Gene silencing activity of siRNA polyplexes based on thiolated N, N, N -trimethylated chitosan

Bioconjugate Chemistry

Volumn 21, Issue 12, 2010, Pages 2339-2346

  Varkouhi, Amir K ^a   Verheul, Rolf J ^a   Schiffelers, Raymond M ^a   Lammers, Twan ^a,b   Storm, Gert ^a   Hennink, Wim E ^a  

^a UTRECHT UNIVERSITY   (Netherlands)

^b RWTH AACHEN UNIVERSITY   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

BIODEGRADABLE POLYMERS; CHITOSAN; ENZYME ACTIVITY; GENE TRANSFER; GENES; HYALURONIC ACID; NUCLEIC ACIDS; STABILITY;

CELLULAR CYTOTOXICITY; EXTRACELLULAR; GENE SILENCING; LIPOFECTAMINE; NONVIRAL VECTORS; NUCLEIC ACIDS DELIVERIES; POLYPLEXES; PROTEIN DELIVERY; THIOL GROUPS; TRIMETHYLATED CHITOSANS;

LIPOSOMES;

CHITOSAN; LIPOFECTAMINE; N,N,N TRIMETHYLATED CHITOSAN; UNCLASSIFIED DRUG;

ARTICLE; CANCER CELL; CLINICAL EVALUATION; CONFOCAL LASER MICROSCOPY; CYTOTOXICITY; DRUG ACTIVITY; DRUG DELIVERY SYSTEM; GENE SILENCING; GENE TARGETING; GENE VECTOR; LUNG CANCER;

EID: 78650392120     PISSN: 10431802     EISSN: 15204812     Source Type: Journal    
DOI: 10.1021/bc1003789     Document Type: Article

References (50)

