Amine-reactive pyridylhydrazone-based PEG reagents for pH-reversible PEI polyplex shielding

European Journal of Pharmaceutical Sciences

Volumn 34, Issue 4-5, 2008, Pages 309-320

  Fella, Carolin ^a   Walker, Greg F ^a   Ogris, Manfred ^a   Wagner, Ernst ^a  

^a UNIVERSITY OF MUNICH   (Germany)

Author keywords

Bioreversible modification; Drug delivery; Gene transfer; PEG pyridylhydrazone NHS ester; PEGylation; Plasmid DNA

Indexed keywords

ALDEHYDE; ALEXA FLUOR 488; AMINE; BUTYRALDEHYDE; CARBOXYPYRIDYLHYDRAZONE; EPIDERMAL GROWTH FACTOR; EPIDERMAL GROWTH FACTOR RECEPTOR; FLUORESCENT DYE; HYDRAZINE DERIVATIVE; HYDRAZONE DERIVATIVE; LUCIFERASE; MACROGOL; N HYDROXYSUCCINIMIDE; NICOTINIC ACID DERIVATIVE; OMEGA 2 PYRIDYLDITHIO POLYETHYLENE GLYCOL PROPIONALDEHYDE; POLYETHYLENEIMINE; PROPIONALDEHYDE; PYRIDINE DERIVATIVE; PYRIDYLHYDRAZONE; REAGENT; SUCCINIMIDE DERIVATIVE; SUCCINIMIDYL HYDRAZINIUMNICOTINATE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; COMPLEX FORMATION; CONTROLLED STUDY; ENDOSOME; FEMALE; FLUORESCENCE CORRELATION SPECTROSCOPY; GENE OVEREXPRESSION; GENETIC TRANSFECTION; HEPATOMA CELL; HUMAN; HUMAN CELL; IN VIVO STUDY; MALE; MEASUREMENT; NONHUMAN; PARTICLE SIZE; PH; PRIORITY JOURNAL; SCID MOUSE; SURFACE PROPERTY; SYNTHESIS; TARGET CELL; TRANSGENE; TUMOR MODEL; ZETA POTENTIAL;

AMINES; ANIMALS; CELL LINE, TUMOR; DNA; ENDOSOMES; EPIDERMAL GROWTH FACTOR; FEMALE; GENES, REPORTER; HUMANS; HYDRAZONES; HYDROGEN-ION CONCENTRATION; LIVER NEOPLASMS, EXPERIMENTAL; LUCIFERASES; MALE; MICE; MICE, SCID; PARTICLE SIZE; POLYETHYLENE GLYCOLS; POLYETHYLENEIMINE; PYRIDINES; RECEPTOR, EPIDERMAL GROWTH FACTOR; SPECTROMETRY, FLUORESCENCE; TIME FACTORS; TRANSFECTION;

EID: 47249130263     PISSN: 09280987     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ejps.2008.05.004     Document Type: Article

References (56)

