A lipid-based delivery system for antisense oligonucleotides derived from a hydrophobic complex

Journal of Drug Targeting

Volumn 10, Issue 8, 2002, Pages 615-623

  Wong, F M P ^a,b,c   Macadam, S A ^a   Kim, A ^a   Oja, C ^a   Ramsay, E C ^a   Bally, M B ^a,b  

^a BRITISH COLUMBIA CANCER AGENCY   (Canada)

^b UNIVERSITY OF BRITISH COLUMBIA   (Canada)

^c ALZA CORPORATION   (United States)

Author keywords

Antisense oligonucleotides; B cell lymphoma cells; Lipid antisense oligonucleotide particles; Poly(ethylene glycol) conjugated phosphatidylethanolamine

Indexed keywords

AMMONIUM DERIVATIVE; ANTISENSE OLIGONUCLEOTIDE; CARBAMIC ACID DERIVATIVE; CHLOROFORM; DIMETHYLDIOCTADECYLAMMONIUM BROMIDE; FLUORESCEIN ISOTHIOCYANATE; LIPID; MACROGOL; MESSENGER RNA; METHANOL; PHOSPHATE; PHOSPHATIDYLETHANOLAMINE; PROTEIN BCL 2; SPERMINE DERIVATIVE; TRIFLUOROACETIC ACID; WATER;

B CELL LYMPHOMA; CONTROLLED STUDY; DOWN REGULATION; DRUG DELIVERY SYSTEM; EXTRACTION; HUMAN; HUMAN CELL; HYDROPHOBICITY; IN VITRO STUDY; INHIBITION KINETICS; LYMPHOMA CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; REVIEW; WESTERN BLOTTING;

BLOTTING, WESTERN; CELL LINE; CHEMISTRY, PHYSICAL; DNA; DRUG CARRIERS; FLUORESCEIN-5-ISOTHIOCYANATE; FLUORESCENT DYES; GENE EXPRESSION REGULATION; GENES, BCL-2; HUMANS; LIPIDS; LIPOSOMES; LYMPHOMA, B-CELL; OLIGONUCLEOTIDES, ANTISENSE; PHOSPHATIDYLETHANOLAMINES; PLASMIDS; POLYETHYLENE GLYCOLS;

EID: 0036965445     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.1080/1061186021000066246     Document Type: Review

References (51)

