Stability and cellular uptake of polymerized siRNA (poly-siRNA)/polyethylenimine (PEI) complexes for efficient gene silencing

Journal of Controlled Release

Volumn 141, Issue 3, 2010, Pages 339-346

  Lee, Seung Young ^a   Huh, Myung Sook ^a,b   Lee, Seulki ^a   Lee, So Jin ^a,c   Chung, Hyunjin ^a,c   Park, Jae Hyung ^b   Oh, Yu Kyoung ^c   Choi, Kuiwon ^a   Kim, Kwangmeyung ^a   Kwon, Ick Chan ^a  

^a Korea Institute of Science and Technology   (South Korea)

^b Kyung Hee University   (South Korea)

^c Seoul National University   (South Korea)

Author keywords

Electrostatic complexes; Gene silencing; Polyethylenimine; Polymerized siRNA; SiRNA

Indexed keywords

ANTISENSE; CELLULAR UPTAKE; CELLULAR UPTAKE EFFICIENCY; CLINICAL APPLICATION; DISULFIDE BONDS; DITHIOLS; ELECTROSTATIC COMPLEXES; ELECTROSTATIC INTERACTIONS; GENE DELIVERY SYSTEMS; GENE SILENCING; HUMAN DISEASE; INTRACELLULAR UPTAKE; LOW MOLECULAR WEIGHT; MELANOMA CELLS; MODIFIED REDS; NANO-SIZED; POLYANIONICS; POLYANIONS; POLYETHYLENIMINES; POSSIBLE SOLUTIONS; SIRNA DELIVERY; SMALL INTERFERING RNA (SIRNA); THERAPEUTIC APPLICATION; THERAPEUTIC STRATEGY;

COMPETITION; COMPLEXATION; ELECTROSTATICS; GENE TRANSFER;

RNA;

DITHIOL DERIVATIVE; OLIGOMER; POLYANION; POLYETHYLENEIMINE; RED FLUORESCENT PROTEIN; SMALL INTERFERING RNA;

5' UNTRANSLATED REGION; ANIMAL CELL; ARTICLE; CELL TRANSPORT; COMPLEX FORMATION; CONTROLLED STUDY; DISULFIDE BOND; DRUG DELIVERY SYSTEM; DRUG DOSAGE FORM COMPARISON; DRUG STABILITY; FRACTIONATION; GENE SILENCING; IN VITRO STUDY; MELANOMA CELL; MOLECULAR WEIGHT; MOUSE; NONHUMAN; PHYSICAL CHEMISTRY; POLYMERIZATION; PRIORITY JOURNAL; RAT; RNA CLEAVAGE; RNA DEGRADATION; RNA INTERFERENCE; RNA STABILITY;

BASE SEQUENCE; DISULFIDES; FLUORESCENT DYES; GENE SILENCING; LUMINESCENT PROTEINS; MOLECULAR SEQUENCE DATA; MOLECULAR WEIGHT; POLYETHYLENEIMINE; POLYMERS; RNA, SMALL INTERFERING; STATIC ELECTRICITY;

EID: 75549089094     PISSN: 01683659     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.jconrel.2009.10.007     Document Type: Article

References (39)

