Application of membrane-active peptides for nonviral gene delivery

Advanced Drug Delivery Reviews

Volumn 38, Issue 3, 1999, Pages 279-289

  Wagner, Ernst ^a  

^a UNIVERSITY OF VIENNA   (Austria)

Author keywords

Amphipathic peptides; Endosomal release; Polyethylenimine; Polylysine; Receptor mediated gene transfer; Transfection

Indexed keywords

AMINO ACIDS; CELL MEMBRANES; DNA; GENE TRANSFER; LIPIDS; VIRUSES;

PEPTIDES;

CONTROLLED DRUG DELIVERY;

BACTERIAL PROTEIN; CARRIER PROTEIN; DENDRIMER; DNA; GLYCEROL; GRAMICIDIN S; PEPTIDE; POLYCATION; POLYETHYLENEIMINE; POLYLYSINE; POLYMER; PROTEIN; VIRUS PROTEIN;

DRUG DELIVERY SYSTEM; GENE TARGETING; GENE TRANSFER; MEMBRANE TRANSPORT; PRIORITY JOURNAL; REVIEW;

EID: 0032764932     PISSN: 0169409X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0169-409X(99)00033-2     Document Type: Article

References (95)

1. Ledley F.D. Nonviral gene therapy: The promise of genes as pharmaceutical products. Hum. Gene Ther. 6:1995;1129-1144.
2. Perales J.C., Ferkol T., Molas M., Hanson R.W. An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur. J. Biochem. 226:1994;255-266.
3. Cotten M., Wagner E. Non-viral approaches to gene therapy. Curr. Opin. Biotechnol. 4:1993;705-710.
4. Baker A., Cotten M. Useful delivery of Bacterial Artificial Chromosomes into mammalian cells using psoralen-inactivated adenovirus carrier. Nucleic Acids Res. 25:1997;1950-1956.
5. Wolff J.A., Malone R.W., Williams P., Chong W., Acsadi G., Jani A., Felgner P.L. Direct gene transfer into mouse muscle in vivo. Science. 247:1990;1465-1468.
6. Hengge U.R., Walker P.S., Vogel J.C. Expression of naked DNA in human, pig, and mouse skin. J. Clin. Invest. 97:1996;2911-2916.
7. Hickman M.A., Malone R.W., Lehmann-Bruinsma K., Sih T.R., Knoell D., Szoka F.C., Walzem R., Carlson D.M., Powell J.S. Gene expression following direct injection of DNA into liver. Hum. Gene Ther. 5:1994;1477-1483.
8. Meyer K.B., Thompson M.M., Levy M.Y., Barron L.G., Szoka F.C. Intratracheal gene delivery to the mouse airway: characterization of plasmid DNA expression and pharmacokinetics. Gene Ther. 2:1995;450-460.
9. Vile R.G., Hart I.R. In vitro and in vivo targeting of gene expression to melanoma cells. Cancer Res. 53:1993;962-967.
10. Mumper R.J., Duguid J.G., Anwer K., Barron M.K., Nitta H., Rolland A.P. Polyvinyl derivatives as novel interactive polymers for controlled gene delivery to muscle. Pharm. Res. 13:1996;701-709.
11. Furth P.A., Shamay A., Wall R.J., Henninghausen L. Gene transfer into somatic tissues by jet injection. Anal. Biochem. 20:1992;365-368.
12. Yang N.-S., Burkholder J., Roberts B., Martinell B., McCabe D. In vivo and in vitro gene transfer to mammalian somatic cells by particle bombardment. Proc. Natl. Acad. Sci. USA. 87:1990;9568-9572.
13. Heller R., Jaroszeski M., Atkin A., Moradpour D., Gilbert R., Wands J., Nicolau C. In vivo gene electroporation and expression in rat liver. FEBS Lett. 389:1996;225-228.
14. Ulmer J.B., Donnelly J.J., Parker S.E., Rhodes G.H., Felgner P.L., Dwarki V.J., Gromkowski S.H., Deck R.R., DeWitt C.M., Friedman A., Hawe L.A., Leander K.R., Martinez D., Perry H.C., Shiver J.W., Montgomery D.L., Liu M.A. Heterologous protection against influenza by injection of DNA encoding a viral protein. Science. 259:1993;1745-1749.
15. Tang D.C., Devit M., Johnston S.A. Genetic immunization is a simple method for eliciting an immune response. Nature. 356:1992;152-154.
16. Behr J.-P. Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconj. Chem. 5:1994;382-389.
