Liposome-polyethylenimine complexes for enhanced DNA and siRNA delivery

Biomaterials

Volumn 31, Issue 26, 2010, Pages 6892-6900

  Schäfer, Jens ^a   Höbel, Sabrina ^a   Bakowsky, Udo ^a   Aigner, Achim ^a  

^a PHILIPPS UNIVERSITY MARBURG   (Germany)

Author keywords

Gene delivery; Lipid coating; Liposomes; Polyethylenimine; RNAi; SiRNAs

Indexed keywords

BIOLOGICAL PROPERTIES; CELLULAR UPTAKE; DNA TRANSFECTION; ENDOSOMAL RELEASE; GENE DELIVERY; GENE KNOCK DOWN; GENE TRANSFECTION; LIPID COATING; LIPID SYSTEMS; LIPIDATION; LOW MOLECULAR WEIGHT; MECHANISM AND KINETICS; NONVIRAL VECTORS; PHYSICOCHEMICAL PROPERTY; POLYETHYLENIMINES; POLYPLEXES; POSITIVELY CHARGED; RNA INTERFERENCE; SIRNA DELIVERY; SMALL INTERFERING RNA; TRANSFECTION REAGENTS;

COATINGS; DNA; GENE TRANSFER; LIPOSOMES; NUCLEIC ACIDS; PHOSPHOLIPIDS; RNA;

GENE THERAPY;

DNA; DRUG VEHICLE; LIPID; LIPOSOME; NUCLEIC ACID; POLYETHYLENEIMINE; SMALL INTERFERING RNA;

ARTICLE; CELL TRANSPORT; CHEMICAL ANALYSIS; COMPLEX FORMATION; CONTROLLED STUDY; CYTOTOXICITY; DNA TRANSFECTION; DRUG COATING; DRUG FORMULATION; GENE DELIVERY SYSTEM; HUMAN; HUMAN CELL; KINETICS; LIPID COMPOSITION; MOLECULAR WEIGHT; PARTICLE SIZE; PHYSICOCHEMICAL MODEL; PRIORITY JOURNAL; ZETA POTENTIAL; ATOMIC FORCE MICROSCOPY; CELL DEATH; DRUG EFFECTS; ENDOCYTOSIS; GENETIC TRANSFECTION; METABOLISM; PROCEDURES; TIME; TOXICITY; TUMOR CELL LINE;

CELL DEATH; CELL LINE, TUMOR; DNA; ENDOCYTOSIS; HUMANS; LIPOSOMES; MICROSCOPY, ATOMIC FORCE; MOLECULAR WEIGHT; PARTICLE SIZE; POLYETHYLENEIMINE; RNA, SMALL INTERFERING; TIME FACTORS; TRANSFECTION;

EID: 77954386915     PISSN: 01429612     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2010.05.043     Document Type: Article

References (59)

