Structure and function of lipid-DNA complexes for gene delivery

Annual Review of Biophysics and Biomolecular Structure

Volumn 29, Issue , 2000, Pages 27-47

  Chesnoy, S ^a   Huang, L ^a  

^a UNIVERSITY OF PITTSBURGH SCHOOL OF MEDICINE   (United States)

Author keywords

Cationic lipid; Plasmid DNA; Toxicity; Transfection; X ray diffraction

Indexed keywords

CLONING VECTOR; COMPLEX FORMATION; DNA BINDING; DNA TRANSPOSITION; GENE THERAPY; HYDROPHOBICITY; PRIORITY JOURNAL; REVIEW; STRUCTURE ACTIVITY RELATION; X RAY DIFFRACTION;

ANIMALS; DNA; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LIPID METABOLISM; LIPIDS; LIPOSOMES; MICROSCOPY, ELECTRON; MODELS, BIOLOGICAL; PLASMIDS; STRUCTURE-ACTIVITY RELATIONSHIP; TRANSFECTION; X-RAY DIFFRACTION;

EID: 0033902946     PISSN: 10568700     EISSN: None     Source Type: Book Series    
DOI: 10.1146/annurev.biophys.29.1.27     Document Type: Review

References (67)

1. 1. Balasubramaniam RP, Bennett MJ, Aberle AM, Malone JG, Nantz MH, et al. 1996. Structural and functional analysis of cationic transfection lipids: the hydrophobic domain. Gene Ther. 3:163
2. 2. Behr JP, Demeneix B, Loeffler JP, Mutul JP. 1989. Efficient gene transfer into mammalian primary endocrine cells with lipopolyamine-coated DNA. Proc. Natl. Acad. Sci. USA 86:6982
3. 3. Blessing T, Remy JS, Behr JP. 1998. Monomolecular collapse of plasmid DNA into stable virus-like particles. Proc. Natl. Acad. Sci. USA 95:1427
4. 4. Boukhnikachvili T, Aguerre-Chariol O, Airiau M, Lesieur S, Ollivon M, et al. 1997. Structure of in-serum transfecting DNA-cationic lipid complexes. FEBS Lett. 409:188
5. 5. Brisson M, He Y, Li S, Yang JP, Huang L. 1999. A novel T7 RNA polymerase autogene for efficient cytoplasmic expression of target genes. Gene Ther. 6:263
6. 6. Chen X, Li Y, Xiong K, Kie Y, Aizicovici S, et al. 1995. A nonviral cytoplasmic gene expression system and its implications in cancer gene therapy. Cancer Gene Ther. 2: 281
7. 7. Crook K, Stevenson BJ, Dubouchet M, Porteous 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
8. 8. Dowty ME, Williams P, Zhang G, Hagstrom J, Wolff JA. 1995. Plasmid DNA entry into postmitotic nuclei of primary rat myotubes. Proc. Natl. Acad. Sci. USA 92:4572
9. 9. El Ouahabi A, Thiry M, Pector V, Fuks R, Ruysschaert JM, et al. 1997. The role of endosome destabilizing activity in the gene transfer process mediated by cationic lipids. FEBS Lett. 414:187
10. 10. Farhood H, Serbina N, Huang L. 1995. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta 1235:289
11. 11. Fasbender A, Zabner J, Zeiher BG, Welsh MJ. 1997. A low rate of cell proliferation and reduced DNA uptake limit cationic lipid-mediated gene transfer to primary cultures of ciliated human airway epithelia. Gene Ther. 4:1173
12. 12. Felgner JH, Kumar R, Sridhar CN, Wheeler CJ, Tsai YJ, et al. 1994. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269:2550
13. 13. Friend DS, Papahadjopoulos D, Debs RJ. 1996. Endocytosis and intracellular processing accompanying transfection mediated by cationic liposomes. Biochim. Biophys. Acta 1278:41
14. 14. Gao X, Huang L. 1993. Cytoplasmic expression of a reporter gene by co-delivery of T7 RNA polymerase and T7 promoter sequence with cationic liposomes. Nucleic Acids Res. 68:2867
15. 15. Gao X, Jaffurs D, Robbins PD, Huang L. 1994. A sustained, cytoplasmic transgene expression system delivered by cationic liposomes. Biochem. Biophys. Res. Commun. 200:1201
16. 16. Gustafsson J, Arvidson G, Karlsson G, Almgren M. 1995. Complexes between cationic liposomes and DNA visualized by cryo-TEM. Biochim. Biophys. Acta 1235:305
17. 17. Hara T, Tan Y, Huang L. 1997. In vivo gene delivery to the liver using reconstituted chylomicron remnants as a novel nonviral vector. Proc. Natl. Acad. Sci. USA 94:14547
18. 18. Hisayasu S, Miyauchi M, Akiyama K, Gotoh T, Satoh S, et al. 1999. In vivo targeted gene transfer into liver cells mediated by a novel galactosyl-D-lysine/ D-serine copolymer. Gene Ther. 6:689
