Obstacles and advances in non-viral gene delivery

Current Opinion in Molecular Therapeutics

Volumn 2, Issue 2, 2000, Pages 136-142

  Lollo, Charles Peter ^a   Banaszczyk, Mariusz G ^a   Chiou, Henry C ^a  

^a Orchestra Therapeutics Inc   (United States)

Author keywords

Cytoplasmic entry; Gene delivery; Non viral; Particle stabilization; Targeting

Indexed keywords

LIPID; LIPOSOME; PLASMID DNA; VIRUS DNA; VIRUS VECTOR;

CELL NUCLEUS; CYTOPLASM; GENE TARGETING; GENETIC ENGINEERING; INTRACELLULAR TRANSPORT; MEMBRANE PERMEABILITY; PLASMID; REVIEW;

ANIMALS; BIOLOGICAL TRANSPORT, ACTIVE; CELL NUCLEUS; CYTOPLASM; DRUG DELIVERY SYSTEMS; DRUG STABILITY; GENE THERAPY; HUMANS; PLASMIDS;

ANIMALIA; DNA VIRUSES;

EID: 0034003408     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (54)

1. Ji L, Bouvet M, Price RE, Roth JA, Fang B: Reduced toxicity, attenuated immunogenicity and efficient mediation of human p53 gene expression in vivo by an adenovirus vector with deleted E1-E3 and inactivated E4 by GAL4-TATA promoter replacement Gene Ther (1999) 6:393-402.
2. O'Riordan CR, Lachapelle A, Delgado C, Parkes V, Wadsworth SC, Smith AE, Francis GE: PEGylation of adenovirus with retention of infectivity and protection from neutralizing antibody in vitro and in vivo. Hum Gene Ther (1999) 10:1349-1358.
3. 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.
4. Wu GY, Wilson JM, Shalaby F, Grossman M, Shafritz DA, Wu CH: Receptor-mediated gene delivery In vivo. J Biol Chem (1991) 266:14338-14342.
5. Lasic DD, Templeton NS: Liposomes in gene therapy. Adv Drug Deliv Rev (1996) 20:21-266.
6. Centers for Disease Control and Prevention: Recommendations for the prevention and control of hepatitis C virus (HCV) infection and HCV-related chronic disease. MMWR Morb Mortal Wkly Rep (1998) 47(Suppl RR-19):1-39.
7. Hoofnagle JH, Di Bisceglie AM: The treatment of chronic viral hepatitis. New Engl J Med (1997) 336:347-356.
8. Olin BR (Ed): Drug Facts and Comparisons. Facts and Comparisons. St Louis, MO (1996).
9. Renault PF, Hoofnagle JH: Side effects of α interferon. Semin Liver Dis (1989) 9:273-277.
10. Okanoue T, Sakamoto S, Itoh Y, Minami M, Yasui K, Sakamoto M, Nishioji K, Katagishi T, Nakagawa Y, Tada H, Sawa Y, Mizuno M, Kagawa K, Kashima K: Side effects of high-dose interferon therapy for chronic hepatitis C. J Hepatol (1996) 25:283-291.
11. Rosenstein DL, Lerner D, Cai J: More on the depressive effects of interferon alpha. New Engl J Med (1999) 341:849-850.
12. Rutherford MN, Kumar A, Coulombe B, Skup D, Carver DH, Williams BRG: Expression of Intracellular interferon constitutively activates ISGF3 and confers resistance to EMC viral infection. J Interferon Cytokine Res (1996) 16:507-510.
13. Kichler A, Mechtler K, Behr JP, Wagner E: Influence of membrane-active peptides on lipospermine/DNA complex mediated gene transfer. Bioconjug Chem (1997) 8:213-221.
14. Capecchi MR: High efficiency transformation by direct micro-injection of DNA into cultured mammalian cells. Cell (1980) 22:479-488.
15. Kabanov AV, Kabanov VA: Interpolyelectrolyte and block ionomer complexes for gene delivery: Physicochemical aspects. Adv Drug Deliv Rev (1998) 30:49-60.
16. Kwoh DY, Coffin CC, Lollo CP, Jovenal J, Banaszczyk MG, Mullen P, Phillips A, Amini A, Fabrycki J, Bartholomew RM, Brostoff SW, Carlo DJ: Stabilization of poly-L-lysine/DNA polyplexes for in vivo gene delivery to the liver. Biochim Biophys Acta (1999) 1444:171-190.