17. Felgner J.H., Kumar R., Sridhar C.N., Wheeler C.J., Tsai Y.J., Border R., Ramsey P., Martin M., Felgner P.L. Enhanced gene delivery and mechanism studies with a novel series of cationic lipid formulations. J. Biol. Chem. 269:1994;2550-2561.
18. Lee E.R., Marshall J., Siegel C.S., Jiang C., Yew N.S., Nichols M.R., Nietupski J.B., Ziegler R.J., Lane M., Wang K.X., Wan N.C., Scheule R.K., Harris D.L., Smith A.E., Cheng S.H. Hum. Gene Ther. 7:1996;1701-1717.
19. Dong Y., Skoultchi A.I., Pollard J.W. Efficient DNA transfection of quiescent mammalian cells using poly-L-ornithine. Nucleic Acids Res. 21:1993;771-772.
20. Lemnaouar M., Cajero-Juarez M., Houdebine L.-M. High efficiency of animal cell DNA transfection using poly-L-ornithine in optimized conditions. Transgenics. 1:1995;671-677.
21. Fritz J.D., Herweijer H., Zhang G., Wolff J.A. Gene transfer into mammalian cells using histone-condensed plasmid DNA. Hum. Gene Ther. 7:1996;1395-1404.
22. Böttger M., Vogel F., Platzer M., Kiessling U., Grade K., Strauss M. Condensation of vector DNA by the chromosomal protein HMG1 results in efficient transfection. Biochim. Biophys. Acta. 950:1988;221-228.
23. Cherng J.-Y., van de Wetering P., Talsma H., Crommelin D.J.A., Hennink W.E. Effect of size and serum proteins on transfection efficiency of poly((2-dimethylamino)ethyl methacrylate)-plasmid nanoparticles. Pharm. Res. 13:1996;1038-1042.
24. Haensler J., Szoka F.C. Polyamidoamine cascade polymers mediate efficient transfection of cells in culture. Bioconj. Chem. 4:1993;372-379.
25. Kukowska-Latallo J.F., Bielinska A.U., Johnson J., Spindler R., Tomalia D.A., Baker J.R. Efficient transfer of genetic material into mammalian cells using starburst polyamidoamine dendrimers. Proc. Natl. Acad. Sci. USA. 93:1996;4897-4902.
26. Boussif O., Lezoualćh F., Zanta M.A., Mergny M., Scherman D., Demeneix B., Behr J.P. A novel, versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethyleneimine. Proc. Natl. Acad. Sci. USA. 92:1995;7297-7301.
27. Wu G.Y., Wu C.H. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J. Biol. Chem. 262:1987;4429-4432.
28. Wagner E., Zenke M., Cotten M., Beug H., Birnstiel M.L. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA. 87:1990;3410-3414.
29. Huckett B., Ariatti M., Hawtrey A.O. Evidence for targeted gene transfer by receptor-mediated endocytosis: Stable expression following insulin-directed entry of neo into HepG2 cells. Biochem. Pharmacol. 40:1990;253-263.
30. Ferkol T., Perales J.C., Eckman E., Kaetzel C.S., Hanson R.W., Davis P.B. Gene transfer into the airway epithelium of animals by targeting the polymeric immunoglobulin receptor. J. Clin. Invest. 95:1995;493-502.
31. Buschle M., Cotten M., Mechtler K., Birnstiel M.L., Wagner E. Receptor-mediated gene transfer into T-lymphocytes via binding of DNA/CD3 antibody particles to the CD3 T cell receptor complex. Hum. Gene Ther. 6:1995;753-761.
32. Lee R.J., Huang L. Folate-targeted, anionic liposome-entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J. Biol. Chem. 271:1996;8481-8487.
33. White J.M. Membrane fusion. Science. 258:1992;917-924.
34. Legendre J.Y., Szoka F.C. Delivery of plasmid DNA into mammalian cell lines using pH-sensitive liposomes: comparison with cationic liposomes. Pharm. Res. 9:1992;1235-1242.
35. Zhou X., Huang L. DNA transfection mediated by cationic liposomes containing lipopolylysine: characterization and mechanism of action. Biochim. Biophys. Acta. 1189:1994;195-203.
36. Behr J.-P. Synthetic gene transfer vectors. Acc. Chem. Res. 26:1993;274-278.
37. Wrobel I., Collins D. Fusion of cationic liposomes with mammalian cells occurs after endocytosis. Biochim. Biophys. Acta. 1235:1995;296-304.