19. 19. Hofland HEJ, Shepard L, Sullivan SM. 1996. Formation of stable cationic lipid/DNA complexes for gene transfer. Proc. Natl. Acad. Sci. USA 93:7305
20. 20. Hong K, Zheng W, Baker A, Papahadjopoulos D. 1997. Stabilization of cationic liposome-plasmid DNA complexes by polyandries and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery. FEBS Lett. 400:233
21. 21. Hui SW, Langner M, Zhao YL, Ross P, Hurley , et al. 1996. The role of helper lipids in cationic liposome-mediated gene transfer. Biophys. J. 71:590
22. 22. Koltover I, Salditt T, Radler JO, Safinya CR. 1998. An inverted hexagonal phase of cationic liposome-DNA complexes related to DNA release and delivery. Science 281:78
23. 23. Krieg AM. 1999. Direct immunologic activities of CpG DNA and implications for gene therapy. J. Gene Med. 1:56
24. 24. Labat-Moleur F, Steffan AM, Brisson C, Perron H, Feugeas O, et al. 1996. An electron microscopy study into the mechanism of gene transfer with lipopolyamines. Gene Ther. 3:1010
25. 25. Lasic DD, Strey H, Stuart MCA, Podgornik R, Frederik PM. 1997. The structure of DNA-liposome complexes. J. Am. Chem. Soc. 119:832
26. 26. Lechardeur D, Sohn KJ, Haardt M, Joshi PB, Monck M, et al. 1999. Metabolic instability of plasmid DNA in the cytosol: a potential barrier to gene transfer. Gene Ther. 6:482
27. 27. Legendre JY, Szoka FC Jr. 1992. Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: comparison with cationic liposomes. Pharm. Res. 9(10):1235
28. 28. Li S, Rizzo MA, Bhattacharya S, Huang L. 1998. Characterization of cationic lipid-protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Ther. 5:930
29. 29. Li S, Tseng WC, Beer Stolz D, Wu SP, Watkins SC, et al. 1999. Dynamic changes in the characteristics of cationic lipidic vectors after exposure to mouse serum: implications for intravenous lipofection. Gene Ther. 6:585
30. 30. Li S, Wu SP, Whitmore M, Loeffert EJ, Wang L, et al. 1999. Effect of immune response on gene transfer to the lung via systemic administration of cationic lipidic vectors, Am. J. Physiol. 276:L796
31. 31. Liu F, Qi H, Huang L, Liu D. 1997. Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration. Gene Ther. 4: 517
32. 32. Liu Y, Mounkes LC, Liggitt HD, Brown CS, Solodin L, et al. 1997. Factors influencing the efficiency of cationic liposome-mediated intravenous gene delivery. Nat. Biotechnol. 15:167
33. 33. Mahato RI, Anwer K, Tagliaferri F, Meaney C, Leonard P, et al. 1998. Biodistribution and gene expression of lipid/plasmid complexes after systemic administration. Hum. Gene Ther. 9:2083
34. 34. Mortimer I, Tam P, MacLachlan I, Graham RW, Saravolac EG, et al. 1999. Cationic lipid-mediated transfection of cells in culture requires mitotic activity. Gene Ther. 6:403
35. 35. Mounkes LC, Zhong W, Cipres-Palacin G, Heath TD, Debs RJ. 1998. Proteoglycans mediate cationic liposome-DNA complex-based gene delivery in vitro and in vivo. J. Biol. Chem. 40:26164
36. 36. Ogris M, Brunner S, Schuller S, Kircheis R, 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
37. 37. Pinnaduwage P, Schmitt L, Huang L. 1989. Use of quaternary ammonium detergent in liposome mediated DNA transfection of mouse L-cells. Biochim. Biophys. Acta 985:33
38. 38. Pitard B, Aguerre O, Airiau M, Lachages AM, Boukhnikachvili T, et al. 1997. Virus-sized self-assembling lamellar complexes between plasmid DNA and cationic micelles promote gene transfer. Proc. Natl. Acad. Sci. USA 94:14412
39. 39. Pitard B, Oudrhiri N, Vigneron JP, Hauchecorne M, Aguerre O. 1999. Structural characteristics of supramolecular assemblies formed by guanidinium-cholesterol reagents for gene transfection. Proc. Natl. Acad. Sci. USA 96:2621
40. 40. Qin L, Ding Y, Pahud DR, Chang E, Imperiale MJ, et al. 1997. Promoter attenuation in gene therapy: interferon-gamma and tumor necrosis factor-alpha inhibit transgene expression. Hum. Gene Ther. 8:2019
41. 41. Radler 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
42. 42. Remy JS, Sirlin C, Vierling P, Behr JP. 1994. Gene transfer with a series of lipophilic DNA-binding molecules. Bioconjugate Chem. 5:647