1. Allen T.M., and Cullis P.R. Drug delivery systems: entering the mainstream. Science 303 (2004) 1818-1822
2. Ambegia E., Ansell S., Cullis P., Heyes J., Palmer L., and MacLachlan I. Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim. Biophys. Acta 1669 (2005) 155-163
3. Bieber T., Meissner W., Kostin S., Niemann A., and Elsasser H.P. Intracellular route and transcriptional competence of polyethylenimine-DNA complexes. J. Control Release 82 (2002) 441-454
4. Blessing T., Kursa M., Holzhauser R., Kircheis R., and Wagner E. Different strategies for formation of pegylated EGF-conjugated PEI/DNA complexes for targeted gene delivery. Bioconjug. Chem. 12 (2001) 529-537
5. Boeckle S., von Gersdorff K., van der Piepen S., Culmsee C., Wagner E., and Ogris M. Purification of polyethylenimine polyplexes highlights the role of free polycations in gene transfer. J. Gene Med. 6 (2004) 1102-1111
6. Boeckle S., and Wagner E. Optimizing targeted gene delivery: chemical modification of viral vectors and synthesis of artificial virus vector systems. AAPS J. 8 (2006) E731-E742
7. Brissault B., Kichler A., Guis C., Leborgne C., Danos O., and Cheradame H. Synthesis of linear polyethylenimine derivatives for DNA transfection. Bioconjug. Chem. 14 (2003) 581-587
8. Childs C.E. The determination of polyethylene glycol in gamma globulin solutions. Microchem. J. 20 (1975) 190-192
9. Chollet P., Favrot M.C., Hurbin A., and Coll J.L. Side-effects of a systemic injection of linear polyethylenimine-DNA complexes. J. Gene Med. 4 (2002) 84-91
10. Croyle M.A., Chirmule N., Zhang Y., and Wilson J.M. "Stealth" adenoviruses blunt cell-mediated and humoral immune responses against the virus and allow for significant gene expression upon readministration in the lung. J. Virol. 75 (2001) 4792-4801
11. de Bruin K., Ruthardt N., von Gersdorff K., Bausinger R., Wagner E., Ogris M., and Brauchle C. Cellular dynamics of EGF receptor-targeted synthetic viruses. Mol. Ther. 15 (2007) 1297-1305
12. Demeneix B.A., and Behr J.P. The proton spronge: a trick the viruses didn't exploit. In: Felgner P.L., Heller M.J., Lehn P., Behr J.P., and Szoka F.C. (Eds). Artificial Self-Assembling Systems for Gene Delivery (1996), American Chemical Society, Washington, USA 146-151
13. Filpula D., Yang K., Basu A., Hassan R., Xiang L., Zhang Z., Wang M., Wang Q.C., Ho M., Beers R., Zhao H., Peng P., Zhou J., Li X., Petti G., Janjua A., Liu J., Wu D., Yu D., Zhang Z., Longley C., FitzGerald D., Kreitman R.J., and Pastan I. Releasable PEGylation of mesothelin targeted immunotoxin SS1P achieves single dosage complete regression of a human carcinoma in mice. Bioconjug. Chem. 18 (2007) 773-784
14. Gabizon A.A., Shmeeda H., and Zalipsky S. Pros and cons of the liposome platform in cancer drug targeting. J. Liposome Res. 16 (2006) 175-183
15. Goren D., Horowitz A.T., Zalipsky S., Woodle M.C., Yarden Y., and Gabizon A. Targeting of stealth liposomes to erbB-2 (Her/2) receptor: in vitro and in vivo studies. Br. J. Cancer 74 (1996) 1749-1756
16. Grace M.J., Cutler D.L., and Bordens R.W. Pegylated IFNs for chronic hepatitis C: an update. Expert. Opin. Drug Deliv. 2 (2005) 219-226
17. Hatakeyama H., Akita H., Kogure K., Oishi M., Nagasaki Y., Kihira Y., Ueno M., Kobayashi H., Kikuchi H., and Harashima H. Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid. Gene Ther. 14 (2007) 68-77
18. Hong K., Zheng W., Baker A., and Papahadjopoulos D. Stabilization of cationic liposome-plasmid DNA complexes by polyamines and poly(ethylene glycol)-phospholipid conjugates for efficient in vivo gene delivery. FEBS Lett. 400 (1997) 233-237
19. Kale A.A., and Torchilin V.P. "Smart" drug carriers: PEGylated TATp-modified pH-sensitive liposomes. J. Liposome Res. 17 (2007) 197-203
20. Kircheis R., Schuller S., Brunner S., Ogris M., Heider K.H., Zauner W., and Wagner E. Polycation-based DNA complexes for tumor-targeted gene delivery in vivo. J. Gene Med. 1 (1999) 111-120
