Lipid encapsulation enables the effective systemic delivery of polyplex plasmid DNA

Molecular Therapy

Volumn 15, Issue 4, 2007, Pages 713-720

  Heyes, James ^a   Palmer, Lorne ^a   Chan, Kitty ^a   Giesbrecht, Cory ^a   Jeffs, Lloyd ^a   MacLachlan, Ian ^a  

^a Protiva Biotherapeutics   (Canada)

Author keywords

[No Author keywords available]

Indexed keywords

AZIRIDINE DERIVATIVE; MACROGOL; PLASMID DNA; POLYLYSINE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DRUG CLEARANCE; DRUG DISTRIBUTION; DRUG FORMULATION; DRUG POTENCY; DRUG TOLERABILITY; ENCAPSULATION; ENDOSOME; GENE DELIVERY SYSTEM; GENE EXPRESSION PROFILING; IN VITRO GENE TRANSFER; IN VIVO STUDY; LIPID BILAYER; LIVER; LIVER TOXICITY; LUNG; MALE; MOUSE; MOUSE STRAIN; NONHUMAN; POLYMERIZATION; REPORTER GENE; SPLEEN; SUBCUTANEOUS TISSUE TUMOR; TUMOR MODEL;

ANIMALS; CELL LINE, TUMOR; CRYOELECTRON MICROSCOPY; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; LIPOSOMES; MALE; MICE; MICE, INBRED A; NEUROBLASTOMA; PLASMIDS; TRANSFECTION;

MUS;

EID: 33947199239     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300101     Document Type: Article

References (41)

