Cationic liposome-nucleic acid complexes for gene delivery and silencing: Pathways and mechanisms for plasmid DNA and siRNA

Topics in Current Chemistry

Volumn 296, Issue , 2010, Pages 191-226

  Ewert, Kai K ^a   Zidovska, Alexandra ^a,b   Ahmad, Ayesha ^a,c   Bouxsein, Nathan F ^a   Evans, Heather M ^a,d   McAllister, Christopher S ^a,e   Samuel, Charles E ^a   Safinya, Cyrus R ^a  

^a UNIVERSITY OF CALIFORNIA   (United States)

^b HARVARD MEDICAL SCHOOL   (United States)

^c Dynavax Technologies Corporation   (United States)

^d National Nanotechnology Coordination Office   (United States)

^e UNIVERSITY OF CALIFORNIA SAN DIEGO   (United States)

Author keywords

Cholesterol; Gene delivery; Multivalent cationic lipid; siRNA; Small angle X ray scattering

Indexed keywords

CATION; LIPOSOME; NUCLEIC ACID; SMALL INTERFERING RNA;

ARTICLE; CHEMISTRY; GENE SILENCING; GENETIC TRANSFECTION; GENETICS; METABOLISM; METHODOLOGY; PLASMID;

CATIONS; GENE SILENCING; LIPOSOMES; NUCLEIC ACIDS; PLASMIDS; RNA, SMALL INTERFERING; TRANSFECTION;

EID: 78649305928     PISSN: 03401022     EISSN: None     Source Type: Book Series    
DOI: 10.1007/128_2010_70     Document Type: Article

References (98)

