In vivo targeted gene transfer into liver cells mediated by a novel galactosyl-D-lysine/D-serine copolymer

Gene Therapy

Volumn 6, Issue 4, 1999, Pages 689-693

  Hisayasu, S ^a   Miyauchi, M ^a   Akiyama, K ^b   Gotoh, T ^b   Satoh, S ^b   Shimada, T ^a  

^a NIPPON MEDICAL SCHOOL   (Japan)

^b HISAMITSU PHARMACEUTICAL CO INC   (Japan)

Author keywords

Asialoglycoprotein receptor; D lysine D serine copolymer; Gene transfer; Liver

Indexed keywords

ASIALOGLYCOPROTEIN RECEPTOR; COPOLYMER; GREEN FLUORESCENT PROTEIN; LUCIFERASE; MACROGOL; PLASMID DNA; POLYPEPTIDE;

ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; COMPLEX FORMATION; CONTROLLED STUDY; DNA BINDING; ENZYME ACTIVITY; GENE TRANSFER; HEART; INTRAVENOUS DRUG ADMINISTRATION; KIDNEY; LIVER CELL; LUNG; MOUSE; NONHUMAN; PRIORITY JOURNAL; REPORTER GENE; SPLEEN;

ANIMALS; FEMALE; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; LIVER; MICE; POLYMERS;

ANIMALIA;

EID: 0032962980     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300868     Document Type: Article

References (21)

1. Wu GY, Wu CH. Receptor-mediated in vitro gene transformation by a soluble DNA carrier system. J Biol Chem 1987; 262: 4429-4432.
2. Wu GY, Wu CH. Receptor-mediated gene delivery and expression in vivo. J Biol Chem 1988; 263: 14621-14624.
3. Wu GY, Wu CH. Evidence for targeted gene delivery to HepG2 hepatoma cells in vitro. Biochemistry 1988; 27: 887-892.
4. Wagner E et al. Transferrin-polycation conjugates as carriers for DNA uptake into cells. Proc Natl Acad Sci USA 1990; 87: 3410-3414.
5. Plank C et al. Gene transfer into hepatocytes using asialoglycoprotein receptor mediated endocytosis of DNA complexed with an artificial tetra-antennary galactose ligand. Bioconj Chem 1992; 3: 533-539.
6. 3, a synthetic glycopeptide ligand for the asialoglycoprotein receptor. Bioconj Chem 1994; 5: 612-620.
7. Perales JC et al. Gene transfer in vivo: sustained expression and regulation of genes introduced into the liver by receptor-targeted uptake. Proc Natl Acad Sci USA 1994; 91: 4086-4090.
8. Fominaya J, Wels W. Target cell-specific DNA transfer mediated by a chimeric multidomain protein. Novel non-viral gene delivery system. J Biol Chem 1996; 271: 10560-10568.
9. Harbottle RP et al. An RGD-oligolysine peptide: a prototype construct for integrin-mediated gene delivery. Hum Gene Ther 1998; 9: 1037-1047.
10. Wagner E, Cotten M, Foisner R, Birnstiel ML. Transferrin-polycation-DNA complexes: the effect of polycations on the structure of the complex and DNA delivery to cells. Proc Natl Acad Sci USA 1991; 88: 4255-4259.
11. Perales JC et al. Biochemical and functional characterization of DNA complexes capable of targeting genes to hepatocytes via the asialoglycoprotein receptor. J Biol Chem 1997; 272: 7398-7407.
12. Katchalski E, Sela M. Synthesis and chemical properties of poly-α-amino acids. II. Synthesis of poly-α-amino acids from N-carboxy-α-amino acid anhydrides (NCAs). Adv Protein Chem 1958; 13: 244-330.
13. Monsigny M, Roche A-C, Midoux P. Uptake of neoglycoproteins via membrane lectin(s) of L1210 cells evidenced by quantitative flow cytofluorometry and drug targeting. Biol Cell 1984; 51: 187-196.
14. Monsigny M, Pett C, Roche A-C. Colorimetric determination of neutral sugars by a resorcinol sulfuric acid micromethod. Anal Biochem 1988; 175: 525-530.
15. Niwa H, Yamamura K, Miyazaki J. Efficient selection for high- expression transfectants with a novel eukaryotic vector. Gene 1991; 108: 193-200.
16. Pinkert CA, Ornitz DM, Brinster RL, Palmiter RD. An albumin enhancer located 10 kb upstream functions along with its promoter to direct efficient liver-specific expression in transgenic mice. Genes Dev 1987; 1: 268-276.
17. Wilson JM et al. Hepatocyte-directed gene transfer in vivo leads to transient improvement of hypercholesterolemia in low density lipoprotein receptor-deficient rabbits. J Biol Chem 1992; 267: 963-967.
18. Stankovics J et al. Overexpression of human methylmalonyl CoA mutase in mice after in vivo gene transfer with asialoglycoprotein/polylysine/DNA complexes. Hum Gene Ther 1994; 5: 1095-1104.
19. Wu CH, Wilson JM, Wu GY. Targeting genes: delivery and persistent expression of a foreign gene driven by mammalian regulatory elements in vivo. J Biol Chem 1989; 264: 16985-16987.
20. Wu GY et al. Receptor-mediated gene delivery in vivo. Partial correction of genetic analbuminemia in Nagase rats. J Biol Chem 1991; 266: 14338-14342.
21. Ferkol T et al. Regulation of the phosphoenolpyruvate carboxykinase/human factor IX gene introduced into the livers of adult rats by receptor-mediated gene transfer. FASEB J 1993; 7: 1081-1091.