1. Hirko, A, Tang, FX and Hughes, JA (2003). Cationic lipid vectors for plasmid DNA delivery. Curr Med Chem 10: 1185-1193.
2. Liu, DX, Ren, T and Gao, X (2003). Cationic transfection lipids. Curr Med Chem 10: 1307-1315.
3. Miller, AD (2003). The problem with cationic liposome/micelle-based non-viral vector systems for gene therapy. Curr Med Chem 10: 1195-1211.
4. Niculescu-Duvaz, D, Heyes, J and Springer, CJ (2003). Structure-activity relationship in cationic lipid mediated gene transfection. Curr Med Chem 10: 1233-1261.
5. Merdan, T, Kopecek, J and Kissel, T (2002). Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv Drug Deliv Rev 54: 715-758.
6. De Smedt, S, Demeester, J and Hennink, W (2000). Cationic polymer based gene delivery systems. Pharm Res 17: 1425-1433.
7. Plank, C, Mechtler, K, Szoka, FC and Wagner, E (1996). Activation of the complement system by synthetic DNA complexes: A potential barrier for intravenous gene delivery. Hum Gene Ther 7: 1437-1446.
8. Verbaan, FJ, Oussoren, C, van Dam, IM, Takakura, Y, Hashida,M and Crommelin, DJ et al. (2001). The fate of poly(2-dimethyl amino ethyl)metharcylate-based polyplexes after intravenous administration. Int JPharm 214: 99-101.
9. Stamatatos, L, Leventis, R, Zuckermann, MJ and Silvius, JR (1988). Interactions of cationic lipid vesicles with negatively charged phospholipid vesicles and biological membranes. Biochemistry 27: 3917-3925.
10. Semple, S, Chonn, A and Cullis, P (1998). Interactions of liposomes and lipid-based carriers with blood proteins: Relation to clearance behavior in vivo. Adv Drug Deliv Rev 32: 3-17.
11. Chonn, A, Semple, SC and Cullis, PR (1992). Association of blood proteins with large unilamellar liposomes in vivo. J Biol Chem 267: 18759-18765.
12. Oupicky, D, Konak, C, Dash, P, Seymour, L and Ulbrich, K (1999). Effect of albumin and polyanion on the structure of DNA complexes with polycation containing hydrophilic nonionic block. Bioconjug Chem 10: 764-772.
13. Kursa, M, Walker, GF, Roessler, V, Ogris, M, Roedl, W and Kircheis, R et al. (2003). Novel shielded transferrin-polyethylene glycol-polyethyleneimine/DNA complexes for systemic tumor-targeted gene transfer. Bioconjug Chem 14: 222-231.
14. Ogris, M, Brunner, S, Schüller, S, Kircheis, R and Wagner, E (1999). PEGylated DNA/transferrin-PEI complexes: Reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther 6: 595-605.
15. Ogris, M, Walker, G, Blessing, T, Kircheis, R, Wolschek, M and Wagner, E (2003). Tumor-targeted gene therapy: Strategies for the preparation of ligand-polyethylene glycol-polyethyleneimine/DNA complexes. J Control Release 91: 173-181.
16. Mok, KWC, Lam, AMI and Cullis, PR (1999). Stabilized plasmid-lipid particles: Factors influencing plasmid entrapment and transfection properties. Biochim Biophys Acta Biomembranes 1419: 137-150.
17. Monck, MA, Mori, A, Lee, D, Tam, P, Wheeler, JJ and Cullis, PR et al. (2000). Stabilized plasmid-lipid particles: Pharmacokinetics and plasmid delivery to distal tumors following intravenous injection. J Drug Target 7: 439-452.
18. Wheeler, JJ, Palmer, L, Ossanlou, M, MacLachlan, I, Graham, RW and Zhang, YP et al. (1999). Stabilized plasmid-lipid particles: construction and characterization. Gene Ther 6: 271-281.
19. Ambegia, E, Ansell, S, Cullis, P, Heyes, J, Palmer, L and MacLachlan, I (2005). Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim Biophys Acta 1669: 155-163.
20. Jeffs, LB, Palmer, LR, Ambegia, EG, Giesbrecht, C, Ewanick, S and MacLachlan, I (2005). A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA. Pharm Res 22: 362-372.
21. Heyes, J, Palmer, L, Bremner, K and MacLachlan, I (2005). Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids. J Control Release 107: 276-287.
22. Heyes, J, Hall, K, Tailor, V, Lenz, R and MacLachlan, I (2006). Synthesis and characterisation of novel poly(ethylene glycol)-lipid conjugates suitable for use in drug delivery. J Control Release 112: 280-290.
23. Lungwitz, U, Breunig, M, Blunk, T and Gopferich, A (2005). Polyethylenimine-based non-viral gene delivery systems. Eur J Pharm Biopharm 60: 247-266.
24. Behr, JP (1994). Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconjug Chem 5: 382-389.
25. Fenske, DB and Cullis, PR (2005). Entrapment of small molecules and nucleic acid-based drugs in liposomes. Methods Enzymol Liposomes 391 (Part E): 7-40.
26. Dvorak, HF, Nagy, JA, Dvorak, JT and Dvorak, AM (1988). Identification and characterization of the blood vessels of solid tumors that are leaky to circulating macromolecules. Am J Pathol 133: 95-109.
27. Almgren, M, Edwards, K and Karlsson, G (2000). Cryo transmission electron microscopy of liposomes and related structures. Colloid Surface A Physiochem Eng Aspects 174: 3-21.
28. Tam, P, Monck, M, Lee, D, Ludkovski, O, Leng, EC and Clow, K et al. (2000). Stabilized plasmid lipid particles for systemic gene therapy. Gene Ther 7: 1867-1874.
29. Li, S and Huang, L (1997). In vivo gene transfer via intravenous administration of cationic lipid-protamine-DNA (LPD) complexes. Gene Ther 4: 891-900.
30. Li, S, Rizzo, MA, Bhattacharya, S and Huang, L (1998). Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther 5: 930-937.
31. Lasic, DD, Strey, H, Stuart, MCA, Podgornik, R and Frederik, PM (1997). The structure of DNA-liposome complexes. J Am Chem Soc 119: 832-833.
32. Li, B, Li, S, Tan, Y, Stolz, DB, Watkins, SC and Block, LH et al. (2000). Lyophilization of cationic lipid-protamine-DNA (LPD) complexes. J Pharm Sci 89: 355-364.
33. Wu, GY and Wu, CH (1987). Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J Biol Chem 262: 4429-4432.
34. Godbey, WT, Wu, KK and Mikos, AG (1999). Tracking the intracellular path of poly(ethylene imine)/DNA complexes for gene delivery. Proc Natl Acad Sci USA 96: 5177-5181.
35. Kunath, K, von Harpe, A, Petersen, H, Fischer, D, Voigt, K and Kissel, T et al. (2002). The structure of PEG-modified poly(ethylene imines) influences biodistribution and pharmacokinetics of their complexes with NF-kappaB decoy in mice. Pharm Res 19: 810-817.
36. Oupicky, D, Howard, K, Konak, C, Dash, P, Ulbrich, K and Seymour, L (2000). Steric stabilization of poly-L-lysine/DNA complexes by the covalent attachment of semitelechelic poly[N-(2-hydroxypropyl)methacrylamide]. Bioconjug Chem 11: 492-501.
37. Kircheis, R, Wightman, L, Schreiber, A, Robitza, B, Rossler, V and Kursa, M et al. (2001). Polyethylenimine/DNA complexes shielded by transferrin target gene expression to tumors after systemic application. Gene Ther 8: 28-40.
38. Mahato, RI, Anwer, K, Tagliaferri, F, Meaney, C, Leonard, P and Wadhwa, MS et al. (1998). Biodistribution and gene expression of lipid/plasmid complexes after systemic administration. Hum Gene Ther 9: 2083-2099.
39. Mortimer, I, Tam, P, MacLachlan, I, Graham, RW, Saravolac, EG and Joshi, PB (1999). Cationic lipid-mediated transfection of cells in culture requires mitotic activity. Gene Ther 6: 403-411.
40. Cox, PM and Goding, CR (1991). Transcription and cancer. Br J Cancer 63: 651-662.
41. Bally, MM, Mayer, LD, Hope, MJ and Nayar, R (1993). Pharmacodynamics of liposomal drug carriers: Methodological considerations. In: Gregoriadis G (ed). Liposome Technology, vol. III. CRC Press: London, UK, pp 27-41.