pH-responsive shielding of non-viral gene vectors

Expert Opinion on Drug Delivery

Volumn 3, Issue 5, 2006, Pages 563-571

  Meyer, Martin ^a   Wagner, Ernst ^a  

^a UNIVERSITY OF MUNICH   (Germany)

Author keywords

Artificial viruses; Bioresponsive; Non viral gene transfer; PEG shielding; Smart delivery systems

Indexed keywords

AMINE MACROGOL COPOLYMER; ANTINEOPLASTIC AGENT; ANTISENSE OLIGODEOXYNUCLEOTIDE; ANTISENSE OLIGODEOXYNUCLEOTIDE COPOLYMER; CARBAMIC ACID DERIVATIVE; COPOLYMER; DIACYLGLYCEROL MACROGOL COPOLYMER; DNA; FLUORESCEIN; HYDRAZONE DERIVATIVE; LIPOSOME; MACROGOL; MELITTIN; MESSENGER RNA; OLIGODEOXYNUCLEOTIDE; OLIGONUCLEOTIDE; PEGYLATED OLIGONUCLEOTIDE; PHOSPHATIDYLCHOLINE; PHOSPHATIDYLGLYCEROL; PHOSPHORODIAMIDATE MORPHOLINOOLIGODEOXYNUCLEOTIDE; POLYCATION; POLYCATION MACROGOL COPOLYMER; POLYLYSINE; POLYMETHACRYLIC ACID DERIVATIVE; POLYMETHACRYLOYL SULFADIMETHOXINE MACROGOL COPOLYMER; UNCLASSIFIED DRUG;

CHEMICAL ANALYSIS; CHEMICAL REACTION; CHEMICAL STRUCTURE; COMPLEX FORMATION; CYTOTOXICITY; DNA BINDING; DNA DEGRADATION; ENCAPSULATION; ENDOCYTOSIS; ENDOSOME; GENE EXPRESSION; GENE VECTOR; HYDROLYSIS; NONVIRAL GENE DELIVERY SYSTEM; OPSONIZATION; OXIDATION REDUCTION POTENTIAL; PH; PHASE TRANSITION; REVIEW; SYNTHESIS;

DRUG DELIVERY SYSTEMS; ENDOCYTOSIS; ENDOSOMES; GENETIC VECTORS; HYDROGEN-ION CONCENTRATION; OLIGONUCLEOTIDES; POLYETHYLENE GLYCOLS; TRANSFECTION;

EID: 33748673542     PISSN: 17425247     EISSN: None     Source Type: Journal    
DOI: 10.1517/17425247.3.5.563     Document Type: Review

References (55)

