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Volumn 31, Issue 4, 2014, Pages 309-315

A biochemical and physicochemical comparison of two recombinant enzymes used for enzyme replacement therapies of hunter syndrome

Author keywords

Enzyme replacement therapy; Formylglycine; Hunter syndrome; Iduronate 2 sulfatase; Mannose 6 phosphate; MPS II

Indexed keywords

FORMYLGLYCINE; GENERIC DRUG; GLYCINE; HUNTERASE; IDURONATE 2 SULFATASE; MANNOSE 6 PHOSPHATE; SIALIC ACID; UNCLASSIFIED DRUG; ALANINE; C(ALPHA)-FORMYLGLYCINE; HYBRID PROTEIN; MANNOSE PHOSPHATE; MANNOSE-6-PHOSPHATE; N ACETYLNEURAMINIC ACID;

EID: 84901857363     PISSN: 02820080     EISSN: 15734986     Source Type: Journal    
DOI: 10.1007/s10719-014-9523-0     Document Type: Article
Times cited : (24)

References (27)
  • 1
    • 84896737145 scopus 로고    scopus 로고
    • Evaluation of idursulfase for the treatment of mucopolysaccharidosis II (Hunter syndrome)
    • Maurizio, S.: Evaluation of idursulfase for the treatment of mucopolysaccharidosis II (Hunter syndrome). Expert Opin Orphan Drugs 1(1), 89-98 (2013)
    • (2013) Expert Opin Orphan Drugs , vol.1 , Issue.1 , pp. 89-98
    • Maurizio, S.1
  • 5
    • 38849176942 scopus 로고    scopus 로고
    • Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome)
    • doi:10.1542/peds.2007-1350
    • Martin, R., Beck, M., Eng, C., Giugliani, R., Harmatz, P., Munoz, V., Muenzer, J.: Recognition and diagnosis of mucopolysaccharidosis II (Hunter syndrome). Pediatrics 121(2), e377-386 (2008). doi:10.1542/peds.2007-1350
    • (2008) Pediatrics , vol.121 , Issue.2
    • Martin, R.1    Beck, M.2    Eng, C.3    Giugliani, R.4    Harmatz, P.5    Munoz, V.6    Muenzer, J.7
  • 6
    • 0020400761 scopus 로고
    • A clinical and genetic study of Hunter 's syndrome. 1. Heterogeneity
    • Young, I.D., Harper, P.S., Archer, I.M., Newcombe, R.G.: A clinical and genetic study of Hunter 's syndrome. 1. Heterogeneity. J Med Genet 19(6), 401-407 (1982)
    • (1982) J Med Genet , vol.19 , Issue.6 , pp. 401-407
    • Young, I.D.1    Harper, P.S.2    Archer, I.M.3    Newcombe, R.G.4
  • 7
    • 84875017970 scopus 로고    scopus 로고
    • Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome)
    • doi:10.1186/1750-1172-8-42
    • Sohn, Y.B., Cho, S.Y., Park, S.W., Kim, S.J., Ko, A.R., Kwon, E.K., Han, S.J., Jin, D.K.: Phase I/II clinical trial of enzyme replacement therapy with idursulfase beta in patients with mucopolysaccharidosis II (Hunter syndrome).Orphanet J Rare Dis 8, 42 (2013). doi:10.1186/1750-1172-8-42
    • (2013) Orphanet J Rare Dis , vol.8 , pp. 42
    • Sohn, Y.B.1    Cho, S.Y.2    Park, S.W.3    Kim, S.J.4    Ko, A.R.5    Kwon, E.K.6    Han, S.J.7    Jin, D.K.8
  • 8
    • 33846899175 scopus 로고    scopus 로고
    • A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome)
    • doi:10.1016/j.ymgme.2006.09.001
    • Muenzer, J., Gucsavas-Calikoglu, M., McCandless, S.E., Schuetz, T.J., Kimura, A.: A phase I/II clinical trial of enzyme replacement therapy in mucopolysaccharidosis II (Hunter syndrome). Mol Genet Metab 90(3), 329-337 (2007). doi:10.1016/j.ymgme.2006.09.001
    • (2007) Mol Genet Metab , vol.90 , Issue.3 , pp. 329-337
    • Muenzer, J.1    Gucsavas-Calikoglu, M.2    McCandless, S.E.3    Schuetz, T.J.4    Kimura, A.5
  • 10
    • 0020417388 scopus 로고
    • Multiple forms of iduronate 2-sulphate sulphatase in human tissues and body fluids
    • Archer, I.M., Harper, P.S., Wusteman, F.S.: Multiple forms of iduronate 2-sulphate sulphatase in human tissues and body fluids. Biochim Biophys Acta 708(2), 134-140 (1982)
    • (1982) Biochim Biophys Acta , vol.708 , Issue.2 , pp. 134-140
    • Archer, I.M.1    Harper, P.S.2    Wusteman, F.S.3
  • 11
    • 0030928564 scopus 로고    scopus 로고
