Liver-selective DNA targeting using the asialoglycoprotein receptor

Biogenic Amines

Volumn 14, Issue 5, 1998, Pages 499-518

  Ruiz, J ^a   Wu, G Y ^a,b  

^a UNIVERSITY OF NAVARRA   (Spain)

^b UNIVERSITY OF CONNECTICUT HEALTH CENTER   (United States)

Author keywords

Asialoglycoprotein receptor; Asialoorosomucoid; DNA protein complexes; Endosomolytic agents; Fusogenic peptides; Gene therapy; Hepatocyte; Receptor mediated endocytosis

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; COMPLEMENTARY DNA; DNA;

ANALBUMINEMIA; DNA STRUCTURE; ENDOCYTOSIS; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; HEMOPHILIA; HEPATITIS B; HEPATITIS C; HUMAN; HYPERBILIRUBINEMIA; HYPERCHOLESTEROLEMIA; LIGAND BINDING; LIVER DISEASE; NONHUMAN; REVIEW;

EID: 0031668004     PISSN: 01688561     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (58)

1. Bartholomew, R.M., Carmichael, E.P., Findeis, M.A., Wu, C.H. and Wu, G.Y. (1995). Targeted delivery of antisense DNA in woodchuck hepatitis virus-infected woodchucks. J. Viral. Hepatitis, 2: 273-278.
2. Bijsterbosch, M.K. and Van Berkel, T.J. (1992). Lactosylated high density lipoprotein: a potential carrier for the site-specific delivery of drugs to parenchymal liver cells. Mol. Pharmacol., 41: 404-411.
3. Bommineni, V.R., Chowdhury, N.R., Wu, G.Y., Wu, C.H., Franki, N., Hays, R.M. and Chowdhury J.R. (1994). Depolymerization of hepatocellular microtubules after partial hepatectomy. J. Biol. Chem., 269: 25200-25205.
4. Boussif, O., Lezoualc'h, F., Zanta, M.A., Mergny, M.D., Scherman, D., Demeneix, B. and Behr, J.P. (1995). A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc. Natl. Acad. Sci. USA, 92: 7297-7301.
5. Chen, J., Stickles, R.J. and Daichendt, K.A. (1994). Galactosylated histone-mediated gene transfer and expression. Hum. Gene Ther., 5: 429-435.
6. Chiou, H.C., Tangco, M., Levine, S., 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 Acid Res., 22: 5439-5449.
7. Chowdhury, N.R., Wu, C.H., Wu, G.Y., Yerneni, P.C., Bommineni, V.R. and Chowdhury, J.R. (1993). Fate of DNA targeted to the liver by asialoglycoprotein receptor-mediated endocytosis in vivo. Prolonged persistence in cytoplasmic vesicles after partial hepatectomy. J. Biol. Chem., 268: 11265-11271.
8. Chowdhury, N.R., Hays, R.M., Bommineni, V.R., Franki, N., Chowdhury, J.R., Wu, C.H. and Wu, G.Y. (1996). Microtubular disruption prolongs the expression of human bilirubin-uridinediphosphoglucuronate-glucuronosyltransferase-1 gene transferred into Gunn rat livers. J. Biol. Chem., 271: 2341-2346.
9. Cotten, M., Langle Rouault, F., Kirlappos, H., Wagner, E., Mechtler, K., Zenke, M., Beugh, H. and Birnstiel, M.L. (1990). Transferrin-polycation-mediated introduction of DNA into human leukemic cells: stimulation by agents that affect the survival of transfected DNA or modulate transferrin receptor levels. Proc. Natl. Acad. Sci. USA, 87: 4033-4037.
10. Cotten, M., Wagner, E., Zatloukal, K., Phillips, S., Curiel, D.T. and Birnstiel, M.L. (1992). High-efficiency receptor-mediated delivery of small and large (48 kilobase) gene constructs using the endosome-disruption activity of defective or chemically inactivated adenovirus particles. Proc. Natl. Acad. Sci. USA, 89: 6094-6098.
