Design and synthesis of novel amphiphilic dendritic galactosides for selective targeting of liposomes to the hepatic asialoglycoprotein receptor

Journal of Medicinal Chemistry

Volumn 42, Issue 4, 1999, Pages 609-618

  Sliedregt, Leo A J M ^a   Rensen, Patrick C N ^a   Rump, Erik T ^a   Van Santbrink, Peter J ^a   Bijsterbosch, Martin K ^a   Valentijn, A Rob P M ^b   Van Der Marel, Gijs A ^b   Van Boom, Jacques H ^b   Van Berkel, Theo J C ^a   Biessen, Erik A L ^a  

^a LEIDEN UNIVERSITY   (Netherlands)

^b LEIDEN UNIVERSITY   (Netherlands)

Author keywords

[No Author keywords available]

Indexed keywords

ASIALOFETUIN; ASIALOGLYCOPROTEIN RECEPTOR; GALACTOSIDE; LIPOSOME;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CHEMICAL STRUCTURE; CONTROLLED STUDY; DRUG BLOOD LEVEL; DRUG DEGRADATION; DRUG LIVER LEVEL; DRUG TARGETING; DRUG UPTAKE; INTRAVENOUS DRUG ADMINISTRATION; LIPOGENESIS; LIVER CELL; MALE; MOUSE; NONHUMAN; PROTEIN LIPID INTERACTION; RAT;

ANIMALS; ASIALOGLYCOPROTEIN RECEPTOR; ASIALOGLYCOPROTEINS; BINDING, COMPETITIVE; DRUG DESIGN; GALACTOSIDES; GLYCOLIPIDS; LIPOSOMES; LIVER; MALE; MICE; MICE, INBRED C57BL; RATS; RECEPTORS, CELL SURFACE; STRUCTURE-ACTIVITY RELATIONSHIP;

EID: 0033602132     PISSN: 00222623     EISSN: None     Source Type: Journal    
DOI: 10.1021/jm981078h     Document Type: Article

References (42)