1. Verma I.M., Somia N. Gene therapy - promises, problems and prospects. Nature 1997, 389(6648):239-242.
2. Itaka K., Kataoka K. Recent development of nonviral gene delivery systems with virus-like structures and mechanisms. Eur J Pharm Biopharm 2009, 71(3):475-483.
3. Kodama K., Katayama Y., Shoji Y., Nakashima H. The features and shortcomings for gene delivery of current non-viral carriers. Curr Med Chem 2006, 13(18):2155-2161.
4. Ravi Kumar M.N., Sameti M., Mohapatra S.S., Kong X., Lockey R.F., Bakowsky U., et al. Cationic silica nanoparticles as gene carriers: synthesis, characterization and transfection efficiency in vitro and in vivo. J Nanosci Nanotechnol 2004, 4(7):876-881.
5. Kumar M.N., Mohapatra S.S., Kong X., Jena P.K., Bakowsky U., Lehr C.M. Cationic poly(lactide-co-glycolide) nanoparticles as efficient in vivo gene transfection agents. J Nanosci Nanotechnol 2004, 4(8):990-994.
6. Fire A., Xu S., Montgomery M.K., Kostas S.A., Driver S.E., Mello C.C. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998, 391(6669):806-811.
7. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411(6836):494-498.
8. Elbashir S.M., Lendeckel W., Tuschl T. RNA interference is mediated by 21- and 22-nucleotide RNAs. Genes Dev 2001, 15(2):188-200.
9. Hamilton A.J., Baulcombe D.C. A species of small antisense RNA in posttranscriptional gene silencing in plants. Science 1999, 286(5441):950-952.
10. Aigner A. Cellular delivery in vivo of siRNA-based therapeutics. Curr Pharm Des 2008, 14(34):3603-3619.
11. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci U S A 1995, 92(16):7297-7301.
12. Neu M., Fischer D., Kissel T. Recent advances in rational gene transfer vector design based on poly(ethylene imine) and its derivatives. J Gene Med 2005, 7(8):992-1009.
13. Godbey W.T., Wu K.K., Mikos A.G. Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle. J Biomed Mater Res 1999, 45(3):268-275.
14. Tang M.X., Szoka F.C. The influence of polymer structure on the interactions of cationic polymers with DNA and morphology of the resulting complexes. Gene Ther 1997, 4(8):823-832.
15. Urban-Klein B., Werth S., Abuharbeid S., Czubayko F., Aigner A. RNAi-mediated gene-targeting through systemic application of polyethylenimine (PEI)-complexed siRNA in vivo. Gene Ther 2005, 12(5):461-466.
16. Grzelinski M., Urban-Klein B., Martens T., Lamszus K., Bakowsky U., Hobel S., et al. RNA interference-mediated gene silencing of pleiotrophin through polyethylenimine-complexed small interfering RNAs in vivo exerts antitumoral effects in glioblastoma xenografts. Hum Gene Ther 2006, 17(7):751-766.
17. Werth S., Urban-Klein B., Dai L., Hobel S., Grzelinski M., Bakowsky U., et al. A low molecular weight fraction of polyethylenimine (PEI) displays increased transfection efficiency of DNA and siRNA in fresh or lyophilized complexes. J Control Release 2006, 112(2):257-270.
18. Zuber G., Dauty E., Nothisen M., Belguise P., Behr J.P. Towards synthetic viruses. Adv Drug Deliv Rev 2001, 52(3):245-253.
19. Behr J.P. The proton sponge: a trick to enter cells the viruses did not exploit. Chimia 1997, 51:34-36.
20. Godbey W.T., Wu K.K., Mikos A.G. Tracking the intracellular path of poly(ethylenimine)/DNA complexes for gene delivery. Proc Natl Acad Sci U S A 1999, 96(9):5177-5181.
21. Pollard H., Remy J.S., Loussouarn G., Demolombe S., Behr J.P., Escande D. Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus in mammalian cells. J Biol Chem 1998, 273(13):7507-7511.
22. Hobel S., Prinz R., Malek A., Urban-Klein B., Sitterberg J., Bakowsky U., et al. Polyethylenimine PEI F25-LMW allows the long-term storage of frozen complexes as fully active reagents in siRNA-mediated gene targeting and DNA delivery. Eur J Pharm Biopharm 2008, 70(1):29-41.