1. Safinya CR, Koltover I (1999) Self assembled structures of lipid-DNA nonviral gene delivery systems from synchrotron X-ray diffraction. In: Huang L, Hung M-C, Wagner E (eds) Non-viral vectors for gene therapy. Academic, San Diego
2. Ewert KK, Slack NL, Ahmad A, Evans HM, Lin A, Samuel CE, Safinya CR (2004) Cationic lipid-DNA complexes for gene therapy: understanding the relationship between complex structures and gene delivery pathways at the molecular level. Curr Med Chem 11:1241-1253
3. Ewert K, Evans H, Ahmad A, Slack L, Lin A, Martin-Herranz A, Safinya CR (2005) Lipoplex structures and their distinct cellular pathways. In: Huang L, Hung M-C, Wagner E (eds) Non-viral vectors for gene therapy, 2nd edn. Part I (Advances in genetics 53). Elsevier, San Diego
4. Ewert K, Ahmad A, Evans H, Safinya CR (2005) Cationic lipid-DNA complexes for non-viral gene therapy: relating supramolecular structures to cellular pathways. Expert Opin Biol Ther 5:33-53
5. Safinya CR, Ewert KK, Ahmad A, Evans HM, Raviv U, Needleman DJ, Lin AJ, Slack NL, George CX, Samuel CE (2006) Cationic liposome-DNA complexes: from liquid crystal science to gene delivery applications. Phil Transact Royal Soc A 364:2573-2596
6. Ewert KK, Samuel CE, Safinya CR (2008) Lipid-DNA interactions: structure-function studies of nanomaterials for gene delivery. In: Dias R, Lindman B (eds) Interaction of DNA with surfactant and polymers. Blackwell, Boston, M.A.
7. Ewert KK, Ahmad A, Bouxsein NF, Evans HM, Safinya CR (2008) Non-viral gene delivery with cationic liposome-DNA complexes. In: Le Doux J (ed) Gene therapy protocols, 3rd edn. Humana, Totowa, NJ
8. Edelstein ML, Abedi MR, Wixon J, Edelstein RM (2004) Gene therapy clinical trials worldwide 1989-2004 - an overview. J Gene Med 6:597-602
9. Edelstein ML, Abedi MR, Wixon JJ (2007) Gene therapy clinical trials worldwide to 2007: an update. Gene Med 9:833-842
10. The journal of gene medicine clinical trial site. http://www.wiley.co.uk/ genetherapy/clinical Accessed 5 Nov 2009
11. Huang L, Hung M-C, Wagner E (eds) (1999) Nonviral vectors for gene therapy. Academic, San Diego, 91-117.
12. Huang L, Hung M-C, Wagner E (2005) Non-viral vectors for gene therapy, 2nd edn, Advances in genetics. Elsevier, San Diego
13. Mahato RI, Kim SW (eds) (2002) Pharmaceutical perspectives of nucleic acid-based therapeutics. Taylor & Francis, London and New York
14. Chesnoy S, Huang L (2000) Structure and function of lipid-DNA complexes for gene delivery. Annu Rev Biophys Biomol Struct 29:27-47
15. Byk G, Dubertret C, Escriou V, Frederic M, Jaslin G, Rangara R, Pitard B, Crouzet J, Wils P, Schartz B, Scherman D (1998) Synthesis, activity, and structure-activity relationship studies of novel cationic lipids for DNA transfer. J Med Chem 41:224-235
16. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeniex B, Behr J-P (1995) A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci USA 92:7297-7301
17. Haensler J, Szoka FC (1993) Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconjug Chem 4:372-379
18. Tang MX, Redemann CT, Szoka FC Jr (1996) In vitro gene delivery by degraded polyami-doamine dendrimers. Bioconjug Chem 7:703-714
19. Thomas CE, Ehrhardt A, Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 4:346-358
20. Tranchant I, Thompson B, Nicolazzi C, Mignet N, Scherman D (2004) Physicochemical optimisation of plasmid delivery with cationic lipids. J Gene Med 6:S24-S35
21. Ahmad A, Evans HM, Ewert K, George CX, Samuel CE, Safinya CR (2005) New multivalent lipids reveal bell-curve for transfection versus membrane charge density: nonviral lipid-DNA complexes for gene delivery. J Gene Med 7:739-748
22. Rädler JO, Koltover I, Salditt T, Safinya CR (1997) Structure of DNA-cationic liposome complexes: DNA intercalation in multilamellar membranes in distinct interhelical packing regimes. Science 275:810-814
23. Koltover I, Salditt T, Rädler JO, Safinya CR (1998) An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. Science 281:78-81
24. Ewert KK, Evans HM, Zidovska A, Bouxsein NF, Ahmad A, Safinya CR (2006) A columnar phase of dendritic lipid-based cationic liposome-DNA complexes for gene delivery: hexago-nally ordered cylindrical micelles embedded in a DNA honeycomb lattice. J Am Chem Soc 128:3998-4006