1. Adlakha-Hutcheon, G., Bally, M.B., Shew, C.R. and Madden, T.D. (1999) "Controlled destabilization of a liposomal drug delivery system enhances mitoxantrone antitumor activity", Nat. Biotechnol. 17, 775-779.
2. Allen, T.M., Hansen, C., Martin, F., Redemann, C. and Yau-Young, A. (1991) "Liposomes containing synthetic lipid derivatives of poly(ethylene glycol) show prolonged circulation half-lives in vivo", Biochim. Biophys. Acta 1066, 29-36.
3. Bally, M.B., Zhang, Y.-P., Wong, F.M.P., Wasan, E.K. and Reimer, D.L. (1997) "Lipid/DNA complexes as an intermediate in the preparation of particles for gene transfer: an alternative to cationic liposome/DNA aggregates", Adv. Drug Delivery Rev. 24, 275-290.
4. Bennett, C.F., Chiang, M.-Y., Chan, H., Shoemaker, J.E. and Mirabelli, C.K. (1992) "Cationic lipids enhance cellular uptake and activity of phosphorothioate antisense oligonucleotides", Mol. Pharmacol. 41, 1023-1033.
5. Biffi, A., Sersale, G., Cassetti, A., Villa, A., Bordignon, C., Assael, B.M. and Conese, M. (1999) "Restoration of bacterial killing activity of human respiratory cystic fibrosis cells through cationic vector-mediated cystic fibrosis transmembrane conductance regulator gene transfer", Hum. Gene Ther. 10, 1923-1930.
6. Bligh, E.G. and Dyer, W.J. (1959) "A rapid method of total lipid extraction and purification", Can. J. Biochem. Physiol. 37, 911-937.
7. Boiziau, C., Kurfurst, R., Cazenave, C., Roig, V., Thuong, N.T. and Toulme, J.T. (1991) "Inhibition of translation initiation by antisense oligonucleotides via an RNase-H independent mechanism", Nucleic Acids Res. 19, 1113-1119.
8. Boutorin, A.S., Gus'kova, L.V., Ivanova, E.M., Kobetz, N.D., Zarytova, V.F., Ryte, A.S., Yurchenko, L.V. and Vlassov, V.V. (1989) "Synthesis of alkylating oligonucleotide derivatives containing cholesterol or phenazinium residues at their 3′-terminus and their interaction with DNA within mammalian cells", FEBS Lett. 254, 129-132.
9. Bradley, A.J., Devine, D.V., Ansell, S.M., Janzen, J. and Brooks, D.E. (1998) "Inhibition of liposome-induced complement activation by incorporated poly(ethylene glycol)-lipids", Arch. Biochem. Biophys. 357, 185-194.
10. Capaccioli, S., Di Pasquale, G., Mini, E., Mazzei, T. and Quattrone, A. (1993) "Cationic lipids improve antisense oligonucleotide uptake and prevent degradation in cultured cells and in human serum", Biochem. Biophys. Res. Commun. 197, 818-825.
11. Citro, G., Perrotti, D., Cucco, C., D'Agnano, I., Sacchi, A., Zupi, G. and Calabretta, B. (1992) "Inhibition of leukemia cell proliferation by receptor-mediated uptake of c-myb antisense oligodeoxynucleotides", Proc. Natl Acad. Sci. USA 89, 7031-7035.
12. Gabizon, A. and Papahadjopoulos, D. (1988) "Liposome formulations with prolonged circulatin time in blood and enhanced uptake by tumors", Proc. Natl Acad. Sci. USA 85, 6949-6953.
13. Goodchild, J., Agrawal, S., Civeira, M.P., Sarin, P.S., Sun, D. and Zamecnik, P.C. (1988) "Inhibition of human immunodeficiency virus replication by antisense oligodeoxynucleotides", Proc. Natl Acad. Sci. USA 85, 5507-5511.
14. Haner, R. and Hall, J. (1997) "The sequence-specific cleavage of RNA by artificial chemical ribonucleases", Antisense Nucleic Acid Drug Dev. 7, 423-430.
15. Harvie, P., Wong, F.M. and Bally, M.B. (1998) "Characterization of lipid DNA interactions. I. Destabilization of bound lipids and DNA dissociation", Biophys. J. 75, 1040-1051.