23. Hobel S., Koburger I., John M., Czubayko F., Hadwiger P., Vornlocher H.P., et al. Polyethylenimine/small interfering RNA-mediated knockdown of vascular endothelial growth factor in vivo exerts anti-tumor effects synergistically with Bevacizumab. J Gene Med 2010, 12(3):287-300.
24. Kichler A. Gene transfer with modified polyethylenimines. J Gene Med 2004, 6(Suppl. 1):S3-S10.
25. Ogris M., Wagner E. Tumor-targeted gene transfer with DNA polyplexes. Somat Cell Mol Genet 2002, 27(1-6):85-95.
26. Malek A., Czubayko F., Aigner A. PEG grafting of polyethylenimine (PEI) exerts different effects on DNA transfection and siRNA-induced gene targeting efficacy. J Drug Target 2008, 16(2):124-139.
27. Nguyen H.K., Lemieux P., Vinogradov S.V., Gebhart C.L., Guerin N., Paradis G., et al. Evaluation of polyether-polyethyleneimine graft copolymers as gene transfer agents. Gene Ther 2000, 7(2):126-138.
28. Erbacher P., Bettinger T., Belguise-Valladier P., Zou S., Coll J.L., Behr J.P., et al. Transfection and physical properties of various saccharide, poly(ethylene glycol), and antibody-derivatized polyethylenimines (PEI). J Gene Med 1999, 1(3):210-222.
29. Fella C., Walker G.F., Ogris M., Wagner E. Amine-reactive pyridylhydrazone-based PEG reagents for pH-reversible PEI polyplex shielding. Eur J Pharm Sci 2008, 34(4-5):309-320.
30. Nimesh S., Chandra R. Polyethylenimine nanoparticles as an efficient in vitro siRNA delivery system. Eur J Pharm Biopharm 2009, 73(1):43-49.
31. Zintchenko A., Philipp A., Dehshahri A., Wagner E. Simple modifications of branched PEI lead to highly efficient siRNA carriers with low toxicity. Bioconjug Chem 2008, 19(7):1448-1455.
32. Thomas M., Klibanov A.M. Enhancing polyethylenimine's delivery of plasmid DNA into mammalian cells. Proc Natl Acad Sci U S A 2002, 99(23):14640-14645.
33. Gusachenko Simonova O., Kravchuk Y., Konevets D., Silnikov V., Vlassov V.V., Zenkova M.A. Transfection efficiency of 25-kDa PEI-cholesterol conjugates with different levels of modification. J Biomater Sci Polym Ed 2009, 20(7-8):1091-1110.
34. Masotti A., Moretti F., Mancini F., Russo G., Di Lauro N., Checchia P., et al. Physicochemical and biological study of selected hydrophobic polyethylenimine-based polycationic liposomes and their complexes with DNA. Bioorg Med Chem 2007, 15(3):1504-1515.
35. Liu C., Zhao G., Liu J., Ma N., Chivukula P., Perelman L., et al. Novel biodegradable lipid nano complex for siRNA delivery significantly improving the chemosensitivity of human colon cancer stem cells to paclitaxel. J Control Release 2009, 140(3):277-283.
36. Han S., Mahato R.I., Kim S.W. Water-soluble lipopolymer for gene delivery. Bioconjug Chem 2001, 12(3):337-345.
37. Kim J.M., Lee M., Kim K.H., Ha Y., Choi J.K., Park S.R., et al. Gene therapy of neural cell injuries in vitro using the hypoxia-inducible GM-CSF expression plasmids and water-soluble lipopolymer (WSLP). J Control Release 2009, 133(1):60-67.
38. Philipp A., Zhao X., Tarcha P., Wagner E., Zintchenko A. Hydrophobically modified oligoethylenimines as highly efficient transfection agents for siRNA delivery. Bioconjug Chem 2009, 20(11):2055-2061.
39. Oberle V., Bakowsky U., Zuhorn I.S., Hoekstra D. Lipoplex formation under equilibrium conditions reveals a three-step mechanism. Biophys J 2000, 79(3):1447-1454.
40. Tabatt K., Kneuer C., Sameti M., Olbrich C., Muller R.H., Lehr C.M., et al. Transfection with different colloidal systems: comparison of solid lipid nanoparticles and liposomes. J Control Release 2004, 97(2):321-332.
