Dual mode polyspermine with tunable degradability for plasmid DNA and siRNA delivery

Biomaterials

Volumn 32, Issue 16, 2011, Pages 4009-4020

  Shim, Min Suk ^a   Kwon, Young Jik ^a,b  

^a University of California at Irvine   (United States)

^b UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Biodegradability; DNA and siRNA delivery; In vitro and in vivo transfection; Intracellular release; Nonviral gene delivery; Polyspermine

Indexed keywords

CATIONIC MOLECULES; CELLULAR UPTAKE; CROSS-LINKERS; CYTOSOLS; CYTOTOXIC; DEGRADABILITY; DEGRADATION RATE; DESIGN COMPONENT; DIACRYLATES; DIFFERENTIAL DEGRADATION; DNA COMPLEXATION; DNA TRANSFECTION; DUAL MODES; ENDOSOMES; ESTER RATIO; GENE CARRIER; GENE SILENCING; GFP EXPRESSION; IN VITRO AND IN VIVO TRANSFECTION; IN-VITRO; IN-VIVO; INTRACELLULAR RELEASE; NON-VIRAL GENE DELIVERY; NONVIRAL; PLASMID DNA; POLYMERIC BACKBONE; POLYMERIC GENE CARRIERS; POLYSPERMINE; RELEASE EFFICIENCY; RNA INTERFERENCE; SIRNA DELIVERY; STIMULI-RESPONSIVE;

BIODEGRADATION; BIOMOLECULES; DEGRADATION; DNA; DYES; ESTERIFICATION; ESTERS; FUNCTIONAL POLYMERS; GENE THERAPY; GENE TRANSFER; POLYMERS; RNA;

NUCLEIC ACIDS;

ACETAL; ACID; GREEN FLUORESCENT PROTEIN; NUCLEIC ACID; PLASMID DNA; POLY(SPERMINE ESTER); POLY(SPERMINE KETAL ESTER); POLYMER; POLYSPERMINE; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

ANIMAL CELL; ARTICLE; COMPLEX FORMATION; CYTOSOL; DNA DEGRADATION; DNA TRANSFECTION; DRUG DELIVERY SYSTEM; ENDOSOME; GENE SILENCING; IN VITRO STUDY; IN VIVO STUDY; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; POLYMERIZATION; PRIORITY JOURNAL; RNA INTERFERENCE;

ANIMALS; DNA; FEMALE; MICE; NIH 3T3 CELLS; PLASMIDS; POLYMERS; RNA, SMALL INTERFERING; SPERMINE;

EID: 79953030154     PISSN: 01429612     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.biomaterials.2011.02.010     Document Type: Article

References (50)