38. Zabner J., Fasbender A.J., Moninger T., Poellinger K.A., Welsh M.J. Cellular and molecular barriers to gene transfer by a cationic lipid. J. Biol. Chem. 270:1995;18997-19007.
39. Zatloukal K., Wagner E., Cotten M., Phillips S., Plank C., Steinlein P., Curiel D., Birnstiel M.L. Transferrinfection: A highly efficient way to express gene constructs in eukaryotic cells. Ann. New York Acad. Sci. 660:1992;136-153.
40. Reston J.T., Gould-Fogerite S., Mannino R.J. Aspects of cellular physiology that influence DNA-mediated gene transfer in NIH3T3 cells. Mol. Cell. Biochem. 145:1995;169-175.
41. Chowdhury N.R., Wu C.H., Wu G.Y., Yerneni P.C., Bommineni V.R., Chowdhury J.R. Fate of DNA targeted to the liver by asialoglycoprotein receptor-mediated endocytosis in vivo. J. Biol. Chem. 268:1993;11265-11271.
42. Chowdhury N.R., Hays R.M., Bommineni V.R., Franki N., Chowdhury J.R., Wu C.H., Wu G.Y. Microtubular disruption prolongs the expression of human bilirubin-uridinediphosphoglucuronate-glucuronsyltransferase-1 gene transferred into Gunn rat livers. J. Biol. Chem. 271:1996;2341-2346.
43. Giulotto E., Israel N. DNA-mediated gene transfer is more efficient during S-phase of the cell cycle. Biochem. Biophys. Res. Commun. 118:1984;310-316.
44. Goldstein S., Fordis C.M., Howard B.H. Enhanced transfection efficiency and improved cell survival after electroporation of G2/M-synchronized cells. Nucleic Acids Res. 17:1989;3959-3971.
45. Wilke M., Fortunati E., van den Broek M., Hoogeveen A.T., Scholte B.J. Efficacy of a peptide-based gene delivery system depends on mitotic activity. Gene Ther. 3:1996;1133-1142.
46. Orrantia E., Chang P.L. Intracellular distribution of DNA internalized through calcium phosphate precipitation. Exp. Cell Res. 190:1990;170-174.
47. Benachir T., Lafleur M. Study of vesicle leakage induced by melittin. Biochim. Biophys. Acta. 1235:1995;452-460.
48. Epand R.M., Cheetham J.J., Epand R.F., Yeagle P.L., Richardson C.D., Rockwell A., DeGrado W.F. Peptide models for the membrane destabilizing action of viral fusion proteins. Biopolymers. 32:1992;309-314.
49. Epand R.M., Shai Y., Segrest J.P., Anantharamaiah G.M. Mechanisms for the modulation of membrane bilayer properties by amphipathic helical peptides. Biopolymers. 17:1995;319-338.
50. Fujii G., Selsted M.E., Eisenberg D. Defensins promote fusion and lysis of negatively charged membranes. Protein Sci. 2:1993;1301-1312.
51. Gazit E., Boman A., Boman H.G., Shai Y. Interaction of the mammalian antibacterial peptide cecropin P1 with phospholipid vesicles. Biochemistry. 34:1995;11479-11488.
52. Shai Y. Pardaxin: channel formation by a shark repellant peptide from fish. Toxicology. 87:1994;109-129.
53. Lüneberg J., Martin I., Nüßler F., Ruysschaert J.-M., Herrmann A. Structure and topology of the influenza virus fusion peptide in lipid bilayers. J. Biol. Chem. 270:1995;27606-27614.
54. Rafalski M., Ortiz A., Rockwell A., van Gickel L.C., Lear J.D., DeGrado W.F., Wilschut J. Membrane fusion activity of the influenza virus hemagglutinin: interaction of HA2 N-terminal peptides with phospholipid vesicles. Biochemistry. 30:1991;10211-10220.
55. Takahashi S. Conformation of membrane fusion active 20-residue peptides with or without lipid bilayers. Implication of alpha-helix formation for membrane fusion. Biochemistry. 29:1990;6257-6264.
56. Plank C., Zauner W., Wagner E. Application of membrane-active peptides for drug and gene delivery across cellular membranes. Adv. Drug Del. Rev. 34:1998;21-35.
57. Cotten M., Laengle-Rouault F., Kirlappos H., Wagner E., Mechtler K., Zenke M., Beug H., Birnstiel M.L. Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc. Natl. Acad. Sci. USA. 87:1990;4033-4037.
