Polyethylenimine as a promising vector for targeted siRNA delivery

Current Clinical Pharmacology

Volumn 7, Issue 2, 2012, Pages 121-130

  Nimesh, Surendra ^a  

^a CLIN RESEARCH INSTITUTE OF MONTREAL   (Canada)

Author keywords

Cationic; Degradation; Delivery; Nanoparticles; Polyethylenimine; Polymer; siRNA

Indexed keywords

ALGINIC ACID; AZIRIDINE; BETA AMINOPROPIONAMIDE; CHITOSAN; CHLOROTOXIN; CHOLANIC ACID; CHOLANIC ACID DERIVATIVE; COPOLYMER; DNA; HYALURONIC ACID; LIPOFECTIN; MACROGOL; MALTOHEPTAOSE; MALTOSE; MALTOTRIOSE; NANOCOMPOSITE; NANOPARTICLE; OLIGOSACCHARIDE; POLYANION; POLYETHYLENEIMINE; PROPIONAMIDE DERIVATIVE; PULLULAN; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; SUPERPARAMAGNETIC IRON OXIDE NANOPARTICLE; TOXIN; TYROSINE; UNCLASSIFIED DRUG;

BIOCOMPATIBILITY; DERIVATIZATION; DISULFIDE BOND; DRUG DISTRIBUTION; DRUG DOSAGE FORM COMPARISON; DRUG FORMULATION; ENZYMATIC DEGRADATION; GENE SILENCING; HUMAN; MOLECULAR STABILITY; MOLECULAR WEIGHT; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PARTICLE SIZE; PRIORITY JOURNAL; REVIEW; RING OPENING METATHESIS POLYMERIZATION; RNA INTERFERENCE; ZETA POTENTIAL;

EID: 84860502021     PISSN: 15748847     EISSN: None     Source Type: Journal    
DOI: 10.2174/157488412800228857     Document Type: Review

References (63)