1. Ashwell, G.; Harford, J. Carbohydrate-specific receptors on the liver. Annu. Rev. Biochem. 1982, 51, 531-554.
2. Meijer, D. K. F.; Jansen, R. W.; Molema, G. Drug targeting systems for antiviral agents: options and limitations. Antiviral Res. 1992, 18, 215-258.
3. Monsigny, M.; Roche, A.-C.; Midoux, P.; Mayer, R. Glycoconjugates as carriers for specific delivery of therapeutic drugs and genes. Adv. Drug Delivery Rev. 1994, 14, 1-24.
4. Fiume, L.; Busi, C.; Di Stefano, G.; Mattioli, A. Targeting of antiviral drugs to the liver using glycoprotein carriers. Adv. Drug Delivery Rev. 1994, 14, 51-65.
5. Lee, R. T.; Lin, P.; Lee, Y. C. New synthetic cluster ligands for galactose/N-acetylgalactosamine-specific lectin of mammalian liver. Biochemistry 1984, 23, 4255-4261.
6. Lee, R. T.; Lee, Y. C. Preparation of cluster glycosides of N-acetylgalactosamine that have subnanomolar binding constants towards the mammalian hepatic Gal/GalNAc-specific receptor. Glycoconjugate J. 1987, 4, 317-328.
7. Lee, R. T.; Lee, Y. C. Facile synthesis of a high-affinity ligand for mammalian hepatic lectin containing three terminal N-acetylgalactosamine residues. Bioconjugate Chem. 1997, 8, 762-765.
8. Findeis, M. A. Stepwise synthesis of a GalNAc-containing cluster glycoside ligand of the asialoglycoprotein receptor. Int. J. Pept. Protein Res. 1994, 43, 477-485.
9. Valentijn, A. R. P. M.; Van der Marel, G. A.; Sliedregt, L. A. J. M.; Van Berkel, T. J. C.; Biessen, E. A. L.; Van Boom, J. H. Solid-phase synthesis of lysine-based cluster galactosides with high affinity for the asialoglycoprotein receptor. Tetrahedron 1997, 53, 759-770.
10. Kichler, A.; Schuber, F. Versatile synthesis of bi-and triantennary galactose ligands: interaction with the Gal/GalNAc receptor of human hepatoma cells. Glycoconjugate J. 1995, 12, 275-281.
11. Haensler, J.; Szoka, F. C. Synthesis and characterization of a trigalactosylated bisacridine compound to target DNA to hepatocytes. Bioconjugate Chem. 1993, 4, 85-93.
12. Plank, C.; Zatloukal, K.; Cotten, M.; Mechtler, K.; Wagner, E. Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetraantennary galactose ligand. Bioconjugate Chem. 1992, 3, 533.
13. Merwin, J. R.; Noell, G. S.; Thomas, W. L.; Chiou, H. C.; De Rome, M. E.; McKee, T. D.; Spitalny, G. L.; Findeis, M. A. Targeted delivery of DNA using YEE(GalNAcAH)3, a synthetic glycopeptide ligand for the asialoglycoprotein receptor. Bioconjugate Chem. 1994, 5, 612-620.
14. Hangeland, J. J.; Levis, J. T.; Lee, Y. C.; Ts'o, P. O. P. Cell-type specific and ligand specific enhancement of cellular uptake of oligodeoxynucleoside methylphosphonates covalently linked with a neoglycopeptide, YEE(ah-GalNAc)3. Bioconjugate Chem. 1995, 6, 695-701.
15. Kempen, H. J. M.; Hoes, C.; Van Boom, J. H.; Spanjer, H. H.; De Lange, J.; Langendoen, A.; Van Berkel, T. J. C. A water-soluble cholesteryl-containing trisgalactoside: synthesis, properties, and use in directing lipid-containing particles to the liver. J. Med. Chem. 1984, 27, 1306-1312.
16. Spanjer, H. H.; Van Berkel, T. J. C.; Scherphof, G. L.; Kempen, H. J. M. The effect of a water-soluble tris-galactoside terminated cholesterol derivative on the in-vivo fate of small unilamellar vesicles in rats. Biochim. Biophys. Acta 1985, 816, 396.
17. Van Berkel, T. J. C.; Kar Kruijt, J.; Spanjer, H. H.; Nagelkerke, J. F.; Harkes, L.; Kempen, H. J. M. The effect of a water-soluble tris-galactoside-terminated cholesterol derivative on the fate of low-density lipoproteins and liposomes. J. Biol. Chem. 1985, 260, 2694-2699.
18. Spanjer, H. H.; Scherphof, G. L. Targeting of lactosylceramide-containing liposomes to hepatocytes in vivo. Biochim. Biophys. Acta 1983, 734, 40-47.
19. Haensler, J.; Schuber, F. Influence of the galactosyl ligand structure on the interaction of galactosylated liposomes with mouse peritoneal macrophages. Glycoconjugate J. 1991, 8, 116-124.
20. Murahashi, N.; Sasaki, A. In vivo behavior of liposomes modified with a novel galactosyllipid derivative. Biol. Pharm. Bull. 1996, 19, 418-423.
21. Remy, J. S.; Kichler, A.; Mordvinov, V.; Schuber, F.; Behr, J. P. Targeted gene transfer into hepatoma cells with lipopolyamine-condensed DNA particles presenting galactose ligands: a stage toward artificial virusses. Proc. Natl. Acad. Sci. U.S.A. 1995, 92, 1744-1748.