17. Torchilin VP, Omelyanenko VG, Papisov MI, Bogdanov AA, Trubetskoy VS, Herron JN, Gentry CA: Poly(ethylene glycol) on the liposome surface: On the mechanism of polymer-coated liposome longevity. Biochim Biophys Acta (1994) 1195:11-20.
18. Uster PS, Allen TM, Daniel BE, Mendez CJ, Newman MS, Zhu GZ: Insertion of poly(ethylene glycol) derivatized phospholipid into pre-formed liposomes results in prolonged in vivo circulation time. FEBS Lett (1996) 386:243-246.
19. Watrous-Peltier N, Uhl J, Steel V, Brophy L, Merisko-Liversidge E: Direct suppression of phagocytosis by amphipathic polymeric surfactants. Pharm Res (1992) 9:1177-1183.
20. Toncheva V, Wolfert MA, Dash PR, Oupicky D, Ulbrich K, Seymour LW, Schacht EH: Novel vectors for gene delivery formed by self-assembly of DNA with poly(L-lysine) grafted with hydrophilic polymers. Biochim Biophys Acta (1998) 1380:354-368.
21. Lasic DD, Needham D: The 'Stealth' liposome: A prototypical biomaterial. Chem Rev (1995) 95:2601-2628.
22. Zanta MA, Belguise-Valladier P, Behr JP: Gene delivery: A single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc Natl Acad Sci USA (1999) 96:91-96.
23. Curiel DT, Wagner E, Cotten M, Bimstiel ML, Agarwal S, Li CM, Loechel S, Hu PC: High-efficiency gene transfer mediated by adenovirus coupled to DNA-polylysine complexes. Hum Gene Ther (1992) 3:147-154.
24. Trubetskoy VS, Loomis A, Slattum PM, Hagstrom JE, Budker VG, Wolff JA: Caged DNA does not aggregate in high ionic strength solutions. Bioconjug Chem (1999) 10:624-628.
25. Kakizawa Y, Harada A, Kataoka K: Environment-sensitive stabilization of core-shell structured polyion complex micelle by reversible crosslinking of the core through disulfide bond. J Am Chem Soc (1999) 121:11247-11248.
26. 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.
27. Adami RC, Rice KG: Metabolic stability of glutaraldehyde crosslink ed peptide DNA condensates. J Pharm Sci (1999) 88:739-746.
28. Banaszczyk MG, Lollo CP, Kwoh DY, Phillips AT, Amini A, Wu DP, Mullen PM, Coffin CC, Brostoff SW, Carlo DJ: Poly-L-Lysine-Graft - PEG comb-type polycation copolymers for gene delivery. Pure Appl Chem (1999) A36(7-8):1061-1084.
29. Erbacher P, Remy JS, Behr JP: Gene transfer with synthetic virus-like particles via the integrin-mediated endocytosis pathway. Gene Ther (1999) 6:138-145.
30. Diebold SS, Kursa M, Wagner E, Cotten M, Zenke M: Mannose polyethylenimine conjugates for targeted DNA delivery into dendritic cells. J Biol Chem (1999) 274:19087-19094.
31. Hisayasu S, Miyauchi M, Akiyama K, Gotoh T, Satoh S, Shimada T: In vivo targeted gene transfer into liver cells mediated by a novel galactosyl-D-lysine/D-serine copolymer. Gene Ther (1999) 6:689-693.
32. Xu L, Pirollo KF, Tang W-H, Rait A, Chang EH: Transferrin-liposome-mediated systemic p53 gene therapy in combination with radiation results in regression of human head and neck cancer xenografts. Hum Gene Ther(1999) 10:2941-2952.
33. Fujii G: To fuse or not to fuse: the effects of electrostatic interactions, hydrophobia forces and structural amphiphilicity on protein-mediated membrane destabilization. Adv Drug Deliv Rev (1999) 38:257-277.
34. Wagner E: Application of membrane-active peptides for non-viral gene delivery. Adv Drug Deliv Rev (1999) 38:279-289.
