Controlled cytoplasmic and nuclear localization of plasmid DNA and siRNA by differentially tailored polyethylenimine

Journal of Controlled Release

Volumn 133, Issue 3, 2009, Pages 206-213

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

^a Case Western Reserve University   (United States)

^b University of California at Irvine   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

Controlled intracellular delivery; Ketalized polyethylenimine; Nonviral gene delivery; RNA interference; Transfection

Indexed keywords

AMINES; BIOMOLECULES; CIVIL AVIATION; DNA; EVOLUTIONARY ALGORITHMS; GENE EXPRESSION; GENE TRANSFER; GENES; MOLECULAR WEIGHT; ORGANIC ACIDS; RNA;

CONTROLLED INTRACELLULAR DELIVERY; KETALIZED POLYETHYLENIMINE; NONVIRAL GENE DELIVERY; RNA INTERFERENCE; TRANSFECTION;

NUCLEIC ACIDS;

NUCLEIC ACID; PLASMID DNA; POLYETHYLENEIMINE; SMALL INTERFERING RNA;

ANIMAL CELL; ARTICLE; CONTROLLED STUDY; CYTOPLASM; CYTOTOXICITY; DNA TRANSFECTION; GENE EXPRESSION; GENE LOCATION; MOUSE; NONHUMAN; PRIORITY JOURNAL; RNA INTERFERENCE;

4-AMINOPYRIDINE; ACIDS; ANIMALS; CELL NUCLEUS; CHROMATOGRAPHY, GEL; CYTOPLASM; GENE EXPRESSION; GREEN FLUORESCENT PROTEINS; HYDROGEN-ION CONCENTRATION; HYDROLYSIS; LUCIFERASES, FIREFLY; MICE; MICROSCOPY, CONFOCAL; MOLECULAR WEIGHT; NIH 3T3 CELLS; PARTICLE SIZE; PLASMIDS; POLYETHYLENEIMINE; RNA INTERFERENCE; RNA, SMALL INTERFERING; TRANSFECTION;

EID: 58549114410     PISSN: 01683659     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jconrel.2008.10.007     Document Type: Article

References (45)

