Cellular internalization and gene silencing of siRNA polyplexes by cytocleavable cationic polyrotaxanes with tailored rigid backbones

Biomaterials

Volumn 34, Issue 10, 2013, Pages 2480-2491

  Tamura, Atsushi ^a   Yui, Nobuhiko ^a  

^a TOKYO MEDICAL AND DENTAL UNIVERSITY   (Japan)

Author keywords

Disulfide linkage; Polyplex; Polyrotaxane; SiRNA; Supermolecule

Indexed keywords

DISULFIDE LINKAGES; POLYPLEXES; POLYROTAXANES; SIRNA; SUPER MOLECULES;

CYCLODEXTRINS; HYDROGELS; POLYETHYLENE GLYCOLS;

GENETIC ENGINEERING;

ALPHA CYCLODEXTRIN; CATION; LUCIFERASE; MACROGOL; N,N DIMETHYLAMINOETHYL; POLY(ETHYLENIMINE); POLYMER; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ARTICLE; BINDING KINETICS; CHEMICAL MODIFICATION; CHEMICAL STRUCTURE; CONTROLLED STUDY; CYTOTOXICITY; DRUG DELIVERY SYSTEM; FEMALE; FLOW CYTOMETRY; GENE SILENCING; HELA CELL; HUMAN; HUMAN CELL; MOLECULAR WEIGHT; PHYSICAL CHEMISTRY; PRIORITY JOURNAL; PROTEIN EXPRESSION; STRUCTURE ACTIVITY RELATION; SYNTHESIS; UTERINE CERVIX CARCINOMA;

GENE SILENCING; MODELS, CHEMICAL; POLYETHYLENE GLYCOLS; RNA, SMALL INTERFERING; ROTAXANES;

EID: 84872679923     PISSN: 01429612     EISSN: 18785905     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2012.12.006     Document Type: Article

References (41)