21. Knorr V., Allmendinger L., Walker G.F., Paintner F.F., and Wagner E. An acetal-based PEGylation reagent for pH-sensitive shielding of DNA polyplexes. Bioconjug. Chem. 18 (2007) 1218-1225
22. Kopatz I., Remy J.S., and Behr J.P. A model for non-viral gene delivery: through syndecan adhesion molecules and powered by actin. J. Gene Med. 6 (2004) 769-776
23. Kreppel F., and Kochanek S. Modification of adenovirus gene transfer vectors with synthetic polymers: a scientific review and technical guide. Mol. Ther. 16 (2008) 16-29
24. Kursa M., Walker G.F., Roessler V., Ogris M., Roedl W., Kircheis R., and Wagner E. Novel shielded transferrin-polyethylene glycol-polyethylenimine/DNA complexes for systemic tumor-targeted gene transfer. Bioconjug. Chem. 14 (2003) 222-231
25. Li W., Huang Z., MacKay J.A., Grube S., and Szoka Jr. F.C. Low-pH-sensitive poly(ethylene glycol) (PEG)-stabilized plasmid nanolipoparticles: effects of PEG chain length, lipid composition and assembly conditions on gene delivery. J. Gene Med. 7 (2005) 67-79
26. Meyer M., Philipp A., Oskuee R., Schmidt C., and Wagner E. Breathing life into polycations: functionalization with pH-responsive endosomolytic peptides and polyethylene glycol enables siRNA delivery. J. Am. Chem. Soc. 130 (2008) 3272-3273
27. Meyer M., and Wagner E. pH-responsive shielding of non-viral gene vectors. Expert. Opin. Drug Deliv. 3 (2006) 563-571
28. Murthy N., Campbell J., Fausto N., Hoffman A.S., and Stayton P.S. Design and synthesis of pH-responsive polymeric carriers that target uptake and enhance the intracellular delivery of oligonucleotides. J. Control Release 89 (2003) 365-374
29. Ogris M., Brunner S., Schuller S., Kircheis R., and Wagner E. PEGylated DNA/transferrin-PEI complexes: reduced interaction with blood components, extended circulation in blood and potential for systemic gene delivery. Gene Ther. 6 (1999) 595-605
30. Ogris M., Steinlein P., Carotta S., Brunner S., and Wagner E. DNA/polyethylenimine transfection particles: influence of ligands, polymer size, and PEGylation on internalization and gene expression. AAPS PharmSci. 3 (2001) E21
31. Ogris M., Walker G., Blessing T., Kircheis R., Wolschek M., and Wagner E. Tumor-targeted gene therapy: strategies for the preparation of ligand-polyethylene glycol-polyethylenimine/DNA complexes. J. Control Release 91 (2003) 173-181
32. Oishi M., Nagasaki Y., Itaka K., Nishiyama N., and 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. 127 (2005) 1624-1625
33. Oupicky D., Ogris M., Howard K.A., Dash P.R., Ulbrich K., and Seymour L.W. Importance of lateral and steric stabilization of polyelectrolyte gene delivery vectors for extended systemic circulation. Mol. Ther. 5 (2002) 463-472
34. Plank C., Mechtler K., Szoka Jr. F.C., and Wagner E. Activation of the complement system by synthetic DNA complexes: a potential barrier for intravenous gene delivery. Hum. Gene Ther. 7 (1996) 1437-1446
35. Plank C., Zatloukal K., Cotten M., Mechtler K., and Wagner E. Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconjug. Chem. 3 (1992) 533-539
36. Romanczuk H., Galer C.E., Zabner J., Barsomian G., Wadsworth S.C., and O'Riordan C.R. Modification of an adenoviral vector with biologically selected peptides: a novel strategy for gene delivery to cells of choice. Hum. Gene Ther. 10 (1999) 2615-2626
37. Rozema D.B., Lewis D.L., Wakefield D.H., Wong S.C., Klein J.J., Roesch P.L., Bertin S.L., Reppen T.W., Chu Q., Blokhin A.V., Hagstrom J.E., and Wolff J.A. Dynamic PolyConjugates for targeted in vivo delivery of siRNA to hepatocytes. Proc. Natl. Acad. Sci U.S.A. 104 (2007) 12982-12987
38. Ruponen M., Honkakoski P., Tammi M., and Urtti A. Cell-surface glycosaminoglycans inhibit cation-mediated gene transfer. J. Gene Med. 6 (2004) 405-414
