Efficient siRNA delivery based on PEGylated and partially quaternized polyamine nanogels: Enhanced gene silencing activity by the cooperative effect of tertiary and quaternary amino groups in the core

Journal of Controlled Release

Volumn 146, Issue 3, 2010, Pages 378-387

  Tamura, Atsushi ^a   Oishi, Motoi ^a   Nagasaki, Yukio ^a,b  

^a UNIVERSITY OF TSUKUBA   (Japan)

^b NATIONAL INSTITUTE FOR MATERIALS SCIENCE   (Japan)

Author keywords

Intracellular trafficking; PEGylated nanogel; Polyion complex; SiRNA delivery; Survivin

Indexed keywords

INTRACELLULAR TRAFFICKING; NANOGELS; POLYION COMPLEX; SIRNA DELIVERY; SURVIVIN;

AMMONIUM COMPOUNDS; DYES; ETHYLENE; ETHYLENE GLYCOL; GELS; GENETIC ENGINEERING; NANOSTRUCTURED MATERIALS; POLYETHYLENE GLYCOLS; POLYETHYLENE OXIDES;

RNA;

MACROGOL; NANOMATERIAL; POLY[2 (N,N DIETHYLAMINOETHYL)METHACRYLATE]; POLYAMINE; POLYMER; QUATERNARY AMMONIUM DERIVATIVE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ARTICLE; CANCER CELL CULTURE; CHEMICAL STRUCTURE; CONTROLLED STUDY; CYTOTOXICITY; DRUG DELIVERY SYSTEM; DRUG UPTAKE; GEL; GENE SILENCING; HUMAN; HUMAN CELL; HYDROPHILICITY; PRIORITY JOURNAL; STRUCTURE ANALYSIS;

CELL LINE, TUMOR; CELL SURVIVAL; GENE SILENCING; HUMANS; MICROSCOPY, CONFOCAL; POLYAMINES; POLYETHYLENE GLYCOLS; POLYETHYLENEIMINE; RNA, SMALL INTERFERING;

EID: 77956271401     PISSN: 01683659     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jconrel.2010.05.031     Document Type: Article

References (44)