1. Stevenson M. Therapeutic potential of RNA interference. New Engl J Med 2004, 351(17):1772-1777.
2. Shankar P., Manjunath N., Lieberman J. The prospect of silencing disease using RNA interference. JAMA 2005, 293(11):1367-1373.
3. Kim D.H., Rossi J.J. Strategies for silencing human disease using RNA interference. Nat Rev Genet 2007, 8(3):173-184.
4. Akhtar S., Benter I.F. Nonviral delivery of synthetic siRNAs in vivo. J Clin Invest 2007, 117(12):3623-3632.
5. Whitehead K.A., Langer R., Anderson D.G. Knocking down barriers: advances in siRNA delivery. Nat Rev Drug Discov 2009, 8(2):129-138.
6. Oh Y.K., Park T.G. siRNA delivery systems for cancer treatment. Adv Drug Deliv Rev 2009, 61(10):850-862.
7. Tamura A., Nagasaki Y. Smart siRNA delivery systems based on polymeric nanoassemblies and nanoparticles. Nanomedicine 2010, 5(7):1089-1102.
8. Mok H., Lee S.H., Park J.W., Park T.G. Multimeric small interfering ribonucleic acid for highly effi{ligature}cient sequence-specifi{ligature}c gene silencing. Nat Mater 2010, 9(3):272-278.
9. Alshamsan A., Haddadi A., Incani V., Samuel J., Lavasanifar A., Uludaĝ H. Formulation and delivery of siRNA by oleic acid and stearic acid modifi{ligature}ed polyethylenimine. Mol Pharm 2009, 6(1):121-133.
10. Kim H.J., Ishii A., Miyata K., Lee Y., Wu S., Oba M., et al. Introduction of stearoyl moieties into a biocompatible cationic polyaspartamide derivative, PAsp(DET), with endosomal escaping function for enhanced siRNA-mediated gene knockdown. J Control Release 2010, 145(2):141-148.
11. Matsumoto S., Christie R.J., Nishiyama N., Miyata K., Ishii A., Oba M., et al. Environment-responsive block copolymer micelles with a disulfide cross-linked core for enhanced siRNA delivery. Biomacromolecules 2009, 10(1):119-127.
12. 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(7):1818-1827.
13. Lee S.Y., Huh M.S., Lee S., Lee S.J., Chung H., Park J.H., et al. Stability and cellular uptake of polymerized siRNA (poly-siRNA)/polyethylenimine (PEI) complexes for efficient gene silencing. J Control Release 2010, 141(3):339-346.
14. Harada A. Cyclodextrin-based molecular machines. Acc Chem Res 2001, 34(6):456-464.
15. Wenz G., Han B.H., Müller A. Cyclodextrin rotaxanes and polyrotaxanes. Chem Rev 2006, 106(3):782-817.
16. Li J., Loh X.J. Cyclodextrin-based supramolecular architectures: syntheses, structures, and applications for drug and gene delivery. Adv Drug Deliv Rev 2008, 60(9):1000-1017.
17. Ooya T., Choi H.S., Yamashita A., Yui N., Sugaya Y., Kano A., et al. Biocleavable polyrotaxane-plasmid DNA polyplex for enhanced gene delivery. J Am Chem Soc 2006, 128(12):3852-3853.
18. Yamashita A., Yui N., Ooya T., Kano A., Maruyama A., Akita H., et al. Synthesis of a biocleavable polyrotaxane-plasmid DNA (pDNA) polyplex and its use for the rapid nonviral delivery of pDNA to cell nuclei. Nat Protoc 2006, 1(6):2861-2869.
19. Yamashita A., Kanda D., Katoono R., Yui N., Ooya T., Maruyama A., et al. Supramolecular control of polyplex dissociation and cell transfection: effi{ligature}cacy of amino groups and threading cyclodextrins in biocleavable polyrotaxanes. J Control Release 2008, 131(2):137-144.
20. Shaheen S.M., Akita H., Nakamura T., Takayama S., Futaki S., Yamashita A., et al. KALA-modifi{ligature}ed multi-layered nanoparticles as gene carriers for MHC class-I mediated antigen presentation for a DNA vaccine. Biomaterials 2011, 32(26):6342-6350.
21. Yamada Y., Nomura T., Harashima H., Yamashita A., Yui N. Post-nuclear gene delivery events for transgene expression by biocleavable polyrotaxanes. Biomaterials 2012, 33(15):3952-3958.
22. Meng F., Hennink W.E., Zhong Z. Reduction-sensitive polymers and bioconjugates for biomedical applications. Biomaterials 2009, 30(12):2180-2198.
23. Saito G., Swanson J.A., Lee K.D. Drug delivery strategy utilizing conjugation via reversible disulfi{ligature}de linkages: role and site of cellular reducing activities. Adv Drug Deliv Rev 2003, 55(2):199-215.
24. Meister A., Anderson M.E. Glutathioine. Annu Rev Biochem 1983, 52:711-760.
25. Wu G., Fang Y.Z., Yang S., Lupton J.R., Turner N.D. Glutathione metabolism and its implications for health. J Nutr 2004, 134(3):489-492.
26. Dandekar P., Jain R., Keil M., Loretz B., Muijs L., Schneider M., et al. Cellular delivery of polynucleotides by cationic cyclodextrin polyrotaxanes. J Control Release 2012, 164(3):387-393.
27. Yamada Y., Hashida M., Nomura T., Harashima H., Yamasaki Y., Kataoka K., et al. Different mechanisms for nanoparticle formation between pDNA and siRNA using polyrotaxane as the polycation. ChemPhysChem 2012, 13(5):1161-1165.
28. Oshima H. Electrophoretic mobility of soft particles. Colloid Surf A 1995, 103(3):249-255.
29. LePecq J.B., Paoletti C. A fluorescent complex between ethidium bromide and nucleic acids. physical-chemical characterization. J Mol Biol 1967, 27(1):87-106.
30. Suma T., Miyata K., Ishii T., Uchida S., Uchida H., Itaka K., et al. Enhanced stability and gene silencing ability of siRNA-loaded polyion complexes formulated from polyaspartamide derivatives with a repetitive array of amino groups in the side chain. Biomaterials 2012, 33(9):2770-2779.
31. Griffith O.W., Meister A. Potent and specific inhibition of glutathione synthesis by buthionine sulfoximine (S-n-butyl homocysteine sulfoximine). J Biol Chem 1979, 254(16):7558-7560.
32. Puri R.N., Meister A. Transport of glutathione, as γ-glutamylcysteinylglycyl ester, into liver and kidney. Proc Natl Acad Sci U S A 1983, 80(17):5258-5260.
33. Li J., Yang C., Li H., Wang X., Goh S.H., Ding J.L., et al. Cationic supramolecules composed of multiple oligoethylenimine-grafted β-cyclodextrins threaded on a polymer chain for efficient gene delivery. Adv Mater 2006, 18(22):2969-2974.
34. Kunath K., von Harpe A., Fischer D., Petersen H., Bickel U., Voigt K., et al. Low-molecular-weight polyethylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyethylenimine. J Control Release 2003, 89(1):113-125.
35. Strand S.P., Danielsen S., Christensen B.F., Vårum K.M. Influence of chitosan structure on the formation and stability of DNA-chitosan polyelectrolyte complexes. Biomacromolecules 2005, 6(6):3357-3366.
36. Tamura A., Oishi M., Nagasaki Y. 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. J Control Release 2010, 146(3):378-387.
37. Bolcato-Bellemin A.L., Bonnet M.E., Creusat G., Erbacher P., Behr J.P. Sticky overhangs enhance siRNA-mediated gene silencing. Proc Natl Acad Sci USA 2007, 104(41):16050-16055.
38. 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.
39. Fleury G., Brochon C., Schlatter G., Bonnet G., Lapp A., Hadziioannou A. Synthesis and characterization of high molecular weight polyrotaxanes: towards the control over a wide range of threaded α-cyclodextrins. Soft Matter 2005, 1(5):378-385.
40. Jarroux N., Guégan P., Cheradame H., Auvray L. High conversion synthesis of pyrene end functionalized polyrotaxane based on poly(ethylene oxide) and α-cyclodextrins. J Phys Chem B 2005, 109(50):23816-23822.
41. Austin C.D., Wen X., Gazzard L., Nelson C., Scheller R.H., Scales S.J. Oxidizing potential of endosomes and lysosomes limits intracellular cleavage of disulfide-based antibody-drug conjugates. Proc Natl Acad Sci USA 2005, 102(50):17987-17992.