11. Cristiano, R.J., Smith, L.C. and Woo, S.L.C. (1993a). Hepatic gene therapy: adenovirus enhancement of receptor-mediated gene delivery and expression in primary hepatocytes. Proc. Natl. Acad. Sci. USA, 90: 2122-2126.
12. Cristiano, R.J., Smith, L.C., Kay, M.A., Brinkley, B. and Woo, S.L. (1993b). Hepatic gene therapy: efficient gene delivery and expression in primary hepatocytes utilizing a conjugated adenovirus-DNA complex. Proc. Natl. Acad. Sci. USA, 90: 11548-11552.
13. Curiel, D.T., Agarwal, S., Wagner, E. and Cotten, M. (1991). Adenovirus enhancement of transferrin-polylysine-mediated gene delivery. Proc. Natl. Acad. Sci. USA, 88: 8850-8854.
14. Edwards, R.J., Carpenter, D.S. and Minchin, R.F. (1996). Uptake and intracellular trafficking of asialoglycoprotein-polylysine-DNA complexes in isolated rat hepatocytes. Gene Ther., 3: 937-940.
15. Ferkol, T., Lindberg, G.L., Chen, J., Perales, J.C., Crawford, D.R., Ratnoff, O.D. and Hanson R.W. (1993). Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASEB J., 7: 1081-1091.
16. Fiume, L., Mattioli, A., Balboni, P.G., Tognon, M., Barbanti-Brodano, G., De Vries, J. and Wieland T. (1979). Enhanced inhibition of virus DNA synthesis in hepatocytes by trifluorothymidine coupled to asialofetuin. FEBS Lett., 103: 47-51.
17. Fiume, L., Mattioli, A., Busi, C., Balboni, P.G., Barbanti-Brodano, G., De Vries, J., Altmann, R. and Wieland T. (1980). Selective inhibition of ectromelia virus DNA synthesis in hepatocytes by adenine-9-β-D-arabinofuranoside (ara-A) and adenine-9- β-D-arabinofuranoside 5′-monophosphate (ara-AMP) conjugated to asialofetuin. FEBS Lett., 116: 185-188.
18. Haensler, J. and Szoka, F.C. (1993). Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjug. Chem., 4: 85-93.
19. Hara, T., Aramaki, Y., Takada, S., Koike, K. and Tsuchiya, S. (1995a). Receptor-mediated transfer of pSV2CAT DNA to a human hepatoblastoma cell line HepG2 using asialofetuin-labeled cationic liposomes. Gene, 159: 167-174.
20. Hara, T., Aramaki, Y., Takada, S., Koike, K. and Tsuchiya S. (1995b). Receptor-mediated transfer of pSV2CAT DNA to mouse liver cells using asialofetuin-labeled liposomes. Gene Ther., 2: 784-788.
21. Hara, T., Kuwasawa, H., Aramaki, Y., Takada, S., Koike, K., Ishidate, K., Kato, H. and Tsuchiya, S. (1996). Effects of fusogenic and DNA-binding amphiphilic compounds on the receptor-mediated gene transfer into hepatic cells by asialofetuin-labeled liposomes. Biochim. Biophys. Acta, 1278: 51-58.
22. Koike, K., Hara, T., Aramaki, Y., Takada, S. and Tsuchiya, S. (1994). Receptor-mediated gene transfer into hepatic cells using asialoglycoprotein-labeled liposomes. Ann. N. Y. Acad. Sci., 716: 331-333.
23. Kren, B.T., Bandyopadhyay, P. and Steer C. (1998). In vivo site-directed mutagenesis of the factor IX gene by chimeric RNA/DNA oligonucleotides. Nature Med., 4: 285-290.
24. Lee, Y.C., Townsend, R.R., Hardy, M.R., Lonngren, J., Arnarp, J., Haraldsson, M. and Lonn, J. (1983). Binding of synthetic oligosaccharides to the hepatic Gal/GalNAc lectin. Dependence on fine structural features. J. Biol. Chem., 258: 199-202.
