Lipid-based nanoparticles for nucleic acid delivery

Pharmaceutical Research

Volumn 24, Issue 3, 2007, Pages 438-449

  Li, Weijun ^a,b   Szoka Jr , Francis C ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b BAYER CORPORATION   (United States)

Author keywords

DNA; Gene therapy; Liposome; Oligonucleotide; Small interfering RNA

Indexed keywords

1,2 DIOLEOYLGLYCERO 3 PHOSPHOETHANOLAMINE; ALCOHOL; ANION; ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; CATION; FOLIC ACID; LIPID; LIPOPLEX; MACROGOL; NANOPARTICLE; NUCLEIC ACID; ORGANIC SOLVENT; PLASMID DNA; POLYMER; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

AQUEOUS SOLUTION; BIOCOMPATIBILITY; BLOOD COMPONENT; BRAIN TUMOR; DRUG DELIVERY SYSTEM; DRUG FORMULATION; DRUG HALF LIFE; DRUG SENSITIVITY; DRUG SOLUBILITY; ENCAPSULATION; EXTRACELLULAR MATRIX; GENE DELIVERY SYSTEM; GENE TARGETING; GENE TRANSFER; HUMAN; IN VIVO STUDY; LIPID COMPOSITION; NONHUMAN; PH; PHYSICAL CHEMISTRY; PRIORITY JOURNAL; RETICULOENDOTHELIAL SYSTEM; REVIEW;

ANIMALS; HUMANS; LIPIDS; MODELS, BIOLOGICAL; NANOCAPSULES; NUCLEIC ACIDS; TRANSFECTION;

EID: 33847099637     PISSN: 07248741     EISSN: 1573904X     Source Type: Journal    
DOI: 10.1007/s11095-006-9180-5     Document Type: Review

References (84)