39. Schiedner G., Bloch W., Hertel S., Johnston M., Molojavyi A., Dries V., Varga G., van Rooijen N., and Kochanek S. A hemodynamic response to intravenous adenovirus vector particles is caused by systemic Kupffer cell-mediated activation of endothelial cells. Hum. Gene Ther. 14 (2003) 1631-1641
40. Smrekar B., Wightman L., Wolschek M.F., Lichtenberger C., Ruzicka R., Ogris M., Rodl W., Kursa M., Wagner E., and Kircheis R. Tissue-dependent factors affect gene delivery to tumors in vivo. Gene Ther. 10 (2003) 1079-1088
41. Snyder S.L., and Sobocinski P.Z. An improved 2,4,6-trinitrobenzenesulfonic acid method for the determination of amines. Anal. Biochem. 64 (1975) 284-288
42. Sonawane N.D., Szoka Jr. F.C., and Verkman A.S. Chloride accumulation and swelling in endosomes enhances DNA transfer by polyamine-DNA polyplexes. J. Biol. Chem. 278 (2003) 44826-44831
43. Ten Hagen T.L., Van Der Veen A.H., Nooijen P.T., Van Tiel S.T., Seynhaeve A.L., and Eggermont A.M. Low-dose tumor necrosis factor-alpha augments antitumor activity of stealth liposomal doxorubicin (DOXIL) in soft tissue sarcoma-bearing rats. Int. J Cancer 87 (2000) 829-837
44. van Vlerken L.E., Vyas T.K., and Amiji M.M. Poly(ethylene glycol)-modified nanocarriers for tumor-targeted and intracellular delivery. Pharm. Res. 24 (2007) 1405-1414
45. Verbaan F.J., Oussoren C., Snel C.J., Crommelin D.J., Hennink W.E., and Storm G. Steric stabilization of poly(2-(dimethylamino)ethyl methacrylate)-based polyplexes mediates prolonged circulation and tumor targeting in mice. J. Gene Med. 6 (2004) 64-75
46. Veronese F.M., and Pasut G. PEGylation, successful approach to drug delivery. Drug Discov. Today 10 (2005) 1451-1458
47. von Gersdorff K., Ogris M., and Wagner E. Cryoconserved shielded and EGF receptor targeted DNA polyplexes: cellular mechanisms. Eur. J Pharm. Biopharm. 60 (2005) 279-285
48. Wagner E. Strategies to improve DNA polyplexes for in vivo gene transfer: will "artificial viruses" be the answer?. Pharm. Res. 21 (2004) 8-14
49. Wagner E. Converging Paths of Viral and Non-viral Vector Engineering. Mol. Ther. 16 (2008) 1-2
50. Walker G.F., Fella C., Pelisek J., Fahrmeir J., Boeckle S., Ogris M., and Wagner E. Toward synthetic viruses: endosomal pH-triggered deshielding of targeted polyplexes greatly enhances gene transfer in vitro and in vivo. Mol. Ther. 11 (2005) 418-425
51. Wheeler J.J., Palmer L., Ossanlou M., MacLachlan I., Graham R.W., Zhang Y.P., Hope M.J., Scherrer P., and Cullis P.R. Stabilized plasmid-lipid particles: construction and characterization. Gene Ther. 6 (1999) 271-281
52. Wightman L., Kircheis R., Rossler V., Carotta S., Ruzicka R., Kursa M., and Wagner E. Different behavior of branched and linear polyethylenimine for gene delivery in vitro and in vivo. J. Gene Med. 3 (2001) 362-372
53. Wolff J.A., and Rozema D.B. Breaking the bonds: non-viral vectors become chemically dynamic. Mol. Ther. 16 (2008) 8-15
54. Wolschek M.F., Thallinger C., Kursa M., Rossler V., Allen M., Lichtenberger C., Kircheis R., Lucas T., Willheim M., Reinisch W., Gangl A., Wagner E., and Jansen B. Specific systemic nonviral gene delivery to human hepatocellular carcinoma xenografts in SCID mice. Hepatology 36 (2002) 1106-1114
55. Zimmermann T.S., Lee A.C., Akinc A., Bramlage B., Bumcrot D., Fedoruk M.N., Harborth J., Heyes J.A., Jeffs L.B., John M., Judge A.D., Lam K., McClintock K., Nechev L.V., Palmer L.R., Racie T., Rohl I., Seiffert S., Shanmugam S., Sood V., Soutschek J., Toudjarska I., Wheat A.J., Yaworski E., Zedalis W., Koteliansky V., Manoharan M., Vornlocher H.P., and MacLachlan I. RNAi-mediated gene silencing in non-human primates. Nature 441 (2006) 111-114
56. Zou S.M., Erbacher P., Remy J.S., and Behr J.P. Systemic linear polyethylenimine (L-PEI)-mediated gene delivery in the mouse. J. Gene Med. 2 (2000) 128-134