1. 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) 6:595-605.
2. CHOI YH, LIU F, KIM JS, CHOI YK, PARK JS, KIM SW. Polyethylene glycol-grafted poly-L-lysine as polymeric gene carrier. J. Control. Release (1998) 54:39-48.
3. KWOH DY, COFFIN CC, LOLLO CP et al.: Stabilization of poly-L-lysine/DNA polyplexes for in vivo gene delivery to the liver. Biochim. Biophys. Acta (1999) 1444:171-190.
4. 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-κB decoy in mice. Pharm. Res. (2002) 19:810-817.
5. TAM P, MONCK M, LEE D et al.: Stabilized plasmid-lipid particles for systemic gene therapy. Gene Ther. (2000) 7:1867-1874.
6. OGRIS M, WALKER G, BLESSING T, KIRCHEIS R, WOLSCHEK M, WAGNER E: Tumor-targeted gene therapy: strategies for the preparation of ligand-polyethylene glycol-polyethylenimine/DNA complexes. J. Control Release (2003) 91:173-181.
7. ERBACHER P, BETTINGER. T, BELGUISE-VALLADIER P et al.: Transfection and physical properties of various saccharide, poly(ethylene glycol), and antibody-derivatized polyethylenimines (PEI). J. Gene Med. (1999) 1:210-222.
8. OUPICKY D, OGRIS M, HOWARD KA, DASH PR, ULBRICH K, SEYMOUR LW: Importance of lateral and steric stabilization of polyelectrolyte gene delivery vectors for extended systemic circulation. Mol Ther. (2002) 5:463-472.
9. RUDOLPH C, SCHILLINGER U, PLANK C et al.: Nonviral gene delivery to the lung with copolymer-protected and transferrin-modified polyethyleneimine. Biochim. Biophys. Acta (2002) 1573:75.
10. GUO W, LEE RJ: Receptor-targeted gene delivery via folate-conjugated polyethylenimine. AAPS Pharmsci. (1999) 1:Article 19.
11. WAGNER E, ZENKE M, COTTEN M, BEUG H, BIRNSTIEL ML: Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA (1990) 87:3410-3414.
12. FARHOOD H, BOTTEGA R, EPAND RM, HUANG L: Effect of cationic cholesterol derivatives on gene transfer and protein kinase C activity. Biochim. Biophys. Acta (1992) 1111:239-246.
13. XU Y, SZOKA FC Jr: Mechanism of DNA release from cationic liposome/DNA complexes used in cell transfection. Biochemistry (1996) 35:5616-5623.
14. OGRIS M, CARLISLE RC, BETTINGER T, SEYMOUR LW: Melittin enables efficient vesicular escape and enhanced nuclear access of nonviral gene delivery vectors. J. Biol. Chem. (2001) 276:47550-47555.
15. KULKARNI RP, MISHRA S, FRASER SE, DAVIS ME: Single cell kinetics of intracellular, nonviral, nucleic acid delivery vehicle acidification and trafficking. Bioconjug. Chem. (2005) 16:986-994.
16. HATAKEYAMA H, AKITA H, KOGURE K et al.: Development of a novel systemic gene delivery system for cancer therapy with a tumor-specific cleavable PEG-lipid. Gene Ther. (2006). In press.
17. CARLISLE RC, ETRYCH T, BRIGGS SS, PREECE JA, ULBRICH K, SEYMOUR LW. Polymer-coated polyethylenimine/DNA complexes designed for triggered activation by in tracellular reduction. J. Gene Med. (2004) 6:337-344.
18. GUO X, SZOKA FC Jr: Chemical approaches to triggerable lipid vesicles for drug and gene delivery. Acc. Chem. Res. (2003) 36:335-341.
19. WALKER GF, FELLA C, PELISEK J et al.: Toward synthetic viruses: endosomal pH-triggered deshielding of targeted polyplexes greatly enhances gene transfer in vitro and in vivo. Mol. Ther. (2005) 11:418-425.
20. AKINC A, THOMAS M, KLIBANOV AM, LANGER R: Exploring polyethylenimine-mediated DNA transfection and the proton sponge hypothesis. J. Gene Med. (2005) 7:657-663.
21. KIRCHEIS R, KICHLER A, WALLNER G et al.: Coupling of cell-binding ligands to polyethylenimine for targeted gene delivery. Gene Ther. (1997) 4:409-418.
22. 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. Bioconjug. Chem. (2001) 12:529-537.
23. WOLSCHEK MF, THALLINGER C, KURSA M et al: Specific systemic nonviral gene delivery to human hepatocellular carcinoma xenografts in SCID mice. Hepatology (2002) 36:1106-1114.
24. SHIN J, SHUM P, THOMPSON DH: Acid-triggered release via depegylation of DOPE liposomes containing acid-labile vinyl ether PEG-lipids. J. Control. Release (2003) 91:187-200.
25. CHOI JS, MACKAY JA, SZOKA FC Jr: Low-pH-sensitive PEG-stabilized plasmid-lipid nanoparticles: preparation and characterization. Bioconjug. Chem. (2003) 14:420-429.
26. LI W, HUANG Z, MACKAY JA, GRUBE S, SZOKA FC Jr: 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. (2005) 7:67-79.
27. MURTHY N, CAMPBELL J, FAUSTO N, HOFFMAN AS, STAYTON PS: Design and synthesis of pH-responsive polymeric carriers that target uptake and enhance the intracellular delivery of oligonucleotides. J. Control. Release (2003) 89:365-374.
28. MURTHY N, CAMPBELL J, FAUSTO N, HOFFMAN AS, STAYTON PS: Bioinspired pH-responsive polymers for the intracellular delivery of biomolecular drugs. Bioconjug. Chem. (2003) 14:412-419.
29. OISHI M, NAGATSUGI F, SASAKI S, NAGASAKI Y, KATAOKA K: Stuart polyion complex micelles for targered intracellular delivery of PEGylated antisense oligonucleotides containing acid-labile linkages. Chembiochem. (2005) 6:718-725.