25. Lin AJ, Slack NL, Ahmad A, George CX, Samuel CE, Safinya CR (2003) Three-dimensional imaging of lipid gene-carriers: membrane charge density controls universal transfection behavior in lamellar cationic liposome-DNA complexes. Biophys J 84:3307-3316
26. Ewert K, Ahmad A, Evans HM, Schmidt H-W, Safinya CR (2002) Efficient synthesis and cell-transfection properties of a new multivalent cationic lipid for non-viral gene delivery. J Med Chem 45:5023-5029
27. Zidovska A, Evans HM, Ahmad A, Ewert KK, Safinya CR (2009) The role of cholesterol and structurally related molecules in enhancing transfection by cationic liposome-DNA complexes. J Phys Chem 113:5208-5216
28. Song YK, Liu F, Chu S, Liu D (1997) Characterization of cationic liposome-mediated gene transfer in vivo by intravenous administration. Hum Gene Ther 8:1585-1594
29. Liu Y, Mounkes LC, Liggitt HD, Brown CS, Solodin I, Heath TD, Debs RJ (1997) Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat Biotechnol 15:167-173
30. Audouy S, Molema G, de Leij L, Hoekstra D (2000) Serum as a modulator of lipoplex-mediated gene transfection: dependence of amphiphile, cell type and complex stability. J Gene Med 2:465-476
31. Crook K, Stevenson BJ, Dubouchet M, Poreous DJ (1998) Inclusion of cholesterol in DOTAP transfection complexes increases the delivery of DNA to cells in vitro in the presence of serum. Gene Ther 5:137-143
32. Li S, Tseng WC, Stolz DB, Wu SP, Watkins SC, Huang L (1999) Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection. Gene Ther 6:585-594
33. Ilies MA, Johnson BH, Makori F, Miller A, Seitz WA, Thompson EB, Balaban AT (2005) Pyridinium cationic lipids in gene delivery: an in vitro and in vivo comparison of transfection efficiency versus a tetraalkylammonium congener. Arch Biochem Biophys 435:217-226
34. Kim CK, Haider KH, Choi SH, Choi EJ, Ahn WS, Kim YB (2002) Nonviral vector for efficient gene transfer to human ovarian adenocarcinoma cells. Gynecol Oncol 84:85-93
35. Kim CK, Choi EJ, Choi SH, Park JS, Haider KH, Ahn WS (2003) Enhanced p53 gene transfer to human ovarian cancer cells using the cationic nonviral vector, DDC. Gynecol Oncol 90:265-272
36. Epand RM, Hughes DW, Sayer BG, Borochov N, Bach D, Wachtel E (2003) Novel properties of cholesterol-dioleoylphosphatidylcholine mixtures. Biochim Biophys Acta Biomembr 1616:196-208
37. Huang JY, Buboltz JT, Feigenson GW (1999) Maximum solubility of cholesterol in phospha-tidylcholine and phosphatidylethanolamine bilayers. Biochim Biophys Acta Biomembr 1417:89-100
38. Koltover I, Salditt T, Safinya CR (1999) Phase diagram, stability, and overcharging of lamellar cationic lipid-DNA self-assembled complexes. Biophys J 77:915-924
39. Rädler JO, Koltover I, Jamieson A, Salditt T, Safinya CR (1998) Structure and interfacial aspects of self-assembled cationic lipid-DNA gene carrier complexes. Langmuir 14:4272-4283
40. Slotte JP, Jungner M, Vilcheze C, Bittman R (1994) Effect of sterol side-chain structure on sterol-phosphatidylcholine interactions in monolayers and small unilamellar vesicles. Bio-chim Biophys Acta Biomembr 1190:435-443
41. Tristram-Nagle S, Petrache HI, Nagle JF (1998) Structure and interactions of fully hydrated dioleoylphosphatidylcholine bilayers. Biophys J 75:917-925
42. Israelachvili JN (1992) Intermolecular and surface forces, 2nd edn. Academic, London
43. Hed G, Safran SA (2004) Attractive instability of oppositely charged membranes induced by charge density fluctuations. Phys Rev Lett 93:138101(1-4)
44. Petrache HI, Harries D, Parsegian VA (2004) Alteration of lipid membrane rigidity by cholesterol and its metabolic precursors. Macromol Symp 219:39-50
45. Ewert KK, Evans HM, Zidovska A, Bouxsein NF, Ahmad A, Safinya CR (2006) Dendritic cationic lipids with highly charged headgroups for efficient gene delivery. Bioconjug Chem 17:877-888
46. Zidovska A, Evans HM, Ewert KK, Quispe J, Carragher B, Potter CS, Safinya CR (2009) Liquid crystalline phases of dendritic lipid-DNA self-assemblies: lamellar, hexagonal and DNA bundles. J Phys Chem B 113:3694-3703