1. Yu, H. and Wagner, E. (2009) Bioresponsive polymers for nonviral gene delivery Curr. Opin. Mol. Ther. 11, 165-178
2. Luten, J., van Nostrum, C. F., De Smedt, S. C., and Hennink, W. E. (2008) Biodegradable polymers as non-viral carriers for plasmid DNA delivery J. Controlled Release 126, 97-110
3. De Smedt, S. C., Demeester, J., and Hennink, W. E. (2000) Cationic polymer based gene delivery systems Pharm. Res. 17, 113-126
4. Mintzer, M. A. and Simanek, E. E. (2009) Nonviral vectors for gene delivery Chem. Rev. 109, 259-302
5. Thomas, M. and Klibanov, A. M. (2003) Non-viral gene therapy: polycation-mediated DNA delivery Appl. Microbiol. Biotechnol. 62, 27-34
6. Jere, D. J. and Cho, C. S. (2010) Biodegradable polymer-mediated sh/siRNA delivery for cancer studies Methods Mol. Biol. (Clifton, N.J.) 623, 243-269
7. Patil, M. L., Zhang., M., Betigeri, S., Taratula, O., He, H., and Minko, T. (2008) Surface-modified and internally cationic polyamidoamine dendrimers for efficient siRNA delivery Bioconjugate Chem. 19, 1396-1403
8. Zintchenko, A., Philipp, A., Dehshahri, A., and Wagner, E. (2008) Simple modifications of branched PEI lead to highly efficient siRNA carriers with low toxicity Bioconjugate Chem. 19, 1448-1455
9. Kuo, J. H. S., Jan, M. S., and Sung, K. C. (2003) Evaluation of the stability of polymer-based plasmid DNA delivery systems after ultrasound exposure Int. J. Pharm. 257, 75-84
10. Gary, D. J., Puri, N., and Won, Y. Y. (2007) Polymer-based siRNA delivery: Perspectives on the fundamental and phenomenological distinctions from polymer-based DNA delivery J. Controlled Release 121, 64-73
11. Xia, T., Kovochich, M., Liong, M., Meng, H., Kabehie, S., George, S., Zink, J. I., and Nel, A. E. (2009) Polyethyleneimine coating enhances the cellular uptake of mesoporous silica nanoparticles and allows safe delivery of siRNA and DNA constructs ACS Nano 3, 3273-3286
12. Grigsby, C. L. and Leong, K. W. (2010) Balancing protection and release of DNA: tools to address a bottleneck of non-viral gene delivery J. R. Soc. Interface 7 Suppl 1, S67-82
13. Putnam, D., Gentry, C. A., Pack, D. W., and Langer, R. (2001) Polymer-based gene delivery with low cytotoxicity by a unique balance of side-chain termini Proc. Natl. Acad. Sci. U.S.A. 98, 1200-1205
14. Mao, S., Sun, W., and Kissel, T. (2010) Chitosan-based formulations for delivery of DNA and siRNA Adv. Drug Delivery Rev. 62, 12-27
15. Mishra, N., Goyal, A. K., Tiwari, S., Paliwal, R., Paliwal, S. R., Vaidya, B., Mangal, S., Gupta, M., Dube, D., Mehta, A., and Vyas, S. P. (2010) Recent advances in mucosal delivery of vaccines: Role of mucoadhesive/ biodegradable polymeric carriers Expert Opin. Ther. Pat. 20, 661-679
16. Amidi, M., Mastrobattista, E., Jiskoot, W., and Hennink, W. E. Chitosan-based delivery systems for protein therapeutics and antigens Adv. Drug Delivery Rev. 62, 59-82
17. Verheul, R. J., Amidi, M., van der Wal, S., van Riet, E., Jiskoot, W., and Hennink, W. E. (2008) Synthesis, characterization and in vitro biological properties of O-methyl free N, N, N-trimethylated chitosan Biomaterials 29, 3642-3649
18. Sahni, J. K., Chopra, S., Ahmad, F. J., and Khar, R. K. (2008) Potential prospects of chitosan derivative trimethyl chitosan chloride (TMC) as a polymeric absorption enhancer: Synthesis, characterization and applications J Pharm. Pharmacol. 60, 1111-1119
19. Thanou, M., Florea, B. I., Langemeÿer, M. W. E., Verhoef, J. C., and Junginger, H. E. (2000) N-trimethylated chitosan chloride (TMC) improves the intestinal permeation of the peptide drug buserelin in vitro (caco-2 cells) and in vivo (rats) Pharm. Res. 17, 27-31
20. Thanou, M., Verhoef, J. C., and Junginger, H. E. (2001) Chitosan and its derivatives as intestinal absorption enhancers Adv. Drug Delivery Rev. 50, 91-101
21. Meng, F., Hennink, W. E., and Zhong, Z. (2009) Reduction-sensitive polymers and bioconjugates for biomedical applications Biomaterials 30, 2180-2198
22. Loretz, B., Thaler, M., and Bernkop-Schnurch, A. (2007) Role of sulfhydryl groups in transfection? A case study with chitosan-NAC nanoparticles Bioconjugate Chem. 18, 1028-1035
23. Breunig, M., Hozsa, C., Lungwitz, U., Watanabe, K., Umeda, I., Kato, H., and Goepferich, A. (2008) Mechanistic investigation of poly(ethylene imine)-based siRNA delivery: Disulfide bonds boost intracellular release of the cargo J. Controlled Release 130, 57-63
24. Verheul, R. J., van der Wal, S., and Hennink, W. E. (2010) Tailorable thiolated trimethyl chitosans for covalently stabilized nanoparticles Biomacromolecules 11, 1965-1971
25. Lee, D., Zhang, W., Shirley, S. A., Kong, X., Hellermann, G. R., Lockey, R. F., and Mohapatra, S. S. (2007) Thiolated chitosan/DNA nanocomplexes exhibit enhanced and sustained gene delivery Pharm. Res. 24, 157-167