58. Zauner W., Kichler A., Schmidt W., Sinski A., Wagner E. Glycerol enhancement of ligand-polylysine/DNA transfection. Biotechniques. 20:1996;905-913.
59. Cotten M., Wagner E., Zatloukal K., Phillips S., Curiel D.T., Birnstiel M.L. High-efficiency receptor-mediated delivery of small and large (48kb) gene constructs using the endosome disruption activity of defective or chemically inactivated adenovirus particles. Proc. Natl. Acad. Sci. USA. 89:1992;6094-6098.
60. Zauner W., Blaas D., Küchler E., Wagner E. Rhinovirus mediated endosomal release of transfection complexes. J. Virol. 69:1995;1085-1092.
61. Curiel D.T., Wagner E., Cotten M., Birnstiel M.L., Agarwal S., Li C.-M., Loechel S., Hu P.-C. High-efficiency gene transfer by adenovirus coupled to DNA-polylysine complexes. Hum. Gene Ther. 3:1992;147-154.
62. Wagner E., Zatloukal K., Cotten M., Kirlappos H., Mechtler K., Curiel D.T., Birnstiel M.L. Coupling of adenovirus to transferrin-polylysine/DNA complexes greatly enhances receptor-mediated gene delivery and expression of transfected genes. Proc. Natl. Acad. Sci. USA. 89:1992;6099-6103.
63. Wu G.Y., Zhan P., Sze L.L., Rosenberg A.R., Wu C.H. Incorporation of adenovirus into a ligand-based DNA carrier system results in retention of original receptor specificity and enhances targeted gene expression. J. Biol. Chem. 269:1994;11542-11546.
64. Cotten M., Saltik M., Kursa M., Wagner E., Maass G., Birnstiel M.L. Psoralen treatment of adenovirus particles eliminates virus replication and transcription while maintaining the endosomolytic activity of the virus capsid. Virology. 205:1994;254-261.
65. Gao L., Wagner E., Cotten M., Agarwal S., Harris C., Romer M., Miller L., Hu P.-C., Curiel D. Direct in vivo gene transfer to the airway epithelium employing adenovirus-polylysine-DNA complexes. Hum. Gene Ther. 4:1993;17-24.
66. Nguyen D.M., Wiehle S.A., Roth J.A., Cristiano R.J. Gene delivery into malignant cells in vivo by a conjugated adenovirus/DNA complex. Cancer Gene Ther. 4:1997;183-190.
67. Plank C., Oberhauser B., Mechtler K., Koch C., Wagner E. The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene transfer systems. J. Biol. Chem. 269:1994;12918-12924.
68. Wagner E., Plank C., Zatloukal K., Cotten M., Birnstiel M.L. Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine/DNA complexes: Towards a synthetic virus-like gene transfer vehicle. Proc. Natl. Acad. Sci. USA. 89:1992;7934-7938.
69. Midoux P., Mendes C., Legrand A., Raimond J., Mayer R., Monsigny M., Roche A.C. Specific gene transfer mediated by lactosylated poly-L-lysine into hepatoma cells. Nucleic Acids Res. 21:1993;871-878.
70. Plank C., Zatloukal K., Cotten M., Mechtler K., Wagner E. Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconj. Chem. 3:1992;533-539.
71. Mechtler K., Wagner E. Influenza peptide enhanced gene transfer: The role of peptide sequences. New J. Chem. 21:1997;105-111.
72. Parente R.A., Nir S., Szoka F.C. Mechanism of leakage of phospholipid vesicle contents induced by the peptide GALA. Biochemistry. 29:1990;8720-8728.
73. Gottschalk S., Tweten R.K., Smith L.C., Woo S.L.C. Efficient gene delivery and expression in mammalian cells using DNA coupled with perfringolysin O. Gene Ther. 2:1995;498-503.
74. Fisher K.J., Wilson J.M. The transmembrane domain of diphtheria toxin improves molecular conjugate gene transfer. Biochem. J. 321:1997;49-58.
75. Fender P., Ruigrok R.W.H., Gout E., Buffet S., Chroboczek J. Adenovirus dodecahedron, a new vector for human gene transfer. Nat. Biotechnol. 15:1997;52-56.
