Metabolically stabilized long-circulating PEGylated polyacridine peptide polyplexes mediate hydrodynamically stimulated gene expression in liver

Gene Therapy

Volumn 18, Issue 1, 2011, Pages 23-37

  Fernandez, C A ^a   Baumhover, N J ^a   Duskey, J T ^a   Khargharia, S ^a   Kizzire, K ^a   Ericson, M D ^a   Rice, K G ^a  

^a UNIVERSITY OF IOWA   (United States)

Author keywords

biodistribution; DNA pharmacokinetics; gene delivery; gene expression; peptide

Indexed keywords

ACRIDINE; ARGININE; CYSTINE; DEOXYRIBONUCLEASE; GLUTAMIC ACID; LEUCINE; LUCIFERASE; LYSINE; MACROGOL; PEGYLATED POLYACRIDINE PEPTIDE; PEPTIDE; PLASMID DNA; UNCLASSIFIED DRUG;

ANIMAL TISSUE; ARTICLE; BINDING AFFINITY; CONTROLLED STUDY; DNA METABOLISM; DNA TRANSFECTION; GENE EXPRESSION; HALF LIFE TIME; HYDRODYNAMICS; IN VIVO GENE TRANSFER; INTERCALATION COMPLEX; LIVER; MOLECULAR STABILITY; MOLECULAR WEIGHT; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN EXPRESSION;

ACRIDINES; ANIMALS; DNA; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; HALF-LIFE; HYDRODYNAMICS; LIVER; MICE; PEPTIDES; PLASMIDS; POLYETHYLENE GLYCOLS; TRANSFECTION;

MUS;

EID: 78650989655     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2010.117     Document Type: Article

References (50)