1. Mulligan R.C. The basic science of gene therapy. Science 1993, 260:926-932.
2. Somia N., Verma I.M. Gene therapy: trials and tribulations. Nat Rev Gene 2000, 1:91-99.
3. Luo D., Saltzman W.M. Synthetic DNA delivery systems. Nat Biotechnol 2000, 18:33-37.
4. Liu F., Huang L. Development of non-viral vectors for systemic gene delivery. J Control Release 2002, 78:259-266.
5. Read M.L., Singh S., Ahmed Z., Stevenson M., Briggs S.S., Oupicky D., et al. A versatile reducible polycation-based system for efficient delivery of a broad range of nucleic acids. Nucleic Acids Res 2005, 33:e86.
6. Park T.G., Jeong J.H., Kim S.W. Current status of polymeric gene delivery systems. Adv Drug Deliv Rev 2006, 58:467-486.
7. Merdan T., Kopeck J., Kissel T. Prospects for cationic polymers in gene and oligonucleotide therapy against cancer. Adv Drug Deliv Rev 2002, 54:715-758.
8. Werth S., Urban-Klein B., Dai L., Höbel 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, 112:257-270.
9. 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, 45:268-275.
10. Fischer D., Bieber T., Li Y., Elsässer H., Kissel T. A novel non-viral vector for DNA delivery based on low molecular weight, branched polyethylenimine: effect of molecular weight on transfection efficiency and cytotoxicity. Pharm Res 1999, 16:1273-1279.
11. Wong S.Y., Pelet J.M., Putnam D. Polymer systems for gene delivery-past, present, and future. Prog Polym Sci 2007, 32:799-837.
12. Lynn D.M., Langer R. Degradable poly(β-amino esters): synthesis, characterization, and self-assembly with plasmid DNA. J Am Chem Soc 2000, 122:10761-10768.
13. Tinsley-Bown A.M., Fretwell R., Dowsett A.B., Davis S.L., Farrar G.H. Formulation of poly(D, L-lactic-co-glycolic acid) microparticles for rapid plasmid DNA delivery. J Control Release 2000, 66:229-241.
14. Lim Y.-B., Kim C.-H., Kim K., Kim S.W., Park J.-S. Development of a safe gene delivery system using biodegradable polymer, poly[α-(4-aminobutyl)-L-glycolic acid]. J Am Chem Soc 2002, 122:6524-6525.
15. Gosselin M.A., Guo W., Lee R.J. Efficient gene transfer using reversibly cross-linked low molecular weight polyethylenimine. Bioconjug Chem 2001, 12:989-994.
16. Ou M., Wang X.-L., Xu R., Chang C.-W., Bull D.A., Kim S.W. Novel biodegradable poly(disulfide amine)s for gene delivery with high efficiency and low cytotoxicity. Bioconjug Chem 2008, 19:626-633.
17. Lee Y., Mo H., Koo H., Park J.-Y., Cho M.Y., Jin G.-W., et al. Visualization of the degradation of a disulfide polymer, linear poly(ethylenimine sulfide), for gene delivery. Bioconjug Chem 2007, 18:13-18.
18. Kim Y.H., Park J.H., Lee M., Kim Y.-H., Park T.G., Kim S.W. Polyethylenimine with acid-labile linkages as a biodegradable gene carrier. J Control Release 2005, 103:209-219.
19. Knauf M.J., Bell D.P., Hirtzer P., Luo Z.-P., Young J.D., Katre N.V. Relationship of effective molecular size to systemic clearance in rats of recombinant interleukin-2 chemically modified with water-soluble polymers. J Biol Chem 1988, 263:15064-15070.
20. Hardy J.G., Kostiainen M.A., Smith D.K., Gabrielson N.P., Pack D.W. Dendrons with spermine surface groups as potential building blocks for nonviral vectors in gene therapy. Bioconjug Chem 2006, 17:172-178.
21. Hosseinkhani H., Azzam T., Tabata Y., Domb A.J. Dextran-spermine polycation: an efficient nonviral vector for in vitro and in vivo gene transfection. Gene Ther 2004, 11:194-203.
22. Shim M.S., Kwon Y.J. Controlled delivery of plasmid DNA and siRNA to intracellular targets using ketalized polyethylenimine. Biomacromolecules 2008, 9:444-455.
23. Shim M.S., Kwon Y.J. Controlled cytoplasmic and nuclear localization of plasmid DNA and siRNA by differentially tailored polyethylenimine. J Control Release 2009, 133:206-213.
24. Shim M.S., Kwon Y.J. Acid-responsive linear polyethylenimine for efficient, specific, and biocompatible siRNA delivery. Bioconjug Chem 2009, 20:488-499.
25. Shim M.S., Kwon Y.J. Acid-transforming polypeptide micelles for targeted nonviral gene delivery. Biomaterials 2010, 31:3404-3413.