47. Bosman AW, Janssen HM, Meijer EW (1999) About dendrimers: structure, physical properties, and applications. Chem Rev 99:1665-1688
48. Boger DL, Fink BE, Brunette SR, Tse WC, Hedrick MP (2001) A simple, high-resolution method for establishing DNA binding affinity and sequence selectivity. J Am Chem Soc 123:5878-5891
49. Takahashi T, Harada A, Emi N, Kono K (2005) Preparation of efficient gene carriers using a polyamidoamine dendron-bearing lipid: improvement of serum resistance. Bioconjug Chem 16:1160-1165
50. Veprek P, Jezek J (1999) Peptide and glycopeptide dendrimers. Part I. J Pept Sci 5:5-23
51. Veprek P, Jezek J (1999) Peptide and glycopeptide dendrimers. Part II. J Pept Sci 5:203-220
52. Choi JS, Lee EJ, Choi YH, Jeong YJ, Park JS (1999) Poly(ethylene glycol)-block-poly (L-lysine) dendrimer: novel linear polymer/dendrimer block copolymer forming a spherical water-soluble polyionic complex with DNA. Bioconjug Chem 10:62-65
53. Brabender-van den Berg EMMD, Meijer EW (1993) Poly(propylene imine) dendrimers- large-scale synthesis by heterogeneously catalyzed hydrogenations. Angew Chem Int Ed Engl 32:1308-1311
54. Wörner C, Mülhaupt R (1993) Polynitrile-functional and polyamine-functional poly(trimethy-lene imine) dendrimers. Angew Chem Int Ed Engl 32:1306-1308
55. Behr J-P (1989) Photohydrolysis of DNA by polyaminobenzenediazonium salt. Chem Com-mun 101:101-103
56. Schulze U, Schmidt HW, Safinya CR (1999) Synthesis of novel cationic poly(ethylene glycol) containing lipids. Bioconjug Chem 10:548-552
57. Farago O, Ewert KK, Ahmad A, Evans HM, Grønbech-Jensen N, Safinya CR (2008) Transitions between distinct compaction regimes in complexes of multivalent cationic lipids and DNA. Biophys J 95:836-846
58. Zidovska A, Ewert KK, Quispe J, Carragher B, Potter CS, Safinya CR (2009) Block liposomes from curvature-stabilizing lipids: connected nanotubes, -rods or -spheres. Langmuir 25:2979-2985
59. Vroege GJ, Lekkerkerker HNW (1992) Phase transitions in lyotropic colloidal and polymer liquid crystals. Rep Prog Phys 55:1241-1309
60. Vasilevskaya VV, Khokhlov AR, Matsuzawa Y, Yoshikawa K (1995) Collapse of single DNA molecule in poly(ethylene glycol) solutions. J Chem Phys 102:6595-6602
61. Fire A, Xu SQ, Montgomery MK, Kostas SA, Driver SE, Mello CC (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391:806-811
62. Napoli CA, Lemieux C, Jorgensen R (1990) Introduction of a chimeric chalcone synthase gene into petunia results in reversible co-suppression of homologous genes in trans. Plant Cell 2:279-289
63. Jorgensen RA, Cluster PD, English J, Que Q, Napoli CA (1996) Chalcone synthase cosup-pression phenotypes in petunia flowers: comparison of sense vs antisense constructs and single-copy vs complex T-DNA sequences. Plant Mol Biol 31:957-973
64. Cogoni C, Macino G (2000) Post-transcriptional gene silencing across kingdoms. Curr Opin Genet Dev 10:638-643
65. Hammond SM, Caudy AA, Hannon GJ (2001) Post-transcriptional gene silencing by double-stranded RNA. Nat Rev Genet 2:110-119
66. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T (2001) Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411:494-498
67. Caplen NJ, Parrish S, Imani F, Fire A, Morgan RA (2001) Specific inhibition of gene expression by small double-stranded RNAs in invertebrate and vertebrate systems. Proc Natl Acad Sci USA 98:9742-9747
68. Samuel CE (2004) Knockdown by RNAi-proceed with caution. Nat Biotechnol 22:280-282
69. McAllister CS, Samuel CE (2009) The RNA-activated protein kinase enhances the induction of interferon-beta and apoptosis mediated by cytoplasmic RNA sensors. J Biol Chem 284:1644-1651
70. McManus MT, Sharp PA (2002) Gene silencing in mammals by small interfering RNAs. Nat Rev Genet 3:737-747
71. Elbashir SM, Harborth J, Weber K, Tuschl T (2002) Analysis of gene function in somatic mammalian cells using small interfering RNAs. Methods 26:199-213
72. Martinez LA, Naguibneva I, Lehrmann H, Vervisch A, Tchenio T, Lozano G, Harel-Bellan A (2002) Synthetic small inhibiting RNAs: efficient tools to inactivate oncogenic mutations and restore p53 pathways. Proc Natl Acad Sci USA 99:14849-14854
73. Hannon GJ, Rossi JJ (2004) Unlocking the potential of the human genome with RNA interference. Nature 431:371-378