1. Verma M., and Somia N. Gene therapy-promises, problems and prospects. Nature 389 (1997) 239-242
2. Verma M., and Weitzman M.D. Gene therapy: twenty-first century medicine. Annu. Rev. Biochem. 74 (2005) 711-738
3. Luo D., and Saltzman W.M. Synthetic DNA delivery systems. Nat. Biotechnol. 18 (2000) 33-37
4. Martinez J., Patkaniowska A., Urlaub H., Lührmann R., and Tuschl T. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell 110 (2002) 563-574
5. Zeng Y., and Cullen B.R. RNA is restricted to the cytoplasm. RNA 8 (2002) 855-860
6. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., and Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411 (2001) 494-498
7. Nykänen A., Haley B., and Zamore P.D. ATP requirements and small interfering RNA structure in the RNA interference pathway. Cell 107 (2001) 309-321
8. Bernstein E., Caudy A.A., Hammond S.M., and Hannon G.J. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 409 (2001) 363-366
9. Anderson W.F. Human gene therapy. Nature 392 (1998) 25-30
10. Thomas C.E., Ehrhardt A., and Kay M.A. Progress and problems with the use of viral vectors for gene therapy. Nat. Rev. Genet. 4 (2003) 346-358
11. Zanta M.A., Belguise-Valladier P., and Behr J.-P. Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl. Acad. Sci. U. S. A. 96 (1999) 91-96
12. Brandén L.J., Mohamed A.J., and Smith C.I.E. A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nat. Biotechnol. 17 (1999) 784-787
13. Nigg E.A. Nucleocytoplasmic transport: signals, mechanisms and regulation. Nature 386 (1997) 779-787
14. Goldfarb D.S., Gariepy J., Schoolnik G., and Kornberg R.D. Synthetic peptides as nuclear localization signals. Nature 322 (1986) 641-644
15. Mann A., Richa R., and Ganguli M. DNA condensation by poly-l-lysine at the single molecule level: role of DNA concentration and polymer length. J. Control. Release 125 (2008) 252-262
16. Niidome T., Ohmori N., Ichinose A., Wada A., Mihara H., Hirayama T., and Aoyagi H. Binding of cationic alpha-helical peptides to plasmid DNA and their gene transfer abilities into cells. J. Biol. Chem. 272 (1997) 15307-15312
17. Volcke C., Pirotton S., Grandfils Ch., Humbert C., Thiry P.A., Ydens I., Dubois P., and Raes M. Influence of DNA condensation state on transfection efficiency in DNA/polymer complexes: an AFM and DLS comparative study. J. Biotechnol. 125 (2006) 11-21
18. Wiethoff C.M., and Middaugh C.R. Barriers to nonviral gene delivery. J. Pharm. Sci. 92 (2003) 203-217
19. Blumenthal R., Clague M.J., Durell S.R., and Epand R.M. Membrane fusion. Chem. Rev. 103 (2003) 53-69
20. Boussif O., Lezoualc'h F., Zanta M.A., Mergny M.D., Scherman D., Demeneix B., and Behr J.-P. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethyleneimine. Proc. Natl. Acad. Sci. U. S. A. 92 (1995) 7297-7301
21. Godbey W.T., Wu K.K., and Mikos G.A. Poly(ethylenimine) and its role in gene delivery. J. Control. Release 60 (1999) 149-160
22. Akinc A., Thomas M., Klibanov A.M., and Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J. Gene Med. 7 (2005) 657-663
23. Plank C., Oberhauser B., Mechtler K., Koch C., and Wagner E. The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene transfer systems. J. Biol. Chem. 269 (1994) 12918-12924
24. Liu G., Li D., Pasumarthy M.K., Kowalczyk T.H., Gedeon C.R., Hyatt S.L., Payne J.M., Miller T.J., Brunovskis P., Fink T.L., Muhammad O., Moen R.C., Hanson R.W., and Cooper M.J. Nanoparticles of compacted DNA transfect postmitotic cells. J. Biol. Chem. 278 (2003) 32578-32586
25. Lukacs G.L., Haggie P., Seksek O., Lechardeur D., Freedman N., and Verkman A.S. Size-dependent DNA mobility in cytoplasm and nucleus. J. Biol. Chem. 275 (2000) 1625-1629
26. Huang K., Voss B., Kumar D., Hamm H.E., and Harth E. Dendritic molecular transporters provide control of delivery to intracellular compartments. Bioconjug. Chem. 18 (2007) 403-409
27. Jones N.A., Hill I.R.C., Stolnik S., Bignotti S.F., Davis S.S., and 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 1517 (2000) 1-18
28. Chen D.J., Majors B.S., Zelikin A., and Putnam D. Structure-function relationships of gene delivery vectors in a limited polycation library. J. Control. Release 103 (2005) 273-283
29. Doody A.M., Korley J.N., Dang K.P., Zawaneh P.N., and Putnam D. Characterizing the structure/function parameter space of hydrocarbon-conjugated branched polyethylenimine for DNA delivery in vitro. J. Control. Release 116 (2006) 227-237
30. Mao S., Neu M., Germershaus O., Merkel O., Sitterberg J., Bakowsky U., and Kissel T. 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. 17 (2006) 1209-1218
31. Hou S., Yang K., Yao Y., Liu Z., Feng X., Wang R., Yang Y., and Wang C. DNA condensation induced by a cationic polymer studied by atomic force microscopy and electrophoresis assay. Colloids Surf. B Biointerfaces 62 (2008) 151-156
32. Stanić V., Arntz Y., Richard D., Affolter C., Nguyen I., Crucifix C., Schultz P., Baehr C., Frisch B., and Ogier J. Filamentous condensation of DNA induced by pegylated poly-l-lysine and transfection efficiency. Biomacromolecules 9 (2008) 2048-2055
33. Shim M.S., and Kwon Y.J. Controlled delivery of plasmid DNA and siRNA to intracellular targets using ketalized polyethylenimine. Biomacromolecules 9 (2008) 444-455
34. Kwon Y.J., Yu H., and Peng C.-A. Enhanced retroviral transduction of 293 cells cultured on liquid-liquid interfaces. Biotechnol. Bioeng. 72 (2001) 331-338
35. Ogris M., Steinlein P., Kursa M., Mechtler K., Kircheis R., and Wagner E. The size of DNA/transferrin-PEI complexes is an important factor for gene expression in cultured cells. Gene Ther. 5 (1998) 1425-1433
36. Dunlap D.D., Maggi A., Soria M.R., and Monaco L. Nanoscopic structure of DNA condensed for gene delivery. Nucleic Acids Res. 25 (1997) 3095-3101
37. Hyrup B., and Nielsen P.E. Peptide nucleic acids (PNA): synthesis, properties and potential applications. Bioorg. Med. Chem. 4 (1996) 5-23
38. Dindo D., Dahm F., Szulc Z., Bielawska A., Obeid L.M., Hannun Y.A., Graf R., and Clavien P.-A. Cationic long-chain ceramide LCL-30 induces cell death by mitochondrial targeting in SW403 cells. Mol. Cancer Ther. 5 (2006) 1520-1529
39. Hartmann L., Häfele S., Peschka-Süss R., Antonietti M., and Börner H.G. Tailor-made poly(amidoamine)s for controlled complexation and condensation of DNA. Chem. Eur. J. 14 (2008) 2025-2033
40. Germershaus O., Mao S., Sitterberg J., Bakowsky U., and Kissel T. Gene delivery using chitosan, trimethyl chitosan or polyethylenglycol-graft-trimethyl chitosan block copolymers: establishment of structure-activity relationships in vitro. J. Control. Release 125 (2008) 145-154
41. Geall A.J., Al-Hadithi D., and Blagbrough I.S. Efficient calf thymus DNA condensation upon binding with novel bile acid polyamine amides. Bioconjug. Chem. 13 (2002) 481-490
42. Mahler H.R., and Mehrotra B.D. The interaction of nucleic acids with diamines. Biochim. Biophys. Acta 68 (1963) 211-233
43. Yoshikawa Y., and Yoshikawa K. Diaminoalkanes with an odd number of carbon atoms induce compaction of a single double-stranded DNA chain. FEBS Lett. 361 (1995) 277-281
44. Wang X.L., Jensen R., and Lu Z.R. A novel environment-sensitive biodegradable polydisulfide with protonatable pendants for nucleic acid delivery. J. Control. Release 120 (2007) 250-258
45. Bloomfield V.A. DNA condensation by multivalent cations. Biopolymers 44 (1997) 269-282