26. Slutter, B., Soema, P. C., Ding, Z., Verheul, R., Hennink, W., and Jiskoot, W. Conjugation of ovalbumin to trimethyl chitosan improves immunogenicity of the antigen J. Controlled Release 143, 207-214
27. Yin, L., Ding, J., He, C., Cui, L., Tang, C., and Yin, C. (2009) Drug permeability and mucoadhesion properties of thiolated trimethyl chitosan nanoparticles in oral insulin delivery Biomaterials 30, 5691-5700
28. Langoth, N., Kahlbacher, H., Schöffmann, G., Schmerold, I., Schuh, M., Franz, S., Kurka, P., and Bernkop-Schnürch, A. (2006) Thiolated chitosans: Design and in vivo evaluation of a mucoadhesive buccal peptide drug delivery system Pharm. Res. 23, 573-579
29. Kim, D. H. and Rossi, J. J. (2007) Strategies for silencing human disease using RNA interference Nat. Rev. Genet. 8, 173-184
30. Elbashir, S. M., Harborth, J., Lendeckel, W., Yalcin, A., Weber, K., and Tuschl, T. (2001) Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature 411, 494-498
31. Kurreck, J. (2009) RNA interference: From basic research to therapeutic applications Angew. Chem., Int. Ed. 48, 1378-1398
32. Gilmore, I. R., Fox, S. P., Hollins, A. J., Sohail, M., and Akhtar, S. (2004) The design and exogenous delivery of siRNA for post-transcriptional gene silencing J. Drug Targeting 12, 315-340
33. Jeong, J. H., Mok, H., Oh, Y. K., and Park, T. G. (2009) SiRNA conjugate delivery systems Bioconjugate Chem. 20, 5-14
34. Blackburn, W. H., Dickerson, E. B., Smith, M. H., McDonald, J. F., and Lyon, L. A. (2009) Peptide-functionalized nanogels for targeted siRNA delivery Bioconjugate Chem. 20, 960-968
35. Lesko, E., Miekus, K., Grabowska, A., Gadys, A., and Majka, M. (2008) Optimization of a synthetic siRNA delivery for the treatment of rhabdomyosarcoma Cen. Eur. J. Biol. 3, 371-379
36. Scudiero, D. A., Shoemaker, R. H., Paull, K. D., Monks, A., Tierney, S., Nofziger, T. H., Currens, M. J., Seniff, D., and Boyd, M. R. (1988) Evaluation of a soluble tetrazolium/formazan assay for cell growth and drug sensitivity in culture using human and other tumor cell lines Cancer Res. 48, 4827-4833
37. Ruponen, M., Honkakoski, P., Tammi, M., and Urtti, A. (2004) Cell-surface glycosaminoglycans inhibit cation-mediated gene transfer J. Gene Med. 6, 405-414
38. Ruponen, M., Arkko, S., Reinisalo, M., Urtti, A., and Ranta, V. P. (2008) Intracellular elimination and unpacking kinetics of DNA mediated with various non-viral gene delivery systems Hum. Gene Ther. 19, 1094-1095
39. Neu, M., Sitterberg, J., Bakowsky, U., and Kissel, T. (2006) Stabilized nanocarriers for plasmids based upon cross-linked poly(ethylene imine) Biomacromolecules 7, 3428-3438
40. Chen, T. H. H., Bae, Y., and Furgeson, D. Y. (2008) Intelligent biosynthetic nanobiomaterials (IBNs) for hyperthermic gene delivery Pharm. Res. 25, 683-691
41. Ellis, R. J. (2001) Macromolecular crowding: obvious but underappreciated Trends Biochem. Sci. 26, 597-604
42. Amidi, M., Romeijn, S. G., Borchard., G., Junginger, H. E., Hennink, W. E., and Jiskoot, W. (2006) Preparation and characterization of protein-loaded N-trimethyl chitosan nanoparticles as nasal delivery system J. Controlled Release 111, 107-116
43. Kean, T. and Thanou, M. (2009) Biodegradation, biodistribution and toxicity of chitosan Adv. Drug Delivery Rev. 62, 3-11
44. Wightman, L., Kircheis, R., Rössler, V., Carotta, S., Ruzicka, R., Kursa, M., and Wagner, E. (2001) Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo J. Gene Med. 3, 362-72
45. Elouahabi, A. and Ruysschaert, J. M. (2005) Formation and intracellular trafficking of lipoplexes and polyplexes Mol. Ther. 11, 336-347
46. Höbel, S., Prinz, R., Malek, A., Urban-Klein, B., Sitterberg, J., Bakowsky, U., Czubayko, F., and Aigner, A. (2008) 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. 70, 29-41
47. Hill, I. R. C., Garnett, M. C., Bignotti, F., and Davis, S. S. (1999) In vitro cytotoxicity of poly(amidoamine)s: Relevance to DNA delivery Biochim. Biophys. Acta 1427, 161-174
48. De Wolf, H. K., Luten, J., Snel, C. J., Storm, G., and Hennink, W. E. (2008) Biodegradable, cationic methacrylamide-based polymers for gene delivery to ovarian cancer cells in mice Mol. Pharm. 5, 349-357
49. Van De Wetering, P., Schuurmans-Nieuwenbroek, N. M. E., Hennink, W. E., and Storm, G. (1999) Comparative transfection studies of human ovarian carcinoma cells in vitro, ex vivo and in vivo with poly(2-(dimethylamino)ethyl methacrylate)-based polyplexes J. Gene Med. 1, 156-165
50. Inoue, Y., Kurihara, R., Tsuchida, A., Hasegawa, M., Nagashima, T., Mori, T., Niidome, T., Katayama, Y., and Okitsu, O. (2008) Efficient delivery of siRNA using dendritic poly(l-lysine) for loss-of-function analysis J. Controlled Release 126, 59-66