74. Karagiannis TC, El-Osta A (2005) RNA interference and potential therapeutic applications of short interfering RNAs. Cancer Gene Ther 12:787-795
75. Sioud M (2004) Therapeutic siRNAs. Trends Pharmacol Sci 25:22-28
76. Caplen NJ (2003) RNAi as a gene therapy approach. Expert Opin Biol Ther 3:575-586
77. Spagnou S, Miller AD, Keller M (2004) Lipidic carriers of siRNA: differences in the formulation, cellular uptake, and delivery with plasmid DNA. Biochemistry 43:13348-13356
78. Yin JY, Ma ZY, Selliah N, Shivers DK, Cron RQ, Finkel TH (2006) Effective gene suppression using small interfering RNA in hard-to-transfect human T cells. J Immunol Meth 312:1-11
79. Bouxsein NF, McAllister CS, Ewert KK, Samuel CE, Safinya CR (2007) Structure and gene silencing activities of monovalent and pentavalent cationic lipid vectors complexed with siRNA. Biochemistry 46:4785-4792
80. Faneca H, Simoes S, Pedroso de Lima MC (2004) Association of albumin or protamine to lipoplexes: enhancement of transfection and resistance to serum. J Gene Med 6:681-692
81. Yang J-P, Huang L (1997) Overcoming the inhibitory effect of serum on lipofection by increasing the charge ratio of cationic liposome to DNA. Gene Ther 4:950-960
82. Plank C, Mechtler K, Szoka FC, 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
83. Martin-Herranz A, Ahmad A, Evans HM, Ewert K, Schulze U, Safinya CR (2004) Surface functionalized cationic lipid-DNA complexes for gene delivery: PEGylated lamellar complexes exhibit distinct DNA-DNA interaction regimes. Biophys J 86:1160-1168
84. Hong K, Zheng W, Baker A, Papahadjopoulos D (1997) Stabilization of cationic liposome-plasmid DNA complexes by polyamines and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery. FEBS Lett 400:233-237
85. Kostarelos K, Miller AD (2005) Synthetic, self-assembly ABCD nanoparticles; a structural paradigm for viable synthetic non-viral vectors. Chem Soc Rev 34:970-994
86. Maeda H, Greish K, Fang J (2006) The EPR effect and polymeric drugs: a paradigm shift for cancer chemotherapy in the 21st century. Adv Polym Sci 193:103-121
87. Choi JS, MacKay A, Szoka FC Jr (2003) Low-pH-sensitive PEG-stabilized plasmid-lipid nanoparticles: preparation and characterization. Bioconjug Chem 14:420-429
88. Wheeler JJ, Palmer L, Ossanlou M, MacLachlan I, Graham RW, Zhang YP, Hope MJ, Scherrer P, Cullis PR (1999) Stabilized plasmid-lipid particles: construction and characterization. Gene Ther 6:271-281
89. Ruoslahti E, Duza T, Zhang L (2005) Vascular homing peptides with cell-penetrating properties. Curr Pharm Des 11:3655-3660
90. Ruoslahti E (2004) Vascular zip codes in angiogenesis and metastasis. Biochem Soc Trans 32:397-402
91. Laakkonen P, Akerman ME, Biliran H, Yang M, Ferrer F, Karpanen T, Hoffman RM, Ruoslahti E (2004) Antitumor activity of a homing peptide that targets tumor lymphatics and tumor cells. Proc Natl Acad USA 101:9381-9386
92. Walker GF, Fella C, Pelisek J, Fahrmeir J, Boeckle S, Ogris M, Wagner E (2005) Toward synthetic viruses: endosomal pH-triggered deshielding of targeted polyplexes greatly enhances gene transfer in vitro and in vivo. Mol Ther 11:418-425
93. Boomer JA, Thompson DH (1999) Synthesis of acid-labile diplasmenyl lipids for drug and gene delivery applications. Chem Phys Lipids 99:145-153
94. Zhu J, Munn RJ, Nantz MH (2000) Self-cleaving ortho ester lipids: a new class of pH-vulnerable amphiphiles. J Am Chem Soc 122:2645-2646
95. Bangham AD, Hill MW, Miller NGA (1973) Preparation and use of liposomes as models of biological membranes. Methods Membr Biol 1:1-68
96. Zidovska A, Ewert KK, Quispe J, Carragher B, Potter CS, Safinya CR (2009) The effect of salt and pH on block liposomes studied by cryogenic transmission electron microscopy. Biochim Biophys Acta Biomembr 1788:1869-1876
97. Zidovska A, Ewert KK, Quispe J, Carragher B, Potter CS, Safinya CR (2009) Block lipo-somes: vesicles of charged lipids with distinctly shaped nanoscale sphere-, pear-, tube-, or rod-segments. Meth Enzymol 465:111-128
98. Helfrich W (1973) Elastic properties of lipid bilayers - theory and possible experiments. Z Naturforsch C 28:693-703