26. Shim M.S., Wang X., Ragan R., Kwon Y.J. Dynamics of nucleic acid/cationic polymer complexation and disassembly under biologically simulated conditions using in situ atomic force microscopy. Microsc Res Tech 2010, 73:845-856.
27. Cho SK, Kwon YJ. Polyamine/DNA polyplexes with acid-degradable polymeric shell as structurally and functionally virus-mimicking nonviral vectors. J Control Release doi:10.1016/j.jconrel.2010.12.004.
28. Boussif O., Lenzoualc'h F., Zanta M.A., Mergny M.D., Schlerman 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 USA 1995, 92:7297-7301.
29. Lim Y.-B., Kim S.-M., Suh H., Park J.-S. Biodegradable, endosome disruptive, and cationic network-type polymer as a highly efficient and nontoxic gene delivery carrier. Bioconjug Chem 2002, 13:952-957.
30. Dunlap D.D., Maggi A., Soria M.R., Monaco L. Nanoscopic structure of DNA condensed for gene delivery. Nucleic Acids Res 1997, 25:3095-3101.
31. Gleitsman K.R., Lester H.A., Dougherty D.A. Probing the role of backbone hydrogen bonding in a critical β sheet of the extracellular domain of a cys-loop receptor. ChemBioChem 2009, 10:1385-1391.
32. Kostiainen M.A., Hardy J.G., Smith D.K. High-affinity multivalent DNA binding by using low-molecular-weight dendrons. Angew Chem Int Ed 2005, 44:2556-2559.
33. Mislick K.A., Baldeschwieler J.D. Evidence for the role of proteoglycans in cation-mediated gene transfer. Proc Natl Acad Sci USA 1996, 93:12349-12354.
34. Gabrielson N.P., Pack D.W. Acetylation of polyethylenimine enhances gene delivery via weakened polymer/DNA interactions. Biomacromolecules 2006, 7:2427-2435.
35. Pitard B., Oudrhiri N., Lambert O., Vivien E., Masson C., Wetzer B., et al. Sterically stabilized BGTC-based lipoplexes: structural features and gene transfection into the mouse airways in vivo. J Gene Med 2001, 3:478-487.
36. Guo W., Lee R.J. Efficient gene delivery via non-covalent complexes of folic acid and polyethylenimine. J Control Release 2001, 77:131-138.
37. Goldman C.K., Soroceanu L., Smith N., Gillespie G.Y., Shaw W., Burgess S., et al. In vitro and in vivo gene delivery mediated by a synthetic polycationic amino polymer. Nat Biotechnol 1997, 15:462-466.
38. Murthy N., Campbell J., Fausto N., Hoffman A.S., Stayton P.S. Design and synthesis of pH-responsive polymeric carriers that target uptake and enhance the intracellular delivery of oligonucleotides. J Control Release 2003, 89:365-374.
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. Hartmann L., Häfele S., Peschka-Süss R., Antonietti M., Börner H.G. Tailor-made poly(amidoamine)s for controlled complexation and condensation of DNA. Chem Eur J 2008, 14:2025-2033.
41. Jones N.A., Hill I.R.C., Stolnik S., Bignotti S.F., Davis S.S., Garnett M.C. Polymer chemical structure is a key determinant of physicochemical and colloidal properties of polymer-DNA complexes for gene delivery. Biochim Biophys Acta 2000, 1517:1-18.
42. Anderson D.G., Akinc A., Hossain N., Langer R. Structure/property studies of polymeric gene delivery using a library of poly (β-amino esters). Mol Ther 2005, 11:426-434.
43. Green J.J., Langer R., Anderson D.G. A combinatorial polymer library approach yields insight into nonviral gene delivery. Acc Chem Res 2008, 4:749-759.
44. Liu G., Li D., Pasumarthy M.K., Kowalczyk T.H., Gedeon C.R., Hyatt S.L., et al. Nanoparticles of compacted DNA transfect postmitotic cells. J Biol Chem 2003, 278:32578-32586.
45. 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, 273:7507-7511.
46. Elouahabi A., Ruysschaert J.-M. Formation and intracellular trafficking of lipoplexes and polyplexes. Mol Ther 2005, 11:336-347.
47. Mahato R.I., Kawabata K., Nomura T., Takakura Y., Hashida M. Physicochemical and pharmacokinetic characteristics of plasmid DNA/cationic liposome complexes. J Pharmacol Sci 1995, 84:1267-1271.
48. De Smedt S.C., Demeester J., Hennink W.E. Cationic polymer based gene delivery systems. Pharm Res 2000, 17:113-126.
49. Otsuka H., Nagasaki Y., Kataoka K. Self-assembly of poly(ethylene glycol)-based block copolymers for biomedical applications. Curr Opin Colloid Interface Sci 2001, 6:3-10.
50. 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, 1:210-222.