1. L. C. Heller, K. Ugen, and R. Heller. Electroporation for targeted gene transfer. Expert Opin. Drug Deliv. 2:255-268 (2005).
2. J. E. Knapp and D. Liu. Hydrodynamic delivery of DNA. Methods Mol. Biol. 245:245-250 (2004).
3. R. I. Mahato and S. W. Kim. Pharmaceutical Perspectives of Nucleic Acid-based Therapeutics. Taylor & Francis, London, 2002.
4. P. L. Felgner, T. R. Gadek, M. Holm, R. Roman, H. W. Chan, M. Wenz, J. P. Northrop, G. M. Ringold, and M. Danielsen. Lipofection: a highly efficient, lipid-mediated DNA-transfection procedure. Proc. Natl. Acad. Sci. USA 84:7413-7417 (1987).
5. Y. Xu, S. W. Hui, P. Frederik, and F. C. Szoka Jr. Physicochemical characterization and purification of cationic lipoplexes. Biophys. J. 77:341-353 (1999).
6. J. O. Radler, I. Koltover, T. Salditt, and C. R. Safinya. Structure of DNAYcationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science 275:810-814 (1997).
7. N. S. Templeton, D. D. Lasic, P. M. Frederik, H. H. Strey, D. D. Roberts, and G. N. Pavlakis. Improved DNA: liposome complexes for increased systemic delivery and gene expression. Nat. Biotechnol. 15:647-652 (1997).
8. L. Huang, M. C. Hung, and E. Wagner. Nonviral Vectors for Gene Therapy. Academic, San Diego, 1999.
9. M. L. Edelstein, M. R. Abedi, J. Wixon, and R. M. Edelstein. Gene therapy clinical trials worldwide 1989-2004 - an overview. J. Gene Med. 6:597-602 (2004).
10. A. D. Judge, V. Sood, J. R. Shaw, D. Fang, K. McClintock, and I. MacLachlan. Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA. Nat. Biotechnol. 23:457-462 (2005).
11. J. S. Zhang, F. Liu, and L. Huang. Implications of pharmacokinetic behavior of lipoplex for its inflammatory toxicity. Adv. Drug Deliv. Rev. 57:689-698 (2005).
12. T. L. Jason, J. Koropatnick, and R. W. Berg. Toxicology of antisense therapeutics. Toxicol. Appl. Pharmacol. 201:66-83 (2004).
13. K. L. Brigham, B. Meyrick, B. Christman, M. Magnuson, G. King, and L. C. Berry Jr. In vivo transfection of murine lungs with a functioning prokaryotic gene using a liposome vehicle. Am. J. Med. Sci. 298:278-281 (1989).
14. L. G. Barron, L. Gagne, and F. C. Szoka Jr. Lipoplex-mediated gene delivery to the lung occurs within 60 minutes of intravenous administration. Hum. Gene Ther. 10:1683-1694 (1999).
15. L. G. Barron and F. C. Szoka. The perplexing delivery mechanism of lipoplexes. In L. Huang, M. C. Hung, and E. Wagner (eds.), Nonviral Vectors for Gene Therapy, Academic, San Diego, 1999, pp. 229-266.
16. C. Tros de Ilarduya, M. A. Arangoa, and N. Duzgunes. Transferrin- lipoplexes with protamine-condensed DNA for serum resistant gene delivery. Methods Enzymol. 373:342-356 (2003).
17. S. Simoes, A. Filipe, H. Faneca, M. Mano, N. Penacho, N. Duzgunes, and M. P. Limade. Cationic liposomes for gene delivery. Expert Opin. Drug Deliv. 2:237-254 (2005).
18. P. Harvie, F. M. P. Wong, and M. B. Bally. Use of poly(ethylene glycol)-lipid conjugates to regulate the surface attributes and transfection activity of lipid-DNA particles. J. Pharm. Sci. 89:652-663 (2000).
19. W. J. Li, Z. H. Huang, J. A. MacKay, S. Grube, and F. C. Szoka. Low-pH-sensitive poly(ethylene glycol) (PEG)-stabilized plasmid nanlipoparticles: effects of PEG chain length, lipid composition and assembly conditions on gene delivery. J. Gene Med. 7:67-79 (2005).
20. J. S. Choi, J. A. MacKay, and F. C. Szoka Jr. Low-pH-sensitive PEG-stabilized plasmid-lipid nanoparticles: preparation and characterization. Bioconjug. Chem. 14:420-429 (2003).
21. J. J. Wheeler, L. Palmer, M. Ossanlou, I. MacLachlan, R. W. Graham, Y. P. Zhang, M. J. Hope, P. Scherrer, and P. R. Cullis. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther. 6:271-281 (1999).
22. Y. P. Zhang, L. Sekirov, E. G. Saravolac, J. J. Wheeler, P. Tardi, K. Clow, E. Leng, R. Sun, P. R. Cullis, and P. Scherrer. Stabilized plasmid-lipid particles for regional gene therapy: formulation and transfection properties. Gene Ther. 6: 1438-1447 (1999).