1. Dykxhoorn D.M., Palliser D., Lieberman J. The silent treatment: siRNAs as small molecule drugs. Gene Ther. 2006, 13:541-552.
2. Dykxhoorn D.M., Lieberman J. Running interference: prospects and obstacles to using small interfering RNAs as small molecule drugs. Annu. Rev. Biomed. Eng. 2006, 8:377-402.
3. McManus M.T., Sharp P.A. Gene silencing in mammals by small interfering RNAs. Nat. Rev. Genet. 2002, 3:737-747.
4. Shiver J.W., Fu T.M., Chen L., Casimiro D.R., Davies M.E., Evans R.K., et al. Replication-incompetent adenoviral vaccine vector elicits effective anti-immunodeficiency-virus immunity. Nature 2002, 415:331-335.
5. Song E., Lee S.K., Wang J., Ince N., Ouyang N., Min J., Chen J., Shankar P., Lieberman J. RNA interfence targeting Fas protects mice from fulminant hepatitis. Nat. Med. 2003, 9:347-351.
6. Martinez J., Patkaniowska A., Urlaub H., Lührmann R., Tuschl T. Single-stranded antisense siRNA gudie target RNAs guide target RNA cleavage in RNAi. Cell 2002, 110:563-574.
7. Elbashir S.M., Harborth J., Lendeckel W., Yalcin A., Weber K., Tuschl T. Duplexes of 21-nucleogide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001, 411:494-498.
8. Hannon G.J. RNA interference. Nature 2002, 418:224-251.
9. Caplan N.J., Parrish S., Imani F., Morgan R.A. Specific inhibition of gene expression by small double-stranded RNAs in vertebrate and vertebrate systems. Proc. Natl. Acad. Sci. U.S.A. 2001, 98:9742-9747.
10. Lu P.Y., Xie F., Woodle M.C. In vivo application of RNA interference: from functional genomics to therapeutics. Adv. Genet. 2005, 54:117-142.
11. Oishi M., Nagasaki Y., Itaka K., Nishiyama N., Kataoka K. Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile beta-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. Akhtar S., Benter I.F. Nonviral delivery of synthetic siRNAs in vivo. J. Clin. Invest. 2007, 117(12):3623-3632.
13. Park T.G., Jeong J.H., Kim S.W. Current status of polymeric gene delivery systems. Adv. Drug Deliv. Rev. 2006, 58(4):467-486.
14. Layzer J.M., McCaffrey A.P., Tanner A.K., Huang Z., Kay M.A., Sullenger B.A. In vivo activity of nuclease-resistant siRNAs. RNA 2004, 10:766-771.
15. Hassani Z., Lemkine G.F., Erbacher P., Palmier K., Alfama G., Giovannangeli C., Behr J.P., Demeneix B.A. Lipid-mediated siRNA delivery down-regulates exogenous gene expression in the mouse brain at picomolar levels. J. Gene Med. 2005, 7:198-207.
16. Landen C.N., Chavez-Reves A, Bucana C., Schmandt R., Deavers M.T., Lopez-Berestein G., Sood A.K. Terapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery. Cancer Res. 2005, 65:6910-6918.
17. Zhang S., Zhao B., Jiang H., Wang B., Ma B. Cationic lipids and polymers mediated vectors for delivery of siRNA. J. Control. Release 2007, 123:1-10.
18. Takeshita F., Minakuchi Y., Nagahara S., Honma K., Sasaki H., Hiral K., Teratani T., Namatame N., Yamamoto Y., Hanai K., Kato T., Sano A., Ochiva T. Efficient delivery of small interfering RNA to bone-metastatic tumors by using atelocollagen in vivo. Proc. Natl. Acad. Sci. U.S.A. 2005, 102:12177-12180.
19. Matsumoto G., Kushibiki T., Kinoshita Y., Lee U., Omi Y., Kubota E., Tabata Y. Cationized gelatin delivery of a plasmid DNA expressing small interference RNA for VEGF inhibits murine squamous cell carcinoma. Cancer Sci. 2006, 97:313-321.
20. Chiu Y.L., Ali A., Chu C.Y., Cao H., Rana T.M. Visualizing a correlation between siRNA localization, cellular uptake, and RNAi in living cells. Chem. Biol. 2004, 11:1165-1175.
21. Kim K., Lee M., Park H., Kim J.H., Kim S., Chung H., Choi K., Kim I.S., Seong B.L., Kwon I.C. Cell-permeable and biocompatible polymeric nanoparticles for apoptosis imaging. J. Am. Chem. Soc. 2006, 128:3490-3491.