30. OISHI M, NAGASAKI Y, ITAKA K, NISHIYAMA N, KATAOKA K: Lactosylated poly(ethylene glycol)-siRNA conjugate through acid-labile β-thiopropionate linkage to construct pH-sensitive polyion complex micelles achieving enhanced gene silencing in hepatoma cells. J. Am. Chem. Soc. (2005) 127:1624-1625.
31. OISHI M, SASAKI S, NAGASAKI Y, KATAOKA K: pH-responsive oligodeoxynucleotide (ODN)-poly(ethylene glycol) conjugate through acid-labile β-thiopropionate linkage: preparation and polyion complex micelle formation. Biomacromolecules (2003) 4:1426-1432.
32. MURTHY N, XU M, SCHUCK S, KUNISAWA J, SHASTRI N, FRECHET JM: A macromolecular delivery vehicle for protein-based vaccines: acid-degradable protein-loaded microgels. Proc. Natl. Acad. Sci. USA (2003) 100:4995-5000.
33. JEONG JH, KIM SW, PARK TG: Novel intracellular delivery system of antisense oligonucleotide by self-assembled hybrid micelles composed of DNA/PEG conjugate and cationic fusogenic peptide. Bioconjug. Cbem. (2003) 14:473-479.
34. LEE H, JEONG JH, PARK TG: A new gene delivery formulation of polyethylenimine/DNA complexes coated with PEG conjugated fusogenic peptide. J. Control. Release (2001) 76:183-192.
35. FINSINGER D, REMY JS, ERBACHER P KOCH C, PLANK C: Protective copolymers for nonviral gene vectors: synthesis, vector characterization and application in gene delivery. Gene Ther. (2000) 7:1183-1192.
36. SETHURAMAN VA, NA K, BAE YH: pH-responsive sulfonamide/PEI system for tumor specific gene delivery: an in vitro study. Biomacromolecules (2006) 7:64-70.
37. BLUME G, CEVC G, CROMMELIN MD, BAKKER W, KLUFT C, STORM G: Specific targeting with poly(ethylene glycol)-modified liposomes: coupling of homing devices to the ends of the polymeric chains combines effective target binding with long circulation times. Biochim. Biophys. Acta (1993) 1149:180-184.
38. MILLER CR, BONDURANT B, MCLEAN SD, MCGOVERN KA, O'BRIEN DF: Liposome-cell interactions in vitro: effect of liposome surface charge on the binding and endocytosis of conventional and sterically stabilized liposomes. Biochemistry (1998) 37:12875-12883.
39. MASSON C, GARINOT M, MIGNET N et al.: pH-sensitive PEG lipids containing orthoester linkers: new potential tools for nonviral gene delivery. J. Control. Release (2004) 99:423-434.
40. AMBEGIA E, ANSELL S, CULLIS P, HEYES J, PALMER L, MACLACHLAN I: Stabilized plasmid-lipid particles containing PEG-diacylglycerols exhibit extended circulation lifetimes and tumor selective gene expression. Biochim. Biophys. Acta (2005) 1669:155-163.
41. STRUCK DK, HOEKSTRA D, PAGANO RE: Use of resonance energy transfer to monitor membrane fusion. Biocbemisty (1981) 20:4093-4099.
42. AUGUSTE DT, ARMES SP, BRZEZINSKA KR, DEMING TJ, KOHN J, PRUD'HOMME RK: pH triggered release of protective poly(ethylene glycol)-b-polycation copolymers from liposomes. Biomaterials (2006) 27:2599-2608.
43. LIU D, HU J, QIAO W, LI Z, ZHANG S, CHENG L: Synthesis of carbamate-linked lipids for gene delivery. Bioorg. Med. Chem. Lett. (2005) 15:3147-3150.
44. LIU D, HU J, QIAO W, LI Z, ZHAN S, CHENG L: Synthesis and characterization of a series of carbamate-linked cationic lipids for gene delivery. Lipids (2005) 40:839-848.
45. WAGNER E: Application of membrane-active peptides for nonviral gene delivery. Adv. Drug Deliv. Rev. (1999) 38:279-289.
46. ROZEMA DB, EKENA K, LEWIS DL, LOOMIS AG, WOLFF JA: Endosomolysis by masking of a membrane-active agent (EMMA) for cytoplasmic release of macromolecules. Bioconjug. Chem. (2003) 14:51-57.
47. WIETHOFF CM, WODRICH H, GERACE L, NEMEROW GR: Adenovirus protein VI mediates membrane disruption following capsid disassembly. J. Virol. (2005) 79:1992-2000.
48. MIYATA K, KAKIZAWA Y, NISHIYAMA N et al.: Block catiomer polyplexes with regulated densities of charge and disulfide cross-linking directed to enhance gene expression. J. Am. Chem. Son (2004) 126:2355-2361.
49. READ ML, BREMNER KH, OUPICKY D, GREEN NK, SEARLE PF, SEYMOUR LW. Vectors based on reducible polycations facilitate intracellular release of nucleic acids. J. Gene Med. (2003) 5:232-245.
50. READ ML, SINGH S, AHMED Z et al.: A versatile reducible polycation-based system for efficient delivery of a broad range of nucleic acids. Nucleic Acids Res. (2005) 33:e86.
51. MCKENZIE DL, KWOK KY, RICE KG: A potent new class of reductively activated pepride gene delivery agents. J. Biol. Chem. (2000) 275:9970-9977.
52. KHALIL IA, KOGURE K, AKITA H, HARASHIMA H: Uptake pathways and subsequent intracellular trafficking in nonviral gene delivery. Pharmacol. Rev. (2006) 58:32-45.
53. MEDINA-KAUWE LK, XIE J, HAMM-ALVAREZ S: Intracellular trafficking of nonviral vectors. Gene Ther. (2005) 12:1734-1751.
54. WOLFF JA: The 'grand' problem of synthetic delivery. Nat. Biotechnol. (2002) 20:768-769.
55. WAGNER E: Strategies to improve DNA polyplexes for in vivo gene transfer: will 'artificial viruses' be the answer? Pharm. Res. (2004) 21:8-14.