16. Harvie, P., Wong, F.M.P. and Bally, M.B. (2000) "Use of poly(ethylene glycol)-lipid conjugates to regulate the surface attributes and transfectional activity of lipid-DNA particles", J. Pharm. Sci. 89, 652-663.
17. Ho, P.T. and Parkinson, D.R. (1997) "Antisense oligonucleotides as therapeutics for malignant diseases", Semin. Oncol. 24, 187-202.
18. Holland, J.W., Hui, C., Cullis, P.R. and Madden, T.D. (1996) "Poly(ethylene glycol)-lipid conjugates regulate the calcium-induced fusion of liposomes composed of phosphatidylethanolamine and phosphatidylserine", Biochemistry 35, 2618-2624.
19. Hu, Q., Shew, C.R., Bally, M.B. and Madden, T.D. (2001) "Programmable fusogenic vesicles for intracellular delivery of antisense oligodeoxynucleotides: enhanced cellular uptake and biological effects", Biochim. Biophys. Acta 1514, 1-13.
20. Jansen, B., Schlagmauer-Wadl, H., Brown, B.D., Bryan, R.N., Van Elsas, A., Muller, M., Wolff, K., Eichler, H.G. and Pehamberger, H. (1998) "Bcl-2 antisense therapy chemosensitizes human melanoma in SCID mice", Nat. Med. 4, 232-234.
21. Keith, F.J., Bradbury, D.A., Zhu, Y.M. and Russell, N.H. (1995) "Inhibition of bcl-2 with antisense oligonucleotides induces apoptosis and increases the sensitivity of AML blasts to Ara-C", Leukemia 9, 131-138.
22. Kirpotin, D., Hong, K., Mullah, N. and Zalipsky, S. (1996) "Liposomes with detachable polymer coating: destabilization and fusion of dioleoylphosphatidylethanolamine vesicles triggered by cleavage of surface-grafted poly(ethylene glycol)", FEBS Lett. 388, 115-118.
23. Klasa, R.J., Bally, M.B., Ng, R., Goldie, J.H., Gascoyne, R.D. and Wong, F.M. (2000) "Eradication of human non-Hodgkin's lymphoma in SCID mice by BCL-2 antisense oligonucleotides combined with low-dose cyclophosphamide", Clin. Cancer Res. 6, 2492-2500.
24. Lappalainen, K., Miettinen, R., Kellokoski, J. and Syrjänen, S. (1997) "Intracellular distribution of oligonucleotides delivery by cationic liposomes: light and electron microscopic study", J. Histochem. Cytochem. 45, 265-274.
25. Leonetti, J.P., Degols, G. and Lebleu, B. (1990) "Biological activity of oligonucleotide-poly(L-lysine) conjugates: mechanism of cell uptake", Bioconjug. Chem. 1, 149-153.
26. Leonetti, J.P., Degols, G., Clarenc, J.P., Mechti, N. and Lebleu, B. (1993) "Cell delivery and mechanisms of action of antisense oligonucleotides", Prog. Nucleic Acid Res. Mol. Biol. 44, 143-166.
27. Marcusson, E.G., Bhat, B., Manoharan, M., Bennett, C.F. and Dean, N.M. (1998) "Phosphorothioate oligodeoxyribonucleotides dissociate from cationic lipids before entering the nucleus", Nucleic Acids Res. 26, 2016-2023.
28. Miyashita, T. and Reed, J.C. (1993) "Bcl-2 oncoprotein blocks chemotherapy-induced apoptosis in a human leukemia cell line", Blood 81, 151-157.
29. Monia, B.P., Lesnik, E.A., Gonzalez, C., Lima, W.F., McGee, D., Guinosso, C.J., Kawasaki, A.M., Cook, P.D. and Freier, S.M. (1993) "Evaluation of 2′-modified oligonucleotides containing 2′-deoxy gaps as antisense inhibitors of gene expression", J. Biol. Chem. 268, 14514-14522.
30. Musso, A., Condon, T.P., West, G.A., De La Motte, C., Strong, S.A., Levine, A.D., Bennett, C.F. and Fiocchi, C. (1999) "Regulation of ICAM-1-mediated fibroblast-T cell reciprocal interaction: implications for modulation of gut inflammation", Gastroenterology 117, 546-556.
31. Needham, D., McIntosh, T.J. and Lasic, D.D. (1992) "Repulsive interactions and mechanical stability of polymer-grafted lipid membranes", Biochim. Biophys. Acta 1108, 40-48.