22. Murahashi, N.; Ishihara, H.; Sasaki, A.; Sakagami, M.; Hamana, H. Hepatic accumulation of glutamic acid branched neogalactosyllipid modified liposomes. Biol. Pharm. Bull. 1997, 20, 259-266.
23. Biessen, E. A. L.; Beuting, D. M.; Roelen, H. C. P. F.; Van der Marel, G. A.; Van Boom, J. H.; Van Berkel, T. J. C. Synthesis of cluster galactosides with high affinity for the hepatic asialoglycoprotein receptor. J. Med. Chem. 1995, 38, 1538-1546.
24. Biessen, E. A. L.; Broxterman, H.; Van Boom, J. H.; Van Berkel, T. J. C. The cholesterol derivative of a triantennary galactoside with high affinity for the hepatic asialoglycoprotein receptor: a potent cholesterol lowering agent. J. Med. Chem. 1995, 38, 1846-1852.
25. Biessen, E. A. L.; Vietsch, H.; Van Berkel, T. J. C. Cholesterol derivative of a new triantennary cluster galactoside directs low-and high-density lipoproteins to the parenchymal liver cell. Biochem. J. 1994, 302, 283-289.
26. Newkome, G. R.; Lin, X.; Young, J. K. Syntheses of amine building blocks for dendritic macromolecule construction. Synlett 1992, 53-54.
27. Dourtoglou, V.; Gross, B. O-Benzotriazolyl-N,N,N′,N′-tetramethyluronium hexafluorophosphate as coupling reagent for the synthesis of peptides of biological interest. Synthesis 1984, 572-574.
28. Knorr, R.; Trzeciak, A.; Bannwarth, W.; Gillessen, D. New coupling reagents in peptide chemistry. Tetrahedron Lett. 1989, 30, 1927-1930.
29. Krapcho, A. P.; Kuell, C. S. Monoprotected diamines. N-tert-butoxycarbonyl-α,ω-alkanediamines from α,ω-alkanediamines. Synth. Commun. 1990, 20, 2559-2564.
30. Krieger, M.; McPhaul, M. J.; Goldstein, J. L.; Brown, M. S. Replacement of neutral lipids of low-density lipoprotein with esters of long chain unsaturated fatty acids. J. Biol. Chem. 1979, 254, 3845-3853.
31. Firestone, R. A.; Pisano, J. M.; Falck, J. R.; McPhaul, M. M.; Krieger, M. Selective delivery of cytotoxic compounds to cells by the LDL pathway. J Med. Chem. 1984, 27, 1037-1043.
32. Dubowchik, G. M.; Firestone, R. A. Improved cytotoxicity of antitumor compounds deliverable by the LDL pathway. Bioconjugate Chem. 1995, 6, 427-439.
33. Rump, E. T.; De Vrueh, R. L. A.; Sliedregt, L. A. J. M.; Biessen, E. A. L.; Van Berkel, T. J. C.; Bijsterbosch, M. K. Preparation of conjugates of oligodeoxynucleotides and lipid structures and their interaction with low-density lipoprotein. Bioconjugate Chem. 1998, 9, 341-349.
34. Sliedregt, L. A. J. M.; Biessen, E. A. L. Unpublished results.
35. Rensen, P. C. N.; Schiffelers, R. M.; Versluis, A. J.; Bijsterbosch, M. K.; Van Kuijk-Meuwissen, M. E. M. J.; Van Berkel, T. J. C. Human recombinant apolipoprotein E-enriched liposomes can mimic low-density lipoproteins as carriers for the site-specific delivery of antitumor reagents. Mol. Pharmacol. 1897, 52, 445-455.
36. Van Berkel, T. J. C.; Kruijt, J. K.; Kempen, H. J. M. Specific targeting of high-density lipoproteins to liver hepatocytes by incorporation of a tris-galactoside-terminated cholesterol derivative. J. Biol. Chem. 1985, 260, 12203-12207.
37. Biessen, E. A. L.; Bakkeren, H. F.; Beuting, D. M.; Kuiper, J.; Van Berkel, T. J. C. Ligand size is a major determinant of high-affinity binding of fucose-and galactose-exposing (lipo)proteins by the hepatic fucose receptor. Biochem. J. 1994, 299, 291-296.
38. Goswami, S. K.; Frey, C. F. Manganous chloride spray reagent for cholesterol and bile acids on thin-layer chromatograms. J. Chromatogr. 1970, 53, 389-390.
39. Von Arx, E.; Faupel, M.; Brugger, M. Das 4,4′-tetramethyldiamino-diphenylmethan reagens (TDM); eine modifikation der chlor-o-tolidin farbereaktion für die dünnschicht-chromatographie. J. Chromatogr. 1976, 120, 224-228.
40. Redgrave, T. G.; Roberts, D. C. K.; West, C. E. Separation of plasma lipoproteins by density-gradient ultracentrifugation. Anal. Biockem. 1975, 65, 42-49.
41. Rensen, P. C. N.; Van Dijk, M. C. M.; Havenaar, E. C.; Bijsterbosch, M. K.; Kruijt, J. K.; Van Berkel, T. J. C. Selective liver targeting of antivirals by recombinant chylomicrons - a new therapeutic approach to hepatitis B. Nature Med. 1995, 1, 221-225.
42. Rensen, P. C. N.; Herijgers, N.; Netscher, M. H.; Meskers, S. C. J.; Van Eck, M.; Van Berkel, T. J. C. Particle size determines the specificity of apolipoprotein E-containing triglyceride-rich emulsions for the LDL receptor versus hepatic remnant receptor in vivo. J. Lipid Res. 1997, 38, 1070-1084.