1. Fire A, Xu SQ, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998; 391(6669): 806-11.
2. Elbashir SM, Harborth J, Lendeckel W, Yalcin A, Weber K, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 411(6836): 494-8.
3. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001; 409(6818): 363-6.
4. Hammond SM, Bernstein E, Beach D, Hannon GJ. An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature 2000; 404(6775): 293-6.
5. Liu J, Carmell MA, Rivas FV, et al. Argonaute2 is the catalytic engine of mammalian RNAi. Science 2004; 305(5689): 1437-41.
6. Collins RE, Cheng X. Structural and biochemical advances in mammalian RNAi. J Cell Biochem 2006; 99(5): 1251-66.
7. Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat Rev Genet 2007; 8(3): 173-84.
8. Leung RKM, Whittaker PA. RNA interference: From gene silencing to gene-specific therapeutics. Pharmacol Ther 2005; 107(2): 222-39.
9. Xu D, McCarty D, Fernandes A, Fisher M, Samulski RJ, Juliano RL. Delivery of MDR1 small interfering RNA by selfcomplementary recombinant adeno-associated virus vector. Mol Ther 2005; 11(4): 523-30.
10. Zaiss AK, Muruve DA. Immune responses to adeno-associated virus vectors. Curr Gene Ther 2005; 5(3): 323-31.
11. Jeong JH, Kim SW, Park TG. Molecular design of functional polymers for gene therapy. Prog Polym Sc 2007; 32(11): 1239-74.
12. Boussif O, Lezoualc'h F, Zanta MA, 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): 7297-301.
13. Neu M, Fischer D, Kissel T. Recent advances in rational gene transfer vector design based on poly(ethylene imine) and its derivatives. J Gene Med 2005; 7(8): 992-1009.
14. Jones GD, Langsjoen A, Neumann SMMC, Zomlefer J. The Polymerization of Ethylenimine. J Org Chem 1944; 09(2): 125-47.
15. Brissault B, Kichler A, Guis C, Leborgne C, Danos O, Cheradame H. Synthesis of linear polyethylenimine derivatives for DNA transfection. Bioconjug Chem 2003; 14(3): 581-7.
16. von Harpe A, Petersen H, Li Y, Kissel T. Characterization of commercially available and synthesized polyethylenimines for gene delivery. J Control Release 2000; 69(2): 309-22.
17. Thomas M, Lu JJ, Ge Q, Zhang C, Chen J, Klibanov AM. Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. Proc Natl Acad Sci U S A 2005; 102(16): 5679-84.
18. Thomas M, Klibanov AM. Enhancing polyethylenimine's delivery of plasmid DNA into mammalian cells. Proc Natl Acad Sci U S A 2002; 99(23): 14640-5.
19. Akinc A, Thomas M, Klibanov AM, Langer R. Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J Gene Med 2005; 7(5): 657-63.
20. Goula D, Benoist C, Mantero S, Merlo G, Levi G, Demeneix BA. Polyethylenimine-based intravenous delivery of transgenes to mouse lung. Gene Ther 1998; 5(9): 1291-5.
21. Abdallah B, Hassan A, Benoist C, Goula D, Behr JP, Demeneix BA. A powerful nonviral vector for in vivo gene transfer into the adult mammalian brain: polyethylenimine. Hum Gene Ther 1996; 7(16): 1947-54.
22. Erbacher P, Bettinger T, Brion E, et al. Genuine DNA/polyethylenimine (PEI) complexes improve transfection properties and cell survival. J Drug Target 2004; 12(4): 223-36.
23. Kunath K, von Harpe A, Fischer D, 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): 113-25.
24. Fischer D, Bieber T, Li Y, Elsasser HP, 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(8): 1273-9.
25. Godbey WT, Wu KK, Mikos AG. Size matters: molecular weight affects the efficiency of poly(ethylenimine) as a gene delivery vehicle. J Biomed Mater Res 1999; 45(3): 268-75.
26. Ogris M, Brunner S, Schuller S, Kircheis R, Wagner E. PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther 1999; 6(4): 595-605.
27. Wightman L, Kircheis R, Rossler V, et al. Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J Gene Med 2001; 3(4): 362-72.
28. Grayson AC, Doody AM, Putnam D. Biophysical and structural characterization of polyethylenimine-mediated siRNA delivery in vitro. Pharm Res 2006; 23(8): 1868-76.
29. Spagnou S, Miller AD, Keller M. Lipidic carriers of siRNA: differences in the formulation, cellular uptake, and delivery with plasmid DNA. Biochemistry 2004; 43(42): 13348-56.
30. Kang J-H, Tachibana Y, Kamata W, Mahara A, Harada-Shiba M, Yamaoka T. Liver-targeted siRNA delivery by polyethylenimine (PEI)-pullulan carrier. Bioorg Med Chem 2010; 18(11): 3946-50.
31. 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(7): 1121-31.
32. Jevprasesphant R, Penny J, Jalal R, Attwood D, McKeown NB, D'Emanuele A. The influence of surface modification on the cytotoxicity of PAMAM dendrimers. Int J Pharm 2003; 252(1-2): 263-6.
33. Jiang G, Park K, Kim J, et al. Hyaluronic acid-polyethyleneimine conjugate for target specific intracellular delivery of siRNA. Biopolymers 2008; 89(7): 635-42.
34. Duan Y, Yang C, Zhang Z, Liu J, Zheng J, Kong D. Poly(ethylene glycol)-grafted polyethylenimine modified with G250 monoclonal antibody for tumor gene therapy. Hum Gene Ther 2010; 21(2): 191-8.
35. Godbey WT, Wu KK, Hirasaki GJ, Mikos AG. Improved packing of poly(ethylenimine)/DNA complexes increases transfection efficiency. Gene Ther 1999; 6(8): 1380-8.