23. L. B. Jeffs, L. R. Palmer, E. G. Ambegia, C. Giesbrecht, S. Ewanick, and I. MacLachlan. A scalable, extrusion-free method for efficient liposomal encapsulation of plasmid DNA. Pharma. Res. 22:362-372 (2005).
24. M. C. Woodle, L. R. Collins, E. Sponsler, N. Kossovsky, D. Papahadjopoulos, and F. J. Martin. Sterically stabilized liposomes. Reduction in electrophoretic mobility but not electrostatic surface potential. Biophys. J. 61:902-910 (1992).
25. P. Opanasopit, M. Nishikawa, and M. Hashida. Factors affecting drug and gene delivery: effects of interaction with blood components. Crit. Rev. Ther. Drug Carr. Syst. 19:191-233 (2002).
26. B. Alberts, A. Johnson, J. Lewis, M. Raff, K. Roberts, and P. Walter. Molecular Biology of the Cell. Garland, New York, 2002.
27. I. A. Khalil, K. Kogure, H. Akita, and H. Harashima. Uptake pathways and subsequent intracellular trafficking in nonviral gene delivery. Pharmacol. Rev. 58:32-45 (2006).
28. E. Mastrobattista, M. A. van der Aa, W. E. Hennink, and D. J. Crommelin. Artificial viruses: a nanotechnological approach to gene delivery. Nat. Rev. Drug Discovery 5:115-121 (2006).
29. S. C. Semple, S. K. Klimuk, T. O. Harasym, N. Dos Santos, S. M. Ansell, K. F. Wong, N. Maurer, H. Stark, P. R. Cullis, M. J. Hope, and P. Scherrer. Efficient encapsulation of antisense oligonucleotides in lipid vesicles using ionizable aminolipids: formation of novel small multilamellar vesicle structures. Biochim. Biophys. Acta 1510:152-166 (2001).
30. Z. Huang, W. Li, J. A. MacKay, and F. C. Szoka Jr. Thiocholesterol-based lipids for ordered assembly of bioresponsive gene carriers. Molec. Ther. 11:409-417 (2005).
31. D. P. Siegel and R. M. Epand. The mechanism of lamellar-toinverted hexagonal phase transitions in phosphatidylethanolamine: implications for membrane fusion mechanisms. Biophys. J. 73:3089-3111 (1997).
32. M. E. Hayes, D. C. Drummond, D. B. Kirpotin, W. W. Zheng, C. O. Noble, J. W. Park, J. D. Marks, C. C. Benz, and K. Hong. Genospheres: self-assembling nucleic acidYlipid nanoparticles suitable for targeted gene delivery. Gene Ther. 13:646-651 (2006).
33. R. J. Lee and L. Huang. Folate-targeted, anionic liposome entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J. Biol. Chem. 271:8481-8487 (1996).
34. E. Mastrobattista, R. H. Kapel, M. H. Eggenhuisen, P. J. Roholl, D. J. Crommelin, W. E. Hennink, and G. Storm. Lipid-coated polyplexes for targeted gene delivery to ovarian carcinoma cells. Cancer Gene Ther. 8:405-413 (2001).
35. D. B. Fenske, I. MacLachlan, and P. R. Cullis. Stabilized plasmidYlipid particles: a systemic gene therapy vector. Method Enzymol. 346:36-71 (2002).
36. E. Ruoslahti and D. Rajotte. An address system in the vasculature of normal tissues and tumors. Annu. Rev. Immunol. 18:813-827 (2000).
37. E. S. Scott, J. W. Wiseman, M. J. Evans, and W. H. Colledge. Enhanced gene delivery to human airway epithelial cells using an integrin-targeting lipoplex. J. Gene. Med. 3:125-134 (2001).
38. P. Harvie, B. Dutzar, T. Galbraith, S. Cudmore, D. O'Mahony, P. Anklesaria, and R. Paul. Targeting of lipid-protamine-DNA (LPD) lipopolyplexes using RGD motifs. J. Liposome Res. 13:231-247 (2003).
39. Y. Lu and P. S. Low. Folate-mediated delivery ofmacromolecular anticancer therapeutic agents. Adv. Drug Deliv. Rev. 54:675-693 (2002).
40. W. Guo, T. Lee, J. Sudimack, and R. J. Lee. Receptor-specific delivery of liposomes via folate-PEG-chol. J. Liposome Res. 10:179-195 (2000).
41. L. Xu, K. F. Pirollo, and E. H. Chang. Tumor-targeted p53-gene therapy enhances the efficacy of conventional chemo/radiotherapy. J. Control. Release 74:115-128 (2001).
42. W. Yu, K. F. Pirollo, A. Rait, B. Yu, L. M. Xiang, W. Q. Huang, Q. Zhou, G. Ertem, and E. H. Chang. A sterically stabilized immunolipoplex for systemic administration of a therapeutic gene. Gene Ther. 11:1434-1440 (2004).
43. S. Han, R. I. Mahato, and S. W. Kim. Water-soluble lipopolymer for gene delivery. Bioconjug. Chem. 12:337-345 (2001).