1. Kim D.H., Rossi J.J. Strategies for silencing human disease using RNA interference. Nat. Rev. Genet. 2007, 8:173-184.
2. Akhtar S., Benter I.F. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 2007, 117:3623-3632.
3. Whitehead K.A., Langer R., Anderson D.G. Knocking down barriers: advances in siRNA delivery. Nat. Rev. Drug Discov. 2009, 8:129-138.
4. 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, 12:461-466.
5. Nakamura Y., Kogure K., Futaki S., Harashima H. Octaarginine-modified multifunctional envelope-type nano device for siRNA. J. Control. Release 2007, 119:360-367.
6. Akinc A., Zumbuehl A., Goldberg M., Leshchiner E.S., Busini V., Hossain N., Bacallado S.A., Nguyen D.N., Fuller J., Alvarez R., Borodovsky A., Borland T., Constien R., de Fougerolles A., Dorkin J.R., Jayaprakash K.N., Jayaraman M., John M., Koteliansky V., Manoharan M., Nechev L., Qin J., Racie T., Raitcheva D., Rajeev K.G., Sah D.W.Y., Soutschek J., Toudjarska I., Vornlocher H.-P., Zimmermann T.S., Langer R., Anderson D.G. A combinatorial library of lipid-like materials for delivery of RNAi therapeutics. Nat. Biotechnol. 2008, 26:561-569.
7. Kim W.J., Christensen L.V., Jo S., Yockman J.W., Jeong J.H., Kim Y.-H., Kim S.W. Cholesteryl oligoarginine delivering vascular endothelial growth factor siRNA effectively inhibits tumor growth in colon adenocarcinoma. Mol. Ther. 2006, 14:343-350.
8. Segura T., Hubbell J.A. Synthesis and in vitro characterization of an ABC triblock copolymer for siRNA delivery. Bioconjug. Chem. 2007, 18:736-745.
9. a directed to enhance intracellular gene silencing. J. Am. Chem. Soc. 2004, 126:13612-13613.
10. Meyer M., Philipp A., Oskuee R., Schmidt C., Wagner E. Breathing life into polycations: functionalization with pH-responsive endosomolytic peptides and polyethylene glycol enables siRNA delivery. J. Am. Chem. Soc. 2008, 130:3272-3273.
11. Oishi M., Nagasaki Y., Itaka K., Nishiyama N., Kataoka K. Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile β-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells. J. Am. Chem. Soc. 2005, 127:1624-1625.
12. Kim S.H., Jeong J.H., Lee S.H., Kim S.W., Park T.G. Local and systemic delivery of VEGF siRNA using polyelectrolyte complex micelles for effective treatment of cancer. J. Control. Release 2008, 129:107-116.
13. Maeda H., Wu J., Sawa T., Matsumura Y., Hori K. Tumor vascular permeability and the EPR effect in macromolecular therapeutics: a review. J. Control. Release 2000, 65:271-284.
14. Katayose S., Kataoka K. Remarkable increase in nuclease resistance of plasmid DNA through supramolecular assembly with poly(ethylene glycol)-poly(L-lysine) block copolymer. J. Pharm. Sci. 1998, 87:160-163.
15. Gao S., Dagnaes-Hansen F., Nielsen E.J.B., Wengel J., Besenbacher F., Howard K.A., Kjems J. The effect of chemical modification and nanoparticle formulation on stability and biodistribution of siRNA in mice. Mol. Ther. 2009, 17:1225-1233.
16. Bartlett D.W., Su H., Hildebrandt I.J., Weber W.A., Davis M.E. Impact of tumor-specific targeting on the biodistribution and efficacy of siRNA nanoparticles measured by multimodality in vivo imaging. Proc. Natl. Acad. Sci. U.S.A. 2007, 104:15549-15554.
17. Malek A., Merkel O., Fink L., Czubayko F., Kissel T., Aigner A. In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(-PEG)/siRNA complexes. Toxicol. Appl. Pharm. 2009, 236:97-108.
18. Oishi M., Nagasaki Y. Synthesis, characterization, and biomedical applications of core-shell-type stimuli-responsive nanogels - nanogel composed of poly[2-(N,N-diethylamino)ethyl methacrylate] core and PEG tethered chains. React. Func. Polym. 2007, 67:1311-1329.
19. Oishi M., Hayashi H., Itaka K., Kataoka K., Nagasaki Y. pH-Responsive PEGylated nanogels as targetable and low invasive endosomolytic agents to induce the enhanced transfection efficiency of nonviral gene vectors. Colloid Polym. Sci. 2007, 285:1055-1060.
20. Tamura A., Oishi M., Nagasaki Y. Enhanced cytoplasmic delivery of siRNA using a stabilized polyion complex based on PEGylated nanogels with a cross-linked polyamine structure. Biomacromolecules 2009, 10:1818-1827.
21. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., Behr J.-P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. U.S.A. 1995, 92:7297-7301.
22. Wang J., Gao S.-J., Zhang P.-C., Wang S., Mao H.-Q., Leong K.W. Polyphosphoramidate gene carriers: effect of charge group on gene transfer efficiency. Gene Ther. 2004, 11:1001-1010.
