Tailoring new gene delivery designs for specific targets

Journal of Drug Targeting

Volumn 8, Issue 1, 2000, Pages 1-12

  Benns, Jonathan M ^a   Kim, Sung Wan ^a  

^a UNIVERSITY OF UTAH   (United States)

Author keywords

Endocytosis; Expression; Receptor mediated; Terplex; Transfection

Indexed keywords

DNA; FOLIC ACID; LACTOSE; LIPOFECTIN; LOW DENSITY LIPOPROTEIN; MACROGOL; MONOCLONAL ANTIBODY; NANOPARTICLE; PLASMID DNA; POLYLYSINE; POLYSACCHARIDE;

ADSORPTION; BIODEGRADATION; BIOTECHNOLOGY; CANCER CELL; CELL CULTURE; CELL STRAIN HEPG2; CELL TYPE; CYTOTOXICITY; ENDOTHELIUM CELL; GENE TARGETING; GENETIC TRANSFECTION; HUMAN; LEUKEMIA; NONHUMAN; PARTICLE SIZE; PRIORITY JOURNAL; REVIEW; SMOOTH MUSCLE FIBER; THYMOCYTE;

EID: 0034085960     PISSN: 1061186X     EISSN: None     Source Type: Journal    
DOI: 10.3109/10611860009009205     Document Type: Review

References (52)