76. Gottschalk S., Sparrow J.T., Hauer J., Mims M.P., Leland F.E., Woo S.L.C., Smith L.C. A novel DNA-peptide complex for efficient gene transfer and expression in mammalian cells. Gene Ther. 3:1996;448-457.
77. Wyman T.B., Nicol F., Zelphati O., Scaria P.V., Plank C., Szoka F.C. Design, synthesis and characterization of a cationic peptide that binds to nucleic acids and permeabilizes bilayers. Biochemistry. 36:1997;3008-3017.
78. Kircheis R., Kichler A., Wallner G., Kursa M., Ogris M., Felzmann T., Buchberger M., Wagner E. Coupling of cell-binding ligands to polyethylenimine for targeted delivery. Gene Ther. 4:1997;409-418.
79. Behr J.-P. Gene transfer with synthetic cationic amphiphiles: prospects for gene therapy. Bioconj. Chem. 5:1994;382-389.
80. Gao X., Huang L. Potentiation of cationic liposome-mediated gene delivery by polycations. Biochemistry. 35:1996;1027-1036.
81. Sternberg B., Sorgi F.L., Huang L. New structures in complex formation between DNA and cationic liposomes visualized by freeze-fracture electron microscopy. FEBS Lett. 356:1994;361-366.
82. Xu Y., Szoka F.C. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry. 35:1996;5616-5623.
83. Leventis R., Silvius J.R. Interactions of mammalian cells with lipid dispersions containing novel metabolizable cationic amphiphiles. Biochim. Biophys. Acta. 1023:1990;124-132.
84. Schwartz B., Benoist C., Abdallah B., Scherman D., Behr J.-P., Demeneix B.A. Lipospermine-based gene transfer into newborn mouse brain is optimized by a low lipospermine/DNA charge ratio. Hum. Gene Ther. 6:1995;1515-1524.
85. II) transitions in phosphatidylethanolamine liposomes. Biochemistry. 29:1990;2976-2985.
86. Farhood H., Serbina N., Huang L. The role of dioleoyl phosphatidylethanolamine in cationic liposome mediated gene transfer. Biochim. Biophys. Acta. 1235:1995;289-295.
87. Kaneda Y., Iwai K., Uchida T. Increased expression of DNA cointroduced with nuclear protein in adult rat liver. Science. 243:1989;375-378.
88. von der Leyen H.E., Gibbons G.H., Morishita R., Lewis N.P., Zhang L., Nakajima M., Kaneda Y., Cooke J.P., Dzau V.J. Gene therapy inhibiting neointimal vascular lesion: in vivo transfer of endothelial cell nitric oxide synthase gene. Proc. Natl. Acad. Sci. USA. 92:1995;1137-1141.
89. Mizuguchi H., Nakagawa T., Nakanishi M., Imazu S., Nakagawa S., Mayumi T. Efficient gene transfer into mammalian cells using fusogenic liposomes. Biochem. Biophys. Res. Commun. 218:1996;402-407.
90. Tikchonenko T.I., Glushakova S.E., Kislina O.S., Grodnitskaya N.A., Manykin A.A., Naroditsky B.S. Transfer of condensed viral DNA into eukaryotic cells using proteoliposomes. Gene. 63:1988;321-330.
91. Legendre J.Y., Szoka F.C. Cyclic amphipathic peptide-DNA complexes mediate high-efficiency transfection of adherent cells. Proc. Natl. Acad. Sci. USA. 90:1993;893.
92. Legendre J.Y., Supersaxo A. Short-chain phospholipids enhance amphipathic peptide-mediated gene transfer. Biochem. Biophys. Res. Commun. 217:1995;179-185.
93. Hara T., Kuwasawa H., Aramaki Y., Takada S., Koike K., Ishidate K., Kato H., Tsuchiya S. Effects of fusogenic and DNA-binding amphiphilic compounds on the receptor-mediated gene transfer into hepatic cells by asialofetuin-labeled liposomes. Biochim. Biophys. Acta. 1278:1996;51-58.
94. Kichler A., Mechtler K., Behr J.-P., Wagner E. The influence of membrane-active peptides and helper lipids on lipospermine/DNA complex mediated gene transfer. Bioconj. Chem. 8:1997;213-221.
95. Kamata H., Yagisawa H., Takahashi S., Hirata H. Amphiphilic peptides enhance the efficiency of liposome-mediated DNA transfection. Nucleic Acids Res. 22:1994;536-537.