41. Xu Y., Szoka F.C. Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry 1996, 35(18):5616-5623.
42. Szoka F.C. The future of liposomal drug delivery. Biotechnol Appl Biochem 1990, 12(5):496-500.
43. Ko Y.T., Bhattacharya R., Bickel U. Liposome encapsulated polyethylenimine/ODN polyplexes for brain targeting. J Control Release 2009, 133(3):230-237.
44. Garcia L., Bunuales M., Duzgunes N., Tros de Ilarduya C. Serum-resistant lipopolyplexes for gene delivery to liver tumour cells. Eur J Pharm Biopharm 2007, 67(1):58-66.
45. Lee C.H., Ni Y.H., Chen C.C., Chou C., Chang F.H. Synergistic effect of polyethylenimine and cationic liposomes in nucleic acid delivery to human cancer cells. Biochim Biophys Acta 2003, 1611(1-2):55-62.
46. Pelisek J., Gaedtke L., DeRouchey J., Walker G.F., Nikol S., Wagner E. Optimized lipopolyplex formulations for gene transfer to human colon carcinoma cells under in vitro conditions. J Gene Med 2006, 8(2):186-197.
47. Gaedtke L., Pelisek J., Lipinski K.S., Wrighton C.J., Wagner E. Transcriptionally targeted nonviral gene transfer using a beta-catenin/TCF-dependent promoter in a series of different human low passage colon cancer cells. Mol Pharm 2007, 4(1):129-139.
48. Hanzlikova M., Soininen P., Lampela P., Mannisto P.T., Raasmaja A. The role of PEI structure and size in the PEI/liposome-mediated synergism of gene transfection. Plasmid 2009, 61(1):15-21.
49. Heyes J., Palmer L., Chan K., Giesbrecht C., Jeffs L., MacLachlan I. Lipid encapsulation enables the effective systemic delivery of polyplex plasmid DNA. Mol Ther 2007, 15(4):713-720.
50. Choksakulnimitr S., Masuda S., Tokuda H., Takakura Y., Hashida M. In vitro cytotoxicity of macromolecules in different cell culture systems. J Control Release 1995, 34:233-241.
51. Mao S., Neu M., Germershaus O., Merkel O., Sitterberg J., Bakowsky U., et al. Influence of polyethylene glycol chain length on the physicochemical and biological properties of poly(ethylene imine)-graft-poly(ethylene glycol) block copolymer/SiRNA polyplexes. Bioconjug Chem 2006, 17(5):1209-1218.
52. Anabousi S., Laue M., Lehr C.M., Bakowsky U., Ehrhardt C. Assessing transferrin modification of liposomes by atomic force microscopy and transmission electron microscopy. Eur J Pharm Biopharm 2005, 60(2):295-303.
53. Brodsky F.M., Chen C.Y., Knuehl C., Towler M.C., Wakeham D.E. Biological basket weaving: formation and function of clathrin-coated vesicles. Annu Rev Cell Dev Biol 2001, 17:517-568.
54. Wang L.H., Rothberg K.G., Anderson R.G. Mis-assembly of clathrin lattices on endosomes reveals a regulatory switch for coated pit formation. J Cell Biol 1993, 123(5):1107-1117.
55. Orlandi P.A., Fishman P.H. Filipin-dependent inhibition of cholera toxin: evidence for toxin internalization and activation through caveolae-like domains. J Cell Biol 1998, 141(4):905-915.
56. von Gersdorff K., Sanders N.N., Vandenbroucke R., De Smedt S.C., Wagner E., Ogris M. The internalization route resulting in successful gene expression depends on both cell line and polyethylenimine polyplex type. Mol Ther 2006, 14(5):745-753.
57. Rejman J., Bragonzi A., Conese M. Role of clathrin- and caveolae-mediated endocytosis in gene transfer mediated by lipo- and polyplexes. Mol Ther 2005, 12(3):468-474.
58. Aigner A. Nonviral in vivo delivery of therapeutic small interfering RNAs. Curr Opin Mol Ther 2007, 9(4):345-352.
59. Ripka S., Neesse A., Riedel J., Bug E., Aigner A., Poulsom R., et al. CUX1: target of Akt signalling and mediator of resistance to apoptosis in pancreatic cancer. Gut 2010, [epub ahead of print]. doi:10.1136/gut.2009.189720.