25. Mack, K.D., Walzem, R. and Zeldis, J.B. (1994). Cationic lipid enhances in vitro receptor-mediated transfection. Am. J. Med. Sci., 307: 138-143
26. Martinez-Fong, D., Mullersman, J.E., Purchio, A.F., Armendariz-Borunda, J. and Martinez-Hernandez, A. (1994). Nonenzymatic glycosylation of poly-L-lysine: a new tool for targeted gene delivery. Hepatology, 20: 1602-1608.
27. Merwin, J.R., Noell, G.S., Thomas, W.L., Chiou, H.C., DeRome, M.E., McKee, T.D., Spitalny, G.L. and Findeis, M.A. (1994). Targeted delivery of DNA using YEE(GalNAcAH)3, a synthetic glycopeptide ligand for the asialoglycoprotein receptor. Bioconjug. Chem., 5: 612-620.
28. Nakazono, K., Ito, Y., Wu, C.H. and Wu, G.Y. (1996). Inhibition of hepatitis B virus replication by targeted pretreatment of complexed antisense DNA in vitro. Hepatology, 23: 1297-1303.
29. Perales, J.C., Ferkol, T., Molas, M. and Hanson, R.W. (1994a). An evaluation of receptor-mediated gene transfer using synthetic DNA-ligand complexes. Eur. J. Biochem., 226: 255-266.
30. Perales, J.C., Ferkol, T., Beegen, H., Ratnoff, O.D. and Hanson, R.W. (1994b). Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc. Natl. Acad. Sci. USA., 91: 4086-4090.
31. Perales, J.C., Grossmann, G.A., Molas, M., Liu, G., Ferkol, T., Harpst, J., Oda, H. and Hanson, R.W. (1997). Biochemical and functional characterization of DNA complexes capable of targeting genes to hepatocytes via the asialoglycoprotein receptor. J. Biol. Chem., 272: 7398-7407.
32. Plank, C., Zatloukal, K., Cotton, M., Mechtler, K. and Wagner, E. (1992). Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconjug. Chem., 3: 533-539.
33. Remy, J.S., Kichler, A., Mordvinov, V., Schuber, F. and Behr, J.P. (1995). Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial viruses. Proc. Natl. Acad. Sci. USA, 92: 1744-1748.
34. Rice, K.,G. and Lee, Y.C. (1990). Modification of triantennary glycopeptides into probes for the asialoglycoprotein receptor of hepatocytes. J. Biol. Chem., 265: 18423-18428.
35. Rice, K.,G., Weisz, O.A., Barthel, T., Lee, R.T. and Lee, Y.C. (1990). Defined geometry of binding between triantennary glycopeptides and the asialoglycoprotein receptor of rat hepatocytes. J. Biol. Chem., 265: 18429-18434.
36. Schlepper-Schafer, J., Hulsmann, D., Djovkar, A., Meyer, H.E., Herbertz, L., Kolb, H. and Kolb-Bachofen, V. (1986). Endocytosis via galactose receptors in vivo. Ligand size directs uptake by hepatocytes and/or liver macrophages. Exp. Cell Res., 165: 494-506.
37. Schwartz, A.L., Fridovich, S.E. and Lodish, H.F. (1982). Kinetics of internalization and recycling of the asialoglycoprotein receptor in a hepatoma cell line. J. Biol. Chem., 257: 4230-4237.
38. Stankovics, J., Crane, A.M., Andrews, E., Wu, C.H., Wu, G.Y. and Ledley, F.D. (1994). Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine /DNA complexes. Hum. Gene Ther., 5: 1095-1104.
39. Stockert, R.J. (1995). The asialoglycoprotein receptor: relationships between structure, function, and expression. Physiol. Rev., 75: 591-609.
40. Townsend, R.R., Hardy, M.R., Wong, T.C. and Lee, Y.C. (1986). Binding of N-linked bovine fetuin glycopeptides to isolated rabbit hepatocytes: Gal/GalNAc hepatic lectin discrimination between Gal β(1,4)GlcNAc and Gal β(1,3)GalcNAc in a triantennary structure. Biochemistry, 25: 5716-5725.
