst, T.; Cheng, S.; Welsh, M. J. Effect of co-lipids in enhancing cationic-lipid mediated gene transfer in vitro and in vivo Gene Ther. 1997, 4, 716-725
55. Traber, M.; Sies, H. Vitamin E in humans: Demand and delivery Annu. Rev. Nutr. 1996, 16, 321-347
56. Traber, M. G.; Arai, H. Molecular mechanisms of vitamin E transport Annu. Rev. Nutr. 1999, 19, 343-355
57. Sato, Y.; Hagiwara, K.; Arai, H.; Inoue, K. Purification and characterization of the α-tocopherol transfer protein from rat liver FEBS Lett. 1991, 288, 41-45
58. Hosomi, A.; Arita, M.; Sato, Y.; Kiyose, C.; Ueda, T.; Igarashi, O.; Arai, H.; Inoue, K. Affinity for α-tocopherol transfer protein as a determinant of the biological activities of vitamin E analogs FEBS Lett. 1997, 409, 105-108
59. Halks-Miller, M.; Guo, L. S.; Hamilton, R. L., Jr. Tocopherol- phospholipid liposomes: maximum content and stability to serum proteins Lipids 1985, 20, 195-200