23. Reschel T., Koňák ý., Oupickỳ D., Seymour L.W., Ulbrich K. Physical properties and in vitro transfection efficiency of gene delivery vectors based on complexes of DNA with synthetic polycations. J. Control. Release 2002, 81:201-217.
24. Arigita C., Zuidam N.J., Crommelin D.J.A., Hennink W.E. Association and dissociation characteristics of polymer/DNA complexes used for gene delivery. Pharm. Res. 1999, 16:1534-1541.
25. Izumrudov V.A., Zhiryakova M.V., Kudaibergenov S.E. Controllable stability of DNA-containing polyelectrolyte complexes in water-salt solutions. Biopolymers 1999, 52:94-108.
26. Juan A.S., Letourneur D., Izumrudov V.A. Quaternized poly(4-vinylpyridine)s as model gene delivery polycations: structure-function study by modification of side chain hydrophobicity and degree of alkylation. Bioconjug. Chem. 2007, 18:922-928.
27. Patil M.L., Zhang M., Betigeri S., Taratula O., He H., Minko T. Surface-modified and internally cationic polyamidoamine dendrimers for efficient siRNA delivery. Bioconjug. Chem. 2008, 19:1396-1403.
28. Lee J.H., Lim Y.-B., Choi J.S., Lee Y., Kim T.-I., Kim H.J., Yoon J.K., Kim K., Park J.-S. Polyplexes assembled with internally quaternized PAMAM-OH dendrimer and plasmid DNA have a neutral surface and gene delivery potency. Bioconjug. Chem. 2003, 14:1214-1221.
29. van de Wetering P., Moret E.E., Schuurmans-Nieuwenbroek N.M.E., van Steenbergen M.J., Hennink W.E. Structure-activity relationships of water-soluble cationic methacrylate/methacrylamide polymers for nonviral gene delivery. Bioconjug. Chem. 1999, 10:589-597.
30. Ambrosini G., Adida C., Altieri D.C. A novel anti-apoptosis gene, survivin, expressed in cancer and lymphoma. Nat. Med. 1997, 3:917-921.
31. Altieri D.C. Validating surviving as a cancer therapeutic target. Nat. Rev. Cancer 2003, 3:46-54.
32. Oishi M., Tamura A., Nakamura T., Nagasaki Y. A smart nanoprobe based on fluorescence-quenching PEGylated nanogels containing gold nanoparticles for monitoring the response to cancer therapy. Adv. Func. Mater. 2009, 19:827-834.
33. Kappler M., Bache M., Bartel F., Kotzsch M., Panian M., Würl P., Blümke K., Schmidt H., Meye A., Taubert H. Knockdown of survivin expression by small interfering RNA reduces the clonogenic survival of human sarcoma cell lines independently of p53. Cancer Gene Ther. 2004, 11:186-193.
34. Bütün V., Armes S.P., Billingham N.C. Selective quaternization of 2-(dimethylamino)ethyl methacrylate residues in tertiary amine methacrylate diblock copolymers. Macromolecules 2001, 34:1148-1159.
35. Miyata K., Fukushima S., Nishiyama N., Yamasaki Y., Kataoka K. PEG-based block catiomers possessing DNA anchoring and endosomal escaping functions to form polyplex micelles with improved stability and high transfection efficacy. J. Control. Release 2007, 122:252-260.
36. Vinogradov S.V., Bronich T.K., Kabanov A.V. Self-assembly of polyamine-poly(ethylene glycol) copolymers with phosphorothioate oligonucleotides. Bioconjug. Chem. 1998, 9:805-812.
37. Davis M.E., Chen Z., Shin D.M. Nanoparticle therapeutics: an emerging treatment modality for cancer. Nat. Rev. Drug Discov. 2008, 7:771-782.
38. Moreau E., Domurado M., Chapon P., Vert M., Domurado D. Biocompatibility of polycations: In vitro agglutination analysis of red blood cells and in vivo toxicity. J. Drug Target. 2002, 10:161-173.
39. Fischer D., Li Y., Ahlemeyer B., Krieglstein J., Kissel T. In vitro cytotoxicity testing of polycations: influence of polymer structure on cell viability and hemolysis. Biomaterials 2003, 24:1121-1131.
40. Fuessel S., Herrmann J., Ning S., Kotzsch M., Kraemer K., Schmidt U., Hakenberg O.W., Wirth M.P., Meye A. Chemosensitization of bladder cancer cells by survivin-directed antisense oligodeoxynucleotides and siRNA. Cancer Lett. 2006, 232:243-254.
41. Li S.D., Huang L. Targeted delivery of antisense oligodeoxynucleotide and small interference RNA into lung cancer cells. Mol. Pharm. 2006, 3:579-588.
42. Saito T., Hama S., Izumi H., Yamasaki F., Kajiwara Y., Matsuura S., Morishima K., Hidaka T., Shrestha P., Sugiyama K., Kurisu K. Centrosome amplification induced by survivin suppression enhances both chromosome instability and radiosensitivity in glioma cells. Br. J. Cancer 2008, 98:345-355.
43. Ito T., Shiraki K., Sugimoto K., Yamanaka T., Fujikawa K., Ito M., Takase K., Moriyama M., Kawano H., Hayashida M., Nakano T., Suzuki A. Survivin promotes cell proliferation in human hepatocellular carcinoma. Hapatology 2000, 31:1080-1085.
44. Jackson A.L., Bartz S.R., Schelter J., Kobayashi S.V., Burchard J., Mao M., Li B., Cavet G., Linsley P.S. Expression profiling reveals off-target gene regulation by RNAi. Nat. Biotech. 2003, 21:635-637.