1. Burke RS, Pun SH. Extracellular barriers to in vivo PEI and PEGylated PEI polyplex-mediated gene delivery to the liver. Bioconjug Chem 2008; 19: 693-704.
2. Boeckle S, Von Gersdorff K, van der Piepen S, Culmsee C, Wagner E, Ogris M. Purification of polyethylenimine polyplexes highlights the role of free polycations in gene transfer. J Gene Med 2004; 6: 1102-1111.
3. Rudolph C, Schillinger U, Plank C, Gessner A, Nicklaus P, Muller R, et al. Nonviral gene delivery to the lung with copolymer-protected and transferrin-modified polyethyleni-mine. Biochim Biophys Acta 2002; 1573: 75-83.
4. Guillaume C, Delepine P, Droal C, Montier T, Tymen G, Claude F. Aerosolization of cationic lipid-DNA complexes: lipoplex characterization and optimization of aerosol delivery conditions. Biochem Biophys Res Commun 2001; 286: 464-471.
5. Mahato RI, Anwer K, Tagliaferri F, Meaney C, Leonard P, Wadhwa MS, et al. Biodistribu-tion and gene expression of lipid/plasmid complexes after systemic administration. Hum Gene Ther 1998; 9: 2083-2099.
6. Li S, Rizzo MA, Bhattacharya S, Huang L. Characterization of cationic lipid- protamine-DNA (LPD) complexes for intravenous gene delivery. Gene Therapy 1998; 5: 930-937.
7. Liu F, Qi H, Huang L, Liu D. Factors controlling the efficiency of cationic lipid-mediated transfection in vivo via intravenous administration. Gene Therapy 1997; 4: 517-523.
8. Felgner PL, Tsai YJ, Sukhu L, Wheeler CJ, Manthorpe M, Marshall J, et al. Improved cationic lipid formulations for in vivo gene therapy. Ann N Y Acad Sci 1995; 772: 126-139.
9. Al-Dosari MS, Knapp JE, Liu D, Leaf Huang M, Cha EW. Hydrodynamic delivery. In: Adv Genet, vol. 54. Academic Press, 2005, pp 65-82.
10. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Therapy 1999; 6: 1258-1266.
11. Molnar MJ, Gilbert R, Lu Y, Liu AB, Guo A, Larochelle N, et al. Factors influencing the efficacy, longevity, and safety of electroporation-assisted plasmid-based gene transfer into mouse muscles. Mol Ther 2004; 10: 447-455.
12. Dean DA, Machado-Aranda D, Blair-Parks K, Yeldandi AV, Young JL. Electroporation as a method for high-level nonviral gene transfer to the lung. Gene Therapy 2003; 10: 1608-1615.
13. Fattori E, La Monica N, Ciliberto G, Toniatti C. Electro-gene-transfer: a new approach for muscle gene delivery. Somat Cell Mol Genet 2002; 27: 75-83.
14. Li S, MacLaughlin FC, Fewell JG, Gondo M, Wang J, Nicol F, et al. Muscle-specific enhancement of gene expression by incorporation of SV40 enhancer in the expression plasmid. Gene Therapy 2001; 8: 494-497.
15. Liu F, LH. A syringe electrode device for simultaneous injection of DNA and electrotransfer. Mol Ther 2002; 5: 323-328.
16. Heyes J, Palmer L, Chan K, Giesbrecht C, Jeffs L, MacLachlan I. Lipid encapsulation enables the effective systemic delivery of polyplex plasmid DNA. Mol Ther 2007; 15: 713-720.
17. 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 Therapy 1999; 6: 595-605.
18. Blessing T, Kursa M, Holzhauser R, Kircheis R, Wagner E. Different strategies for formation of PEGylated EGF-conjugated PEI/DNA complexes for targeted gene delivery. Bioconj Chem 2001; 12: 529-537.
19. Mannisto M, Vanderkerken S, Toncheva V, Elomaa M, Ruponen M, Schacht E, et al. Structure-activity relationships of poly(L-lysines): effects of pegylation and molecular shape on physicochemical and biological properties in gene delivery. J Control Rel 2002; 83: 169-182.
20. Kwok KY Park Y, Yongsheng Y, McKenzie DL, Rice KG. In vivo gene transfer using sulfhydryl crosslinked PEG-peptide/glycopeptide DNA co-condensates. J Pharm Sci 2003; 92: 1174-1185.
21. Yang Y, Park Y, Man S, Liu Y, Rice KG. Cross-linked low molecular weight glycopeptide-mediated gene delivery: relationship between DNA metabolic stability and the level of transient gene expression in vivo. J Pharm Sci 2001; 90: 2010-2022.
22. Collard WT, Yang Y, Kwok KY, Park Y, Rice KG. Biodistribution, metabolism, and in vivo gene expression of low molecular weight glycopeptide polyethylene glycol peptide DNA co-condensates. J Pharm Sci 2000; 89: 499-512.
23. Nishikawa M, Yamauchi M, Morimoto K, Ishida E, Takakura Y, Hashida M. Heptocyte-targeted in vivo gene expression by intravenous injection of plasmid DNA complexed with synthetic multi-functional gene delivery system. Gene Therapy 2000; 7: 548-555.
24. Schuster MJ, Wu GY, Walton CM, Wu CH. Multicomponent DNA carrier with a vesicular stomatitis virus G-peptide greatly enhances liver-targeted gene expression in mice. Bioconj Chem 1999; 10: 1075-1083.