44. R. I. Mahato, M. Lee, S. Han, A. Maheshwari, and S. W. Kim. Intratumoral delivery of p2CMVmIL-12 using water-soluble lipopolymers. Molec. Ther. 4:130-138 (2001).
45. X. Gao and L. Huang. Potentiation of cationic liposome mediated gene delivery by polycations. Biochemistry 35: 1027-1036 (1996).
46. J. Dileo, R. Banerjee, M. Whitmore, J. V. Nayak, L. D. Falo Jr., and L. Huang. Lipid-protamine-DNA-mediated antigen delivery to antigen-presenting cells results in enhanced anti-tumor immune responses. Molec. Ther. 7:640-648 (2003).
47. R. E. Eliaz and F. C. Szoka Jr. Robust and prolonged gene expression from injectable polymeric implants. Gene Ther. 9:1230-1237 (2002).
48. N. Shi, Y. Zhang, C. Zhu, R. J. Boado, and W. M. Pardridge. Brain-specific expression of an exogenous gene after i.v. administration. Proc. Natl. Acad. Sci. USA 98:12754-12759 (2001).
49. Y. Zhang, C. Zhu, and W. M. Pardridge. Antisense gene therapy of brain cancer with an artificial virus gene delivery system. Molec. Ther. 6:67-72 (2002).
50. Y. Zhang, R. J. Boado, and W. M. Pardridge. In vivo knockdown of gene expression in brain cancer with intravenous RNAi in adult rats. J. Gene Med. 5:1039-1045 (2003).
51. H. E. Hofland, L. Shephard, and S. M. Sullivan. Formation of stable cationic lipid/DNA complexes for gene transfer. Proc. Natl. Acad. Sci. USA 93:7305-7309 (1996).
52. C. Y. Wang and L. Huang. pH-sensitive immunoliposomes mediate target-cell-specific delivery and controlled expression of a foreign gene in mouse. Proc. Natl. Acad. Sci. USA 84:7851-7855 (1987).
53. K. W. Mok, A. M. Lam, and P. R. Cullis. Stabilized plasmid-lipid particles: factors influencing plasmid entrapment and transfection properties. Biochim. Biophys. Acta 1419:137-150 (1999).
54. P. Tam, M. Monck, D. Lee, O. Ludkovski, E. C. Leng, K. Clow, H. Stark, P. Scherrer, R. W. Graham, and P. R. Cullis. Stabilized plasmid-lipid particles for systemic gene therapy. Gene Ther. 7:1867-1874 (2000).
55. E. Ambegia, S. Ansell, P. Cullis, J. Heyes, L. Palmer, and I. MacLachlan. Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim. Biophys. Acta 1669:155-163 (2005).
56. A. Judge, K. McClintock, J. R. Phelps, and I. Maclachlan. Hypersensitivity and loss of disease site targeting caused by antibody responses to PEGylated liposomes. Molec. Ther. 13:328-337 (2006).
57. X. Guo and F. C. Szoka. Steric stabilization of fusogenic liposomes by a low-pH sensitive PEG-diortho ester-lipid conjugate. Bioconjug. Chem. 12:291-300 (2001).
58. O. T. Jones, J. P. Earnest, and M. G. McNamee. Solubilization and reconstitution of membrane proteins. In J. B. C. Findlay and W. H. Evans (eds.), Biological Membranes: A Practical Approach, IRL, Oxford, 1987, pp. 139-177.
59. S. Batzri and E. D. Korn. Single bilayer liposomes prepared without sonication. Biochim. Biophys. Acta 298:1015-1019 (1973).
60. M. J. Campbell. Lipofection reagents prepared by a simple ethanol injection technique. Biotechniques 18:1027-1032 (1995).
61. P. G. Arscott, C. Ma, J. R. Wenner, and V. A. Bloomfield. DNA condensation by cobalt hexaammine (III) in alcohol-water mixtures: dielectric constant and other solvent effects. Biopolymers 36:345-364 (1995).
62. J. Piskur and A. Rupprecht. Aggregated DNA in ethanol solution. FEBS Lett. 375:174-178 (1995).
63. A. L. Bailey and S. M. Sullivan. Efficient encapsulation of DNA plasmids in small neutral liposomes induced by ethanol and calcium. Biochim. Biophys. Acta 1468:239-252 (2000).
64. N. Maurer, K. F. Wong, H. Stark, L. Louie, D. McIntosh, T. Wong, P. Scherrer, S. C. Semple, and P. R. Cullis. Spontaneous entrapment of polynucleotides upon electrostatic interaction with ethanol-destabilized cationic liposomes. Biophys. J. 80:2310-2326 (2001).
65. D. V.Morrissey, J. A. Lockridge, L. Shaw, K. Blanchard, K. Jensen, W. Breen, K. Hartsough, L. Machemer, S. Radka, V. Jadhav, N. Vaish, S. Zinnen, C. Vargeese, K. Bowman, C. S. Shaffer, L. B. Jeffs, A. Judge, I. MacLachlan, and B. Polisky. Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs. Nat. Biotechnol. 23:1002-1007 (2005).
