Glyco-engineering strategies for the development of therapeutic enzymes with improved efficacy for the treatment of lysosomal storage diseases

키워드

참고문헌 (38)

1. Ohashi T, "Enzyme replacement therapy for lysosomal storage diseases", Pediatr Endocrinol Rev, 10(1), pp.26~34 (2012)
2. Ortolano S, "Treatment of lysosomal storage diseases: recent patents and future strategies", Recent Pat Endocr Metab Immune Drug Discov, 8, pp.9~25 (2014)
3. Baldo BA, "Enzymes approved for human therapy:indications, mechanisms and adverse effects", BioDrugs, 29, pp.31~55 (2015)
4. Mechler K, "Pressure for drug development in lysosomal storage disorders-a quantitative analysis thirty years beyond the US orphan drug act", Orphanet J Rare Dis, 10, pp.46~ (2015)
5. Braulke T, "Sorting of lysosomal proteins", Biochim Biophys Acta, 1793, pp.605~614 (2009)
6. Kim JJ, "Carbohydrate recognition by the mannose-6-phosphate receptors", Curr Opin Struct Biol, 19, pp.534~542 (2009)
7. Coutinho MF, "Mannose-6-phosphate pathway: a review on its role in lysosomal function and dysfunction", Mol Genet Metab, 105, pp.542~550 (2012)
8. Tiede S, "Mucolipidosis II is caused by mutations in GNPTA encoding the alpha", beta GlcNAc-1-phosphotransferase, 2005, pp.11, 1109~1112
9. are encoded by a single cDNA, "J Biol Chem", 2005, 36141~36149
10. Qian Y, "Functions of the alpha, beta, and gamma subunits of UDP-GlcNAc:lysosomal enzyme N-acetylglucosamine-1-phosphotransferase", J Biol Chem, 285, pp.3360~3370 (2010)
11. Sommerlade HJ, "Glyco sylation and phosphorylation of arylsulfatase A", J Biol Chem, 269, pp.20977~20981 (1994)
12. Lee K, "A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease", Glycobiology, 13, pp.305~313 (2003)
13. Sakuraba H, "Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice", J Hum Genet, 51, pp.180~188 (2006)
14. 손영수, "Enhanced sialylation and in vivo efficacy of recombinant human α-galactosidase through in vitro glycosylation", BMB Reports, 46(3), pp.157~162 (2013), 생화학분자생물학회
15. Vedder AC, "Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg", kg, 2007, pp.2, e598~
16. Togawa T, "Comparative study on mannose 6-phosphate residue contents of recombinant lysosomal enzymes", Mol Genet Metab, 111, pp.369~373 (2014)
17. Lauer RM, "Administration of a mixture of fungal glucosidases to a patient with type II glycogenosis(Pompe's disease)", Pediatrics, 42, pp.672~676 (1968)
18. de Barsy T, "Enzyme replacement in Pompe disease: an attempt with purified human acid alpha-glucosidase", Birth Defects Orig Artic Ser, 9, pp.184~190 (1973)
19. Van den Hout JM, "Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk", Pediatrics, 113, pp.448~457 (2004)
20. Kishnani PS, "Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease", J Pediatr, 149, pp.89~97 (2006)
21. McVie-Wylie AJ, "Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease", Mol Genet Metab, 94, pp.448~455 (2008)
22. Tekoah Y, "Glycosylation and functionality of recombinant beta-glucocerebrosidase from various production systems", Biosci Rep, 33 (2013)
23. Grabowski GA, "Taliglucerase alfa: an enzyme replacement therapy using plant cell expression technology", Mol Genet Metab, 112, pp.1~8 (2014)
24. Berger J, "The uptake of recombinant glucocerebrosidases by blood monocytes from type 1 Gaucher disease patients is variable", Br J Haematol, 157, pp.274~277 (2012)
25. Van Patten SM, "Effect of mannose chain length on targeting of glucocerebrosidase for enzyme replacement therapy of Gaucher disease", Glycobiology, 17, pp.467~478 (2007)
26. Koeberl DD, "Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle", Mol Genet Metab, 103, pp.107~112 (2011)
27. Chavez CA, "Domain 5 of the cation-independent mannose 6-phosphate receptor preferentially binds phosphodiesters(mannose 6-phosphate N-acetylglucosamine ester)", Biochemistry, 46, pp.12604~12617 (2007)
28. Zhu Y, "Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice", J Biol Chem, 279, pp.50336~50341 (2004)
29. Zhu Y, "Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice", Biochem J, 389, pp.619~628 (2005)
30. Zhu Y, "Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease", Mol Ther, 17, pp.954~963 (2009)
31. Zhou Q, "Strategies for Neoglycan conjugation to human acid alpha-glucosidase", Bioconjug Chem, 22, pp.741~751 (2011)
32. Zhou Q, "Glycan structure determinants for cation-independent mannose 6-phosphate receptor binding and cellular uptake of a recombinant protein", Bioconjug Chem, 24, pp.2025~2035 (2013)
33. Kishnani PS, "New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond", Pediatr Endocrinol Rev, 12(1), pp.114~124 (2014)
34. Chiba Y, "Production in yeast of alpha-galactosidase A, a lysosomal enzyme applicable to enzyme replacement therapy for Fabry disease", Glycobiology, 12, pp.821~828 (2002)
35. Akeboshi H, "Production of human beta-hexosaminidase A with highly phosphorylated N-glycans by the overexpression of the Ogataea minuta MNN4 gene", Glycobiology, 19, pp.1002~1009 (2009)
36. Tsukimura T, "Efficient uptake of recombinant alpha-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneys", Mol Med, 18, pp.76~82 (2012)
37. Tiels P, "A bacterial glycosidase enables mannose-6-phosphate modification and improved cellular uptake of yeast-produced recombinant human lysosomal enzymes", Nat Biotechnol, 30, pp.1225~1231 (2012)
38. Gil JY, "Increased mannosylphosphorylation of N-glycans by heterologous expression of YlMPO1 in glyco-engineered Saccharomyces cerevisiae for mannose-6-phosphate modification", J Biotechnol, 206, pp.66~74 (2015)

관련 논문AI추천