1. Abuchowski, A., Kazo, G.M., Verhoest Jr., C.R., Van Es, T., Kafkewitz, D., Nucci, M.L., Viau, A.T. and Davis, F.F. (1984) Cancer therapy with chemically modified enzymes. I. Anti-tumor properties of polyethylene glycol asparaginase conjugates. Cancer Biochem. Biophys. 7, 175-86.
2. Ashwell, G. and Harford, J. (1982) Carbohydrate-specific receptors of the liver. Annu. Rev. Biochem. 51, 531-54.
3. Chiou, H.C., Tangco, M.V., Levine, S.M., Robertson, D., Kormis, K., Wu, C.H. and Wu, G.Y. (1994) Enhanced resistance to nuclease degradation of nucleic acids complexed to asialoglycoprotein polylysine carriers. Nucleic Acids Res. 22, 5439-46.
4. Choi, Y.H., Liu, F., Kim, J.S., Choi, Y.K., Park, J.S. and Kim, S.W. (1998a) Polyethylene glycol-graftcd poly-L-lysine as polymeric gene carrier. J. Controlled Release 54, 39-48.
5. Choi, Y.H., Liu, F., Park, J.S. and Kim, S.W. (1998b) Lactosepoly(ethylene glycol)-grafted poly-L-lysine as hepatoma cell-targeted gene carrier. Bioconjug. Chem. 9, 708-18.
6. Chung, J.K., So, Y., Hong, M.K., Choi, S.R., Jeong, J.M., Lee, D.S., Lee, M.C., Koh, C.S., Choi, E.Y. and Park, S.H. (1997) In vitro and in vivo properties of murine monoclonal antibody for a novel immature thymocyte-differentiated antigen, JL1. Nucl. Med. Biol. 24, 433-7.
7. Cristiano, R.J. and Roth, J.A. (1995) Molecular conjugates: a targeted gene delivery vector for molecular medicine. J. Mol. Med. 73, 479-86.
8. Flotte, T.R., Afione, S.A. and Zeitlin, P.L. (1994) Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am. J. Respir. Cell Mol. Biol. 11, 517-21.
9. Haensler, J. and Szoka Jr., F.C. (1993) Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjug. Chem. 4, 85-93.
10. Kabanov, A.V. and Kabanov, V.A. (1995) DNA complexes with polycations for the delivery of genetic material into cells. Bioconjug. Chem. 6, 7-20.
11. Katre, N.V., Knauf, M.J. and Laird, W.J. (1987) Chemical modification of recombinant interleukin 2 by polyethylene glycol increases its potency in the murine Meth A sarcoma model. Proc. Natl. Acad. Sci. USA 84, 1487-91.
12. Kim, J.S., Kim, B.I. Maruyama, A., Akaike, T. and Kim, S.W. (1998a) A new non-viral DNA delivery vector: the terplex system. J. Controlled Release 53, 175-82.
13. Kim, J.S., Maruyama, A., Akaike, T. and Kim, S.W. (1998b) Terplex DNA delivery system as a gene carrier. Pharm. Res. 15, 116-21.
14. Kolb, H., Vogt, D., Herbertz, L., Corfield, A., Schauer, R. and Schlepper-Schafer, J. (1980) The galactose-specific lectins on rat hepatocytes and Kupffer cells have identical binding characteristics. Hoppe Seylers Z. Physiol. Chem. 361, 1747-50.
15. Kotin, R.M., Siniscalco, M., Samulski, R.J., Zhu, X.D., Hunter, L., Laughlin, C.A., McLaughlin, S., Muzyczka, N., Rocchi, M. and Berns, K.I. (1990) Site-specific integration by adeno-associated virus. Proc. Natl. Acad. Sci. USA 87, 2211-15.
16. Kwoh, D.Y., Coffin, C.C., Lollo, C.P., Jovenal, J., Banaszezyk, M.G., Mullen, P., Phillips, A., Amini, A., Fabrycki, J., Bartholomew, R.M., Brostoff, S.W. and Carlo, D.J. (1999) Stabilization of poly-L-lysine/DNA polyplexes for in vivo gene delivery to the liver. Biochim. Biophys. Acta 1444, 171-90.
17. Laemmli, U.K. (1975) Characterization of DNA condensates induced by poly(ethylene oxide) and polylysine. Proc. Natl. Acad. Sci. USA 72, 4288-92.
18. Leamon, C.P. and Low, P.S. (1991) Delivery of macromolecules into living cells: a method that exploits folate receptor endocytosis. Proc. Natl. Acad. Sci. USA 88, 5572-6.
19. Ledley, F.D. (1995) Nonviral gene therapy: the promise of genes as pharmaceutical products. Hum. Gene. Ther. 6, 1129-44.
20. Ledley, F.D. (1996) Pharmaceutical approach to somatic gene therapy. Pharm. Res. 13, 1595-614.
21. Lee, R.J. and Huang, L. (1996) Folate-targeled, anionic liposome-entrapped polylysine-condensed DNA for tumor cell-specific gene transfer. J. Biol. Chem. 271, 8481-7.
22. Lee, R.J. and Low, P.S. (1994) Delivery of liposomes into cultured KB cells via folate receptor-mediated endocytosis. J. Biol. Chem. 269, 3198-204.
23. Lentz, M.J. and Kim, S.W. (1998) Terplex system for delivery of DNA and oligonucleotides. In: A.V. Kabanov, P.L. Felgner and L.W. Seymour (Eds.), Self Assembling Complexes for Gene Delivery. John Wiley & Sons Ltd., New York.
24. Maruyama, A., Ishihara, T., Adachi, N. and Akaike, T. (1994) Preparation of nanoparticles hearing high density carbohydrate chains using carbohydrate-carrying polymers as emulsifier. Biomaterials 15, 1035-42.
25. Maruyama, A., Ishihara, T., Kim, J.S., Kim, S.W. and Akaike, T. (1997) Nanoparticle DNA carrier with poly(L-lysine) grafted polysaccharide copolymer and poly(D.L-lactic acid). Bioconjug. Chem. 8, 735-42.
26. Mislick, K.A., Baldeschwieler, J.D., Kayyem, J.F. and Meade, T.J. (1995) Translation of folate polylysine DNA complexes: evidence for lysosomal delivery. Bioconjug. Chem. 6, 512-15.
27. Mulligan, R.C. (1993) The basic science of gene therapy. Science 260, 926-32.