25. Zhang G, Budker V, Wolff JA. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther 1999; 10: 1735-1737.
26. Dash PR, Read ML, Barrett LB, Wolfert MA, Seymour LW. Factors affecting blood clearance and in vivo distribution of polyelectrolyte complexes for gene delivery. Gene Therapy 1999; 6: 643-650.
27. Ueyama H, Waki M, Takagi M, Takenaka S. Novel synthesis of a tetra-acridinyl peptide as a new DNA polyintercalator. Nucleic Acids Symp Ser 2000; 44: 133-134.
28. Haensler J, Szoka JFC. Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjug Chem 1993; 4: 85-93.
29. Boulanger C, Di Giorgio C, Vierling P. Synthesis of acridine-nuclear localization signal (NLS) conjugates and evaluation of their impact on lipoplex and polyplex-based transfection. Eur J Med Chem 2005; 40: 1295-1306.
30. Shiraishi T, Hamzavi R, Nielsen PE. Targeted delivery of plasmid DNA into the nucleus of cells via nuclear localization signal peptide conjugated to DNA intercalating bis- and trisacridines. Bioconj Chem 2005; 16: 1112-1116.
31. Vinogradov SV, Zhang H, Mitin A, Warren G. Intercalating conjugates of Peg with nuclear localization signal (Nls) peptide. Polymer Prep 2008; 49: 434-435.
32. Zhang H, Mitin A, Vinogradov SV. Efficient transfection of blood-brain barrier endothe-lial cells by lipoplexes and polyplexes in the presence of nuclear targeting NLS-EG- acridine conjugates. Bioconjug Chem 2009; 20: 120-128.
33. Zhang H, Vinogradov SV. Short biodegradable polyamines for gene delivery and transfection of brain capillary endothelial cells. J Control Rel 2010; 143: 359-366.
34. Baumhover NJ, Anderson K, Fernandez CA, Rice KG. Synthesis and in vitro testing of new potent polyacridine-melittin gene delivery peptides. Bioconj Chem 2010; 21: 74-86.
35. Anderson K, Fernandez CA, Rice KG. N-Glycan targeted gene delivery to the dendritic cell SIGN receptor. Bioconj Chem 2010, e-pub ahead of print 22 May 2010, DOI: 10.1021/bc1000824.
36. Fernandez CA, Baumhover NJ, Anderson K, Rice KG. Discovery of metabolically stablized electronegative polyacridine-PEG peptide DNA open polyplexes. Bioconj Chem 2010; 21: 723-730.
37. 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: 1947-1954.
38. Blessing T, Remy JS, Behr JP. Monomolecular collapse of plasmid DNA into stable virus-like particles. Proc Natl Acad Sci USA 1998; 95: 1427-1431.
39. Kwok KY, McKenzie DL, Evers DL, Rice KG. Formulation of highly soluble poly(ethylene glycol)-peptide DNA condensates. J Pharm Sci 1999; 88: 996-1003.
40. Chen CP, Kim JS, Liu D, Rettig GR, McAnuff MA, Martin ME, et al. Synthetic PEGylated glycoproteins and their utility in gene delivery. Bioconj Chem 2007; 18: 371-378.
41. Kunath K, Von Harpe A, Petersen H, Fischer D, Voigt K, Kissel T, et al. The structure of PEG-modified poly(ethylene imines) influences biodistribution and pharmacokinetics of their complexes with NF-kB decoy in mice. Pharm Res 2002; 19: 810-817.
42. Li S-D, Chen Y-C, Hackett MJ, Huang L. Tumor-targeted delivery of siRNA by Self-assembled Nanoparticles. Mol Ther 2007; 16: 163-169.
43. Morille M, Montier T, Legras P, Carmoy N, Brodin P, Pitard B, et al. Long-circulating DNA lipid nanocapsules as new vector for passive tumor targeting. Biomaterials 2010; 31: 321-329.
44. Oupicky D, Ogris M, Howard KA, Dash PR, Ulbrich L, Seymour LW. Importance of lateral an steric stabilization of polyelectrolyte gene delivery vectors for extended systemic circulation. Mol Ther 2002; 5: 463-472.
45. Ko YT, K A, Hartner WC, Papahadjopoulos-Sternberg B, Torchilin VP. Self-assembling micelle-like nanoparticles based on phospholipid- polyethyleneimine conjugates for systemic gene delivery. J Control Rel 2009; 133: 132-138.
46. Zhou Q-H, Wu C, Manickam D, Oupický D. Evaluation of pharmacokinetics of bioreducible gene delivery vectors by real-time PCR. Pharm Res 2009; 26: 1581-1589.
47. Wadhwa MS, Collard WT, Adami RC, McKenzie DL, Rice KG. Peptide-mediated gene delivery: influence of peptide structure on gene expression. Bioconj Chem 1997; 8: 81-88.
48. Adami RC, Rice KG. Metabolic stability of glutaraldehyde cross-linked peptide DNA condensates. J Pharm Sci 1999; 88: 739-746.
49. Terebesi J, Kwok KY, Rice KG. Iodinated plasmid DNA as a tool for studying gene delivery. Anal Biochem 1998; 263: 120-123.
50. Rettig G, McAnuff M, Kim J, Liu D, Rice KG. Quantitative bioluminescence imaging of transgene expression in vivo. Anal Biochem 2006; 335: 90-94.