36. Godbey WT, Wu KK, Mikos AG. Poly(ethylenimine)-mediated gene delivery affects endothelial cell function and viability. Biomaterials 2001; 22(5): 471-80.
37. Breunig M, Hozsa C, Lungwitz U, et al. Mechanistic investigation of poly(ethylene imine)-based siRNA delivery: disulfide bonds boost intracellular release of the cargo. J Control Release 2008; 130(1): 57-63.
38. Oskuee RK, Philipp A, Dehshahri A, Wagner E, Ramezani M. The impact of carboxyalkylation of branched polyethylenimine on effectiveness in small interfering RNA delivery. J Gene Med 2010; 12(9): 729-38.
39. Philipp A, Zhao X, Tarcha P, Wagner E, Zintchenko A. Hydrophobically modified oligoethylenimines as highly efficient transfection agents for siRNA delivery. Bioconjug Chem 2009; 20(11): 2055-61.
40. Kim WJ, Chang C-W, Lee M, Kim SW. Efficient siRNA delivery using water soluble lipopolymer for anti-angiogenic gene therapy. J Control Release 2007; 118(3): 357-63.
41. Wu Y, Wang W, Chen Y, et al. The investigation of polymer-siRNA nanoparticle for gene therapy of gastric cancer in vitro. Int J Nanomedicine 2010; 5: 129-36.
42. Veiseh O, Kievit FM, Fang C, et al. Chlorotoxin bound magnetic nanovector tailored for cancer cell targeting, imaging, and siRNA delivery. Biomaterials 2010; 31(31): 8032-42.
43. Creusat G, Rinaldi AS, Weiss E, et al. Proton sponge trick for pH-sensitive disassembly of polyethylenimine-based siRNA delivery systems. Bioconjug Chem 2010; 21(5): 994-1002.
44. Patnaik S, Aggarwal A, Nimesh S, et al. PEI-alginate nanocomposites as efficient in vitro gene transfection agents. J Control Release 2006; 114(3): 398-409.
45. Nimesh S, Chandra R. Polyethylenimine nanoparticles as an efficient in vitro siRNA delivery system. Eur J Pharm Biopharm 2009; 73(1): 43-9.
46. Biswal B, Debata N, Verma R. Development of a targeted siRNA delivery system using FOL-PEG-PEI conjugate. Mol Biol Rep 2010; 37(6): 2919-26.
47. Huh MS, Lee S-Y, Park S, et al. Tumor-homing glycol chitosan/polyethylenimine nanoparticles for the systemic delivery of siRNA in tumor-bearing mice. J Control Release 2010; 144(2): 134-43.
48. Werth S, Urban-Klein B, Dai L, 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(2): 257-70.
49. Hobel S, Prinz R, Malek A, et al. 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 2008; 70(1): 29-41.
50. Lee SY, Huh MS, Lee S, et al. Stability and cellular uptake of polymerized siRNA (poly-siRNA)/polyethylenimine (PEI) complexes for efficient gene silencing. J Control Release 2010; 141(3): 339-46.
51. Geisbert TW, Hensley LE, Kagan E, et al. Postexposure protection of guinea pigs against a lethal ebola virus challenge is conferred by RNA interference. J Infect Dis 2006; 193(12): 1650-7.
52. Segura T, Schmokel H, Hubbell JA. RNA interference targeting hypoxia inducible factor 1alpha reduces post-operative adhesions in rats. J Surg Res 2007; 141(2): 162-70.
53. Ge Q, Filip L, Bai A, Nguyen T, Eisen HN, Chen J. Inhibition of influenza virus production in virus-infected mice by RNA interference. Proc Natl Acad Sci U S A 2004; 101(23): 8676-81.
54. Lively TN, Kossen K, Balhorn A, et al. Effect of chemically modified IL-13 short interfering RNA on development of airway hyperresponsiveness in mice. J Allergy Clin Immunol 2008; 121(1): 88-94.
55. Thomas M, Lu JJ, Ge Q, Zhang C, Chen J, Klibanov AM. Full deacylation of polyethylenimine dramatically boosts its gene delivery efficiency and specificity to mouse lung. Proc Natl Acad Sci U S A 2005; 102(16): 5679-84.
56. Tarcha PJ, Pelisek J, Merdan T, et al. Synthesis and characterization of chemically condensed oligoethylenimine containing beta-aminopropionamide linkages for siRNA delivery. Biomaterials 2007; 28(25): 3731-40.
57. Jere D, Xu CX, Arote R, Yun CH, Cho MH, Cho CS. Poly(beta-amino ester) as a carrier for si/shRNA delivery in lung cancer cells. Biomaterials 2008; 29(16): 2535-47.
58. Xu CX, Jere D, Jin H, et al. Poly(ester amine)-mediated, aerosoldelivered Akt1 small interfering RNA suppresses lung tumorigenesis. Am J Respir Crit Care Med 2008; 178(1): 60-73.
59. Petersen H, Fechner PM, Martin AL, et al. Polyethylenimine-graftpoly(ethylene glycol) copolymers: influence of copolymer block structure on DNA complexation and biological activities as gene delivery system. Bioconjug Chem 2002; 13(4): 845-54.
60. Kunath K, von Harpe A, Petersen H, et al. The structure of PEG-modified poly(ethylene imines) influences biodistribution and pharmacokinetics of their complexes with NF-kappaB decoy in mice. Pharm Res 2002; 19(6): 810-7.
61. Malek A, Merkel O, Fink L, Czubayko F, Kissel T, Aigner A. In vivo pharmacokinetics, tissue distribution and underlying mechanisms of various PEI(-PEG)/siRNA complexes. Toxicol Appl Pharmacol 2009; 236(1): 97-108.
62. Beyerle A, Hobel S, Czubayko F, et al. In vitro cytotoxic and immunomodulatory profiling of low molecular weight polyethylenimines for pulmonary application. Toxicol In Vitro 2009; 23(3): 500-8.
63. Akhtar S, Benter I. Toxicogenomics of non-viral drug delivery systems for RNAi: potential impact on siRNA-mediated gene silencing activity and specificity. Adv Drug Deliv Rev 2007; 59(2-3): 164-82.