35. Liu F, Song YK, Liu D: Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther (1999) 6:1258-1266.
36. 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.
37. Lollo CP, Kwoh DY, Mockler TC, Ley PM, Guido MS, Coffin CC, Aleman R, Bartholomew RM, Carlo DJ: Non-viral gene delivery: Vehicle and delivery characterization. Blood Coagul Fibrinolysis (1997) 8:S31-38.
38. Von der Leyen HE, Braun-Dullaeus R, Mann MJ, Zhang L, Niebauer J, Dzau VJ: A pressure-mediated non-viral method for efficient arterial gene and oligonucleotide transfer. Hum Gene Ther (1999) 10:2355-2364.
39. Budker V, Zhang G, Danko I, Williams P, Wolff J: The efficient expression of intravascularly delivered DNA in rat muscle. Gene Ther (1998) 5:272-276.
40. Rekhter MD, Shah N, Simari RD, Work C, Kim JS, Nabel GJ, Nabel EG, Gordon D: Graft permeabilization facilitates gene therapy of transplant arterioscleriosis in a rabbit model. Circulation (1998) 98:1335-1341.
41. Davis HL, Whalen RG, Demenix BA: Direct gene transfer into skeletal muscle in vivo: Factors affecting efficiency of transfer and stability of expression. Hum Gene Ther (1993) 4:151-159.
42. Mir LM, Bureau MF, Gehl J, Rangara R, Rouy D, Caillaud J-M, Delaere P, Branellec D, Schwartz B, Scherman D: High-efficiency gene transfer into skeletal muscle mediated by electric pulses. Proc Natl Acad Sci USA (1999) 96:4262-4267.
43. Aihara H, Miyazaki J: Gene transfer into muscle by electroporation in vivo. Nature Biotechnol (1998) 16:867-870.
44. Harrison RL, Byme BJ, Tung L: Electroporation-mediated gene transfer in cardiac tissue. FEBS Lett (1998) 435:1-5.
45. Oshima Y, Sakamoto T, Yamanaka I, Nishi T, Ishibashi T, Inomata H: Targeted gene transfer to corneal endothelium In vivo by electric pulse. Gene Ther (1998) 5:1347-1354.
46. Moroianu J: Nuclear import and export pathways. J Cell Biochem (1999) (Suppl 32-33):76-83.
47. Neves C, Byk G, Scherman D, Wils P: Coupling of a targeting peptide to plasmid DNA by covalent triple helix formation. FEBS Lett (1999) 453:41-45.
48. Branden LJ, Mohamed AJ, Smith CIE: A peptide nucleic acid-nuclear localization signal fusion that mediates nuclear transport of DNA. Nature Biotechnol (1999) 17:784-787.
49. Subramanian A, Ranganathan P, Diamond SL: Nuclear targeting peptide scaffolds for lipofection of non-dividing mammalian cells. Nature Biotechnol (1999) 17:873-877.
50. Chan CK, Jans DA: Enhancement of polylysine-mediated transferrinfection by nuclear localization sequences: Polylysine does not function as a nuclea r localization sequence. Hum Gene Ther (1999) 10:1695-1702.
51. Zauner W, Brunner S, Buschle M, Ogris M, Wagner E: Differential behavior of lipid based and polycation-based gene transfer systems in transfecting primary human fibroblasts: A potential role of polylysine in nuclear transport Biochim Biophys Acta (1999) 1428:57-67.
52. Pollard H, Remy JS, Loussouam G, Demolombe S, Behr JP, Escande D: Polyethylenimine but not cationic lipids promotes transgene delivery to the nucleus In mammalian cells. J Biol Chem (1998) 273:7507-7511.
53. Vacik J, Dean BS, Zimmer WE, Dean DA: Cell-specific nuclear import of plasmid DNA. Gene Ther (1999) 6:1006-1014.
54. Dean DA, Dean BS, Muller S, Smith LC: Sequence requirements for plasmid nuclear import. Exp Cell Res (1999) 253:713-722.