66. A. D. Judge, G. Bola, A. C. Lee, and I. MacLachlan. Design of noninflammatory synthetic siRNA mediating potent gene silencing in vivo. Molec. Ther. 13:494-505 (2006).
67. T. S. Zimmermann, A. C. Lee, A. Akinc, B. Bramlage, D. Bumcrot, M. N. Fedoruk, J. Harborth, J. A. Heyes, L. B. Jeffs, M. John, A. D. Judge, K. Lam, K. McClintock, L. V. Nechev, L. R. Palmer, T. Racie, I. Rohl, S. Seiffert, S. Shanmugam, V. Sood, J. Soutschek, I. Toudjarska, A. J. Wheat, E. Yaworski, W. Zedalis, V. Koteliansky, M. Manoharan, H. P. Vornlocher, and I.MacLachlan. RNAi-mediated gene silencing in non-human primates. Nature 441:111-114 (2006).
68. W. Li and F. C. Szoka. Bioresponsive targeted charge neutral lipid vesicles for systemic gene delivery. In T. Friedmann and J. Rossi (eds.), Gene Transfer: Delivery and Expression of DNA and RNA, Cold Spring Harbor Laboratory Press, New York, 2006, pp. 441-450.
69. E. A. Murphy, A. J. Waring, S. M. Haynes, and K. J. Longmuir. Compaction of DNA in an anionic micelle environment followed by assembly into phosphatidylcholine liposomes. Nucleic Acids Res. 28:2986-2992 (2000).
70. E. A. Murphy, A. J. Waring, J. C. Murphy, R. C. Willson, and K. J. Longmuir. Development of an effective gene delivery system: a study of complexes composed of a peptide-based amphiphilic DNA compaction agent and phospholipid. Nucleic Acids Res. 29:3694-3704 (2001).
71. C. Chittimalla, L. Zammut-Italiano, G. Zuber, and J. P. Behr. Monomolecular DNA nanoparticles for intravenous delivery of genes. J. Am. Chem. Soc. 127:11436-11441 (2005).
72. G. Zuber, L. Zammut-Italiano, E. Dauty, and J. P. Behr. Targeted gene delivery to cancer cells: Directed assembly of nanometric DNA particles coated with folic acid. Angew. Chem., Int. Ed. Engl. 42:2666-2669 (2003).
73. M. Ouyang, J. S. Remy, and F. C. Szoka. Controlled template assisted assembly of plasmid DNA into nanometric particles with high DNA concentration. Bioconjug. Chem. 11:104-112 (2000).
74. E. Dauty, J. S. Remy, T. Blessing, and J. P. Behr. Dimerizable cationic detergents with a low cmc condense plasmid DNA into nanometric particles and transfect cells in culture. J. Am. Chem. Soc. 123:9227-9234 (2001).
75. T. Blessing, E. Dauty, J. S. Remy, and J. P. Behr. Dimerizable detergents as gene transfer vectors. J. Liposome Res. 10:321-327 (2000).
76. J. P. Rolland, B. W. Maynor, L. E. Euliss, A. E. Exner, G. M. Denison, and J. M. DeSimone. Direct fabrication and harvesting of monodisperse, shape-specific nanobiomaterials. J. Am. Chem. Soc. 127:10096-10100 (2005).
77. F. C. Szoka, Y. Xu, and O. Zelphati. How are nucleic acids released in cells from cationic lipid-nucleic acid complexes. J. Liposome Res. 6:567-587 (1996).
78. Y. Xu and F. C. Szoka Jr. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 35:5616-5623 (1996).
79. O. Zelphati and F.C. Szoka Jr. Mechanism of oligonucleotide release from cationic liposomes. Proc. Natl. Acad. Sci. USA 93:11493-11498 (1996).
80. E. Dauty and A. S. Verkman. Actin cytoskeleton as the principal determinant of size-dependent DNA mobility in cytoplasm: a new barrier for non-viral gene delivery. J. Biol. Chem. 280:7823-7828 (2005).
81. M. A. Monck, A. Mori, D. Lee, P. Tam, J. J. Wheeler, P. R. Cullis, and P. Scherrer. Stabilized plasmid-lipid particles: pharmacokinetics and plasmid delivery to distal tumors following intravenous injection. J. Drug Target. 7:439-452 (2000).
82. D. B. Fenske, I. MacLachlan, and P. R. Cullis. Long-circulating vectors for the systemic delivery of genes. Curr. Opin. Mol. Ther. 3:153-158 (2001).
83. M. E. Hayes, D. C. Drummond, K. Hong, J. W. Park, J. D. Marks, and D. B. Kirpotin. Assembly of nucleic acid-lipid nanoparticles from aqueous-organic monophases. Biochim. Biophys. Acta 1758:429-442 (2006).
84. X. Guo, J. A. MacKay, and F. C. Szoka. Mechanism of pH triggered collapse of phosphatidylethanolamine liposomes stabilized by an ortho ester polyethyleneglycol lipid. Biophys. J. 84:1784-1795 (2003).