41. Trouet, A., Baurain, R., Deprez de Campeneere, D., Masquelier, M. and Pirson, P. (1981). Targeting of antitumor and antiprotozoal drugs by covalent linkage to protein carriers. In: Targeting of Drugs, G. Gregoriadis, J. Senior and A. Trouet (Eds.), Plenum, New York, pp. 28.
42. Vera, D.R., Stadalnik, R.C. and Krohn, K.A. (1985). Technetium 99m galactosyl-neoglycoalbumin: preparation and preclinical studies. J. Nucl. Med., 26: 1157-1167.
43. 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: 283-291.
44. 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: 7934-7938.
45. Wilson, J.M., Grossman, M., Cabrera, J.A., Wu, C.H. and Wu, G.Y. (1992). A novel mechanism for achieving transgene persistence in vivo following somatic gene transfer into hepatocytes. J. Biol. Chem., 267: 11483-11489.
46. Wu, G.Y., Wu, C.H. and Stockert, R.J. (1983). A model for the specific rescue of normal hepatocytes during methotrexate treatment of hepatic malignancy. Proc. Natl. Acad. Sci. USA, 80: 3878-3880.
47. Wu, G.Y., Wu, C.H. and Rubin, M.I. (1985). Acetaminophen hepatotoxicity and targeted rescue: a model for specific chemotherapy for hepatocellular carcinoma. Hepatology., 5: 709-713.
48. Wu, G.Y. and Wu, C.H. (1987). Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J. Biol. Chem., 262: 4429-4432.
49. Wu, G.Y. and Wu C.H. (1988a). Evidence for targeted gene delivery to Hep G2 hepatoma cells in vitro. Biochemistry, 27: 887-892.
50. Wu, G.Y. and Wu, C.H. (1988b). Receptor-mediated gene delivery and expression in vivo. J. Biol. Chem., 263: 14621-14624.
51. Wu, G.Y., Wilson, J.M. and Wu, C.H. (1989). Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J. Biol. Chem., 264: 16985-16987.
52. Wu, G.Y., Wilson, J.M., Shalaby, F., Grossman, M., Shafritz, D.A. and Wu, C.H. (1991). Receptor-mediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats. J. Biol. Chem., 266: 14338-14342.
53. Wu, G.Y. and Wu, C.H. (1992). Specific inhibition of hepatitis B viral gene expression in vitro by targeted antisense oligonucleotides. J. Biol. Chem., 267: 12436-12439.
54. Wu, G.Y., Zhan, P., Sze, L.L., Rosenberg, A.R. and Wu, C.H. (1994). Incorporation of adenovirus into a ligand-based DNA carrier system results in retention of original receptor specificity and enhances targeted gene expression. J. Biol. Chem., 269: 11542-11546.
55. Wu, C.H., Zhan, P., Carmichael, E., Chowdhury, J.R., Chowdhury, N.R. and Wu, G.Y. (1997). A tri-component DNA carrier: progress toward a synthetic virus. Hepatology., 26 (suppl.): 129A.
56. Wu, C.H. and Wu, G.Y. (1998). Targeted inhibition of hepatitis C virus-directed gene expression in human hepatoma cell lines. Gastroenterology, 114: 1304-1312.
57. Zanta, M.A., Boussif, O., Adib, A. and Behr, J.P. (1997). In vitro gene delivery to hepatocytes with galactosylated polyethylenimine. Bioconjug. Chem., 8: 839-844.
58. Zhang, Y., Wu, C.H. and Wu, G.Y. (1994). Enhancement of targeted gene expression by incorporation of a listerial lytic peptide into protein-DNA complexes. Hepatology, 20 (suppl.): 271A.