43. 43. Remy JS, Kichler A, Mordvinov V, Schuber F, Behr JP. 1995. Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial viruses. Proc. Natl. Acad. Sci. USA 92:1744
44. 44. Ross PC, Hui SW. 1999. Lipoplex size is a major determinant of in vitro lipofection efficiency. Gene Ther. 6:651
45. 45. Salditt T, Koltover I, Radler JO, Safinya CR. Two-dimensional smectic ordering of linear DNA chains in self-assembled DNA-cationic liposome mixtures. Phys. Rev. Lett. 79:2582
46. 46. Smyth Templeton N, Lasid DD, Frederik PM, Strey HH, Roberts DD, et al. 1997. Improved DNA:liposome complexes for increased systemic delivery and gene expression. Nat. Biotechnol. 15:647
47. 47. Solodin I, Brown CS, Bruno MS, Chow CY, Jang EH, et al. 1995. A novel series of amphiphilic imidazolinium compounds for in vitro and in vivo gene delivery. Biochemistry 34:13537
48. 48. Song Y, Liu F, Chu SY, Liu DX. 1997. Characterization of cationic liposome-mediated gene transfer in vivo by intravenous administration. Hum. Gene Ther. 8:1585
49. 49. Song YK, Liu F, Liu D. 1998. Enhanced gene expression in mouse lung by prolonging the retention time of intravenously injected plasmid DNA. Gene Ther. 5:1531
50. 50. Sternberg B, Sorgi FL, Huang L. 1994. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy. FEBS Lett. 356:361
51. 51. Tan Y, Li S, Pitt BR, Huang L. 1999. The inhibitory role of CpG immunostimulatory motifs in cationic lipid vector-mediated transgene expression in vivo. Gene Ther. In press
52. 52. Thierry AR, Rabinovich P, Peng B, Mahan LC, Bryant JL, et al. 1997. Characterization of liposome-mediated gene delivery: expression, stability and pharmacokinetics of plasmid DNA. Gene Ther. 4:226
53. 53. Van der Woude I, Willy Visser H, Ter Beest MBA, Wagenaar A, Ruiters MHJ, et al. 1995. Parameters influencing the introduction of plasmid DNA into cells by the use of synthetic amphiphiles as a carrier system. Biochim. Biophys. Acta 1240:34
54. 54. Vigneron JP, Oudrhiri N, Fauquet M, Vergely L, Bradley JC, et al. 1996. Guanidinium-cholesterol cationic lipids: efficient vectors for the transfection of eukaryotic cells. Proc. Natl. Acad. Sci. USA 93:9682
55. 55. Wattiaux R, Jadot M, Warnier-Pirotte MT, Wattiaux-De Coninck S. 1997. Cationic lipids destabilize lysosomal membrane in vitro. FEBS Lett. 417:199
56. 56. Wheeler CJ, Fegner PL, Tsai YJ, Marshall J, Sukhu L, et al. 1996. A novel cationic lipid greatly enhances plasmid DNA delivery and expression in mouse lung. Proc. Natl. Acad. Sci. USA 93:11454
57. 57. Wheeler JJ, Palmer L, Ossanlou M, MacLachlan I, Graham RW, et al. 1999. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther. 6:271
58. 58. Wrobel I, Collins D. 1995. Fusion of cationic liposomes with mammalian cells occurs after endocytosis. Biochim. Biophys. Acta 1235:296
59. 59. Xu Y, Szoka FC. 1996. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 35:5616
60. 60. Yang JP, Huang L. 1998. Time-dependent maturation of cationic liposome-DNA complex for serum resistance. Gene Ther. 5:380
61. 61. Yang JP, 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
62. 62. Yew NS, Wang KX, Przybylska M, Bagley RG, Stedman M. 1999. Contribution of plasmid DNA to inflammation in the lung after administration of cationic lipid:DNA complexes. Hum. Gene Ther. 10:223
63. 63. Yoshikawa Y, Emi N, Kanbe T, Yoshikawa K, Saito H. 1996. Folding and aggregation of DNA chains induced by complexation with lipospermine: formation of a nucleosome-like structure and network assembly. FEBS Lett. 396:71
64. 64. Zabner J, Fasbender AJ, Moninger T, Poellinger KA, Welsh MJ. 1995. Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270:18997
65. 65. Zanta MA, Belguise-Valladier P, Behr JP. 1999. Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl. Acad. Sci. USA 96:91
66. 66. Zhou X, Huang L. 1994. DNA transfection mediated by cationic liposomes containing lipopolylysine: characterization and mechanism of action. Biochim. Biophys. Acta 1189:195
67. 67. Zhu N, Liggitt D, Liu Y, Debs R. 1993. Systemic gene expression after intravenous delivery DNA delivery into adult mice. Science 261:209