32. Pastorino, F., Stuart, D., Ponzoni, M. and Allen, T.M. (2001) "Targeted delivery of antisense oligonucleotides in cancer", J. Control. Release 74, 69-75.
33. Reed, J.C. (1997) "Double identity for proteins of the Bcl-2 family", Nature 387, 773-776.
34. Reed, J.C., Kitada, S., Takayama, S. and Miyashita, T. (1994) "Regulation of chemoresistance by the bcl-2 oncoprotem in non-Hodgkin's lymphoma and lymphocytic leukemia cell lines", Ann. Oncol. 5, 61-65.
35. Reimer, D.L., Zhang, Y., Kong, S., Wheeler, J.J., Graham, R.W. and Bally, M.B. (1995) "Formation of novel hydrophobic complexes between cationic lipids and plasmid DNA", Biochemistry 34, 12877-12883.
36. Rex, S., Zuckermann, M.J., Lafleur, M. and Silvius, J.R. (1998) "Experimental and Monte Carlo simulation studies of the thermodynamics of polyethyleneglycol chains grafted to lipid bilayers", Biophys. J. 75, 2900-2914.
37. Sambrook, J., Fritsch, E.F. and Maniatis, T. (1989) Molecular Cloning: A laboratory Manual (Cold Spring Harbor Laboratory, Cold Spring Harbor, NY).
38. Shi, F., Wasungu, L., Nomden, A., Stuart, M.C., Polushkin, E., Engberts, J.B. and Hoekstra, D. (2002) "Interference of poly(ethylene glycol)-lipid analogues with cationic- lipid-mediated delivery of oligonucleotides; role of lipid exchangeability and non-lamellar transitions", Biochem. J. 366, 333-341.
39. Stephenson, M.L. and Zamecnik, P.C. (1978) "Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide", Proc. Natl Acad. Sci. USA 75, 285-288.
40. Stuart, D.D., Kao, G.Y. and Allen, T.M. (2000) "A novel, long-circulating, and functional liposomal formulation of antisense oligodeoxynucleotides targeted against MDR1", Cancer Gene Ther. 7, 466-475.
41. Szoka, F.C., Xu, Y. and Zelphati, O. (1996) "How are nucleic acids released in cells from cationic lipid-nucleic acid complexes?", J. Liposome Res. 6, 567-587.
42. Walton, M.I., Whysong, D., O'Connor, P.M., Hockenbery, D., Korsmeyer, S.J. and Kohn, K.W. (1993) "Constitutive expression of human Bcl-2 modulates nitrogen mustard and camptothecin induced apoptosis", Cancer Res. 53, 1853-1861.
43. Webb, A., Cunningham, D., Cotter, F., Clarke, P.A., di Stefano, F., Ross, P., Corbo, M. and Dziewanowska, Z. (1997) "BCL-2 antisense therapy in patients with non-Hodgkin lymphoma", Lancet 349, 1137-1141.
44. Wong, F.M.P., Reimer, D.L. and Bally, M.B. (1996) "Cationic lipid binding to DNA: characterization of complex formation", Biochemistry 35, 5756-5763.
45. Wong, F.M.P., Harvie, P., Zhang, Y.-P., Ramsay, E.C. and Bally, M.B. (2002) "Phosphatidylethanolamine mediated destabilization of lipid based pDNA delivery systems", Int. J. Pharm., In press.
46. Xu, Y. and Szoka, F.C.J. (1996) "Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection", Biochemistry 35, 5616-5623.
47. Yang, E. and Korsmeyer, S.J. (1996) "Molecular thanatopsis: a discourse on the BCL2 family and cell death", Blood 88, 386-401.
48. Zelphati, O. and Szoka, F.C.J. (1996a) "Intracellular distribution and mechanism of delivery of oligonucleotides mediated by cationic lipids", Pharm. Res. 13, 1367-1372.
49. Zelphati, O. and Szoka, F.C. (1996b) "Mechanism of oligonucleotide release from cationic liposomes", Proc. Natl Acad. Sci. USA 93, 11493-11498.
50. Zamecnik, P.C. and Stephenson, M.L. (1978) "Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide", Proc. Natl Acad. Sci. USA 75, 280-284.
51. Zhang, Y-P., Reimer, D.L., Zhang, G., Lee, P.H. and Bally, M.B. (1997) "Self-assembling DNA-lipid particles for gene transfer", Pharm. Res. 14, 190-196.