28. Nishikawa, M., Takemura, S., Takakura, Y. and Hashida, M. (1998) Targeted delivery of plasmid DNA to hepatocytes in vivo: optimization of the pharmacokinetics of plasmid DNA/galactosylated poly(L-lysine) complexes by controlling their physicochemical properties. J. Pharmacol. Exp. Ther. 287, 408-15.
29. Park, S.H., Bae, Y.M., Kwon, H.J., Kim, T.J., Kim, J., Lee, S.J. and Lee, S.K. (1993) JL1, a novel differentiation antigen of human cortical thymocyte. J. Exp. Med, 178, 1447-51.
30. Phillips, M.L., Nudelman, E., Gaeta, F.C., Perez, M., Singhal, A.K., Hakomori, S. and Paulson, J.C. (1990) ELAM-I mediates cell adhesion by recognition of a carbohydrate ligand, sialyl-Lex, Science 250, 1130-2.
31. Plank, C., Zatloukal, K., Cotten, M., Mechtler, K. and Wagner, F. (1992) Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconjug. Chem. 3, 533-9.
32. Plank, C., Oberhauser, B., Mechtler, K., Koch, G. and Wagner, E. (1994) The influence of endosome-disruptive peptides on gene transfer using synthetic virus-like gene transfer systems. J. Biol. Chem. 269, 12918-24.
33. Ross, J.F., Chaudhuri, P.K. and Ratnam, M. (1994) Differential regulation of folate receptor isoforms in normal and malignant tissues in vivo and in established cell lines. Physiologic and clinical implications. Cancer 73, 2432-43.
34. Suh, W.H. and Kim, S.W. (1998) Gene delivery system targeted to JL1 antigen of leukemia cells. In: American Society of Gene Therapy, Seattle, WA.
35. Suh, H.R., Koh, J.J. and Kim, S.W. (1999a) Biodistribution and in vivo gene expression of terplex system following intravenous administration in rats and mice (submitted).
36. Suh, W.H., Park, J.S., Chung, J. and Kim, S.W. (1999b) Gene delivery system targeted to JL1 antigen of leukemia cells. (in preparation).
37. Tabin, C.J., Hoffmann, J.W., Goff, S.P. and Weinberg, R.A. (1982) Adaptation of a retrovirus as a eucaryotic vector transmitting the herpes simplex virus thymidine kinase gene. Mol. Cell Biol. 2, 426-36.
38. Wadhwa, M.S., Knoell, D.L., Young, A.P. and Rice, K.G. (1995) Targeted gene delivery with a low molecular weight glycopeptide carrier. Bioconjug. Chem. 6, 283-91.
39. Wagner, E., Zenke, M., Cotten, M., Beug, H. and Birnstiel, M.L. (1990) Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc. Natl. Acad. Sci. USA 87, 3410-14.
40. Wagner, E., Cotten, M., Foisner, R. and Birnstiel, M.L. (1991) Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc. Natl. Acad. Sci. USA 88, 4255-9.
41. Wagner, E., Plank, C., Zatloukal, K., Cotten, M. and Birnstiel, M.L. (1992) Influenza virus hemagglutinin HA-2 N-terminal fusogenic peptides augment gene transfer by transferrin-polylysine DNA complexes: toward a synthetic virus-like gene-transfer vehicle. Proc. Natl. Acad. Sci. USA 89, 7934-8.
42. Wang, S., Lee, R.J., Cauchon, G., Gorenstein, D.G. and Low, P.S. (1995) Delivery of antisense oligodeoxyribonucleotides against the human epidermal growth factor receptor into cultured KB cells with liposomes conjugated to folate via polyethylene glycol. Proc. Natl. Acad. Sci. USA 92, 3318-22.
43. Wang, S. and Low, P.S. (1998) Folate-mediated targeting of antineoplastic drugs, imaging agents, and nucleic acids to cancer cells. J. Controlled Release 53, 39-48.
44. Wang, S., Luo, J., Lantrip, D.A., Waters, D.J., Mathias, C.J., Green, M.A., Fuchs, P.L. and Low, P.S. (1997) Design and synthesis of [111In]DTPA-folate for use as a tumor-targeted radiopharmaceutical. Bioconjug. Chem. 8, 673-9.
45. Weitman, S.D., Lark, R.H., Coney, L.R., Fort, D.W., Frasca, V., Zurawski Jr., V.R. and Kamen, B.A. (1992) Distribution of the folate receptor GP38 in normal and malignant cell lines and tissues. Cancer Res. 52, 3396-401.
46. Wilson, J.M., Grossman, M., Cabrera, J.A., Wu, C.H. and Wu, G. Y. (1992a) A novel mechanism for achieving transgene persistence in vivo after somatic gene transfer into hepatocytes. J. Biol. Chem. 267, 11 483-9.
47. Wilson, J.M., Grossman, M., Wu, C.H., Chowdhury, N.R., Wu, G.Y. and Chowdhury, J.R. (1992b) Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. J. Biol. Chem. 267, 963-7.
48. Wu, C.H., Wilson, J.M. and Wu, G.Y. (1989) Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J. Biol. Chem. 264, 16 985-7.
49. Wu, G.Y. and Wu, C.H. (1987) Receptor-mediated in vitro gene transformation by a soluble DNA carrier system [published erratum appears in J. Biol. Chem. 1988 Jan 5; 263(1): 588]. J. Biol. Chem. 262, 4429-32.
50. Yokoyama, M., Okano, T., Sakurai, Y., Ekimoto, H., Shibazaki, C. and Kataoka, K. (1991) Toxicity and antitumor activity against solid tumors of micelle-forming polymeric anticancer drug and its extremely long circulation in blood. Cancer Res. 51, 3229-36.
51. Zanta, M.A., Belguise-Valladier, P. and Behr, J.P. (1999) Gene delivery: a single nuclear localization signal peptide is sufficient to carry DNA to the cell nucleus. Proc. Natl. Acad. Sci. USA 96, 91-6.
52. Zhou, X.H., Klibanov, A.L. and Huang, L. (1991) Lipophilic polylysines mediate efficient DNA transfection in mammalian cells. Biochim. Biophys. Acta 1065, 8-14.