22. Kim J.H., Lee S., Park K., Nam H.Y., Jang S.Y., Youn I., Kim K., Jeon H., Park R.W., Kim I.S., Choi K., Kwon I.C. Protein-phosphorylation-responsive polymeric nanoparticles for imaging protein kinase activities in single living cells. Angew. Chem. Int. Ed. 2007, 46:5779-5782.
23. Ko Y.T., Kale A., Hartner W.C., Papahadjopoulos-Sternberg B., Torchilin V.P. Self-assembling micelle-like nanoparticles based on phospholipids-polyethyleneimine conjugates for systemic gene delivery. J. Control. Release 2009, 133:132-138.
24. Deshpands M.C., Prausnitz M.R. Synergistic effect of ultrasound and PEI on DNA transfection in vitro. J. Control. Release 2007, 118:126-135.
25. Breunig M., Hozsa C., Lungwitz U., Watanabe K., Umeda I., Kato H., Goepferich A. Mechanistic investigation of poly(ethyleneimine)-based siRNA delivery: disulfide bonds boost intracellular release of the cargo. J. Control. Release 2008, 130:57-63.
26. Urban-Klein B., Werth S., Abuharbeid S., Czubayko F., Aigner A. RNAi-mediated gene-targeting through systemic application of polyehtylenimine (PEI)-complexed siRNA in vivo. Gene Ther. 2005, 12:461-466.
27. Merdan T., Kunath K., Fischer D., Kopecek J., Kissel T. Intracellular processing of poly(ethylene imine)/ribozyme complexes can be observed in living cells by using confocal laser scanning microscopy and inhibitor experiments. Pharm. Res. 2002, 19:140-146.
28. Akine A., Thomas M., Klibanov A.M., Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J. Gene Med. 2005, 7:657-663.
29. 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. U.S.A. 2007, 104:16050-16055.
30. Jere D., Kim J.-E., Arote R., Jiang H.-L., Kim Y.-K., Choi Y.J., Yun C.-H., Cho M.-H., Cho C.-S. Akt1 silencing efficiencies in lung cancer cells by sh/si/ssiRNA transfection using a reductable polyspermine carrier. Biomaterials 2009, 30:1635-1647.
31. Derfus A.M., Chen A.A., Min D.-H., Ruoslahti E., Bhatia S.N. Targeted quantum dot conjugates for siRNA delivery. Bioconjug. Chem. 2007, 18:1391-1396.
32. Kim S.H., Jeong J.H., Lee S.H., Kim S.W., Park T.G. PEG conjugated VEGF siRNA for anti-angiogenic gene therapy. J. Control. Release 2006, 116:123-129.
33. Jeong J.H., Mok H., Oh Y.-K., Park T.G. siRNA conjugate delivery systems. Bioconjug. Chem. 2009, 20:5-14.
34. Bonnet M.-E., Erbacher P., Bolcato-Bellemin A.-L. Systemic delivery of DNA or siRNA mediated by linear polyethyleimine (L-PEI) dose not induce an inflammatory response. Pharm. Res. 2008, 25:2972-2982.
35. Mok H., Park T.G. Self-crosslinked and reducible fusogenic peptides for intracellular delivery of siRNA. Biopolymers 2008, 89:881-888.
36. Kim S.H., Jeong J.H., Lee S.H., Kim S.W., Park T.G. PEG conjugated VEGF siRNA for anti-angiogenic gene therapy. J. Control. Release 2006, 116:123-129.
37. Kim S.W., Ogawa T., Tabata Y., Nishimura I. Efficacy and cytotoxicity of cationic-agent-mediated nonviral gene transfer into osteoblasts. J. Biomed. Mater. Res. 2004, 71:308-315.
38. Kunath K., Harpe A.V., Fischer D., Petersen H., Bickel U. K. Voigt, T. Kissel, Low-molecular-weight polyehtylenimine as a non-viral vector for DNA delivery: comparison of physicochemical properties, transfection efficiency and in vivo distribution with high-molecular-weight polyehtylenimine. J. Control. Release 2003, 89:113-125.
39. Florea B.I., Meaney C., Junginger H.E., Borchard G. Transfection efficiency and toxicity of polyethylenimine in differentiated Calu-3 and nondifferentiated COS-1 cell cultures. AAPS PharmSci. 2002, 4:1-11.