    • Characterization of iduronate sulphatase mutants affecting N-glycosylation sites and the cysteine-84 residue
    • Millat, G., Froissart, R., Maire, I., Bozon, D.: Characterization of iduronate sulphatase mutants affecting N-glycosylation sites and the cysteine-84 residue. Biochem J 326(Pt 1), 243-247 (1997)
    • (1997) Biochem J , vol.326 , Issue.PART 1 , pp. 243-247
    • Millat, G.1    Froissart, R.2    Maire, I.3    Bozon, D.4
  • 12
    • 14844325339 scopus 로고    scopus 로고
    • Analysis of normal and mutant iduronate-2-sulphatase conformation
    • doi:10.1042/bj20040739
    • Parkinson-Lawrence, E., Turner, C., Hopwood, J., Brooks, D.: Analysis of normal and mutant iduronate-2-sulphatase conformation. Biochem J 386(Pt 2), 395-400 (2005). doi:10.1042/bj20040739
    • (2005) Biochem J , vol.386 , Issue.PART 2 , pp. 395-400
    • Parkinson-Lawrence, E.1    Turner, C.2    Hopwood, J.3    Brooks, D.4
  • 13
    • 0037099599 scopus 로고    scopus 로고
    • Mannose 6-phosphate quantitation in glycoproteins using high-pH anion-exchange chromatography with pulsed amperometric detection
    • Zhou, Q., Kyazike, J., Edmunds, T., Higgins, E.: Mannose 6-phosphate quantitation in glycoproteins using high-pH anion-exchange chromatography with pulsed amperometric detection. Anal Biochem 306(2), 163-170 (2002)
    • (2002) Anal Biochem , vol.306 , Issue.2 , pp. 163-170
    • Zhou, Q.1    Kyazike, J.2    Edmunds, T.3    Higgins, E.4
  • 14
    • 0031887174 scopus 로고    scopus 로고
    • Analysis of the N-acetylneuraminic acid and N-glycolylneuraminic acid contents of glycoproteins by high-pH anion-exchange chromatography with pulsed amperometric detection
    • Rohrer, J.S., Thayer, J., Weitzhandler, M., Avdalovic, N.: Analysis of the N-acetylneuraminic acid and N-glycolylneuraminic acid contents of glycoproteins by high-pH anion-exchange chromatography with pulsed amperometric detection. Glycobiology 8(1), 35-43 (1998)
    • (1998) Glycobiology , vol.8 , Issue.1 , pp. 35-43
    • Rohrer, J.S.1    Thayer, J.2    Weitzhandler, M.3    Avdalovic, N.4
  • 15
    • 0034661970 scopus 로고    scopus 로고
    • Analyzing sialic acids using high-performance anion-exchange chromatography with pulsed amperometric detection
    • doi:10.1006/abio.2000.4643
    • Rohrer, J.S.: Analyzing sialic acids using high-performance anion-exchange chromatography with pulsed amperometric detection. Anal Biochem 283(1), 3-9 (2000). doi:10.1006/abio.2000.4643
    • (2000) Anal Biochem , vol.283 , Issue.1 , pp. 3-9
    • Rohrer, J.S.1
  • 16
    • 13444260275 scopus 로고    scopus 로고
    • The absolute quantification strategy: A general procedure for the quantification of proteins and post-translational modifications
    • (San Diego, Calif) doi:10.1016/j.ymeth.2004.08.018
    • Kirkpatrick, D.S., Gerber, S.A., Gygi, S.P.: The absolute quantification strategy: a general procedure for the quantification of proteins and post-translational modifications. Methods (San Diego, Calif) 35(3), 265-273 (2005). doi:10.1016/j.ymeth.2004.08.018
    • (2005) Methods , vol.35 , Issue.3 , pp. 265-273
    • Kirkpatrick, D.S.1    Gerber, S.A.2    Gygi, S.P.3
  • 17
    • 0037795741 scopus 로고    scopus 로고
    • Absolute quantification of proteins and phosphoproteins from cell lysates by tandem MS
    • doi:10.1073/pnas.0832254100
    • Gerber, S.A., Rush, J., Stemman, O., Kirschner, M.W., Gygi, S.P.: Absolute quantification of proteins and phosphoproteins from cell lysates by tandem MS. Proc Natl Acad Sci U S A 100(12), 6940-6945 (2003). doi:10.1073/pnas.0832254100
    • (2003) Proc Natl Acad Sci U S A , vol.100 , Issue.12 , pp. 6940-6945
    • Gerber, S.A.1    Rush, J.2    Stemman, O.3    Kirschner, M.W.4    Gygi, S.P.5
  • 18
    • 0035194407 scopus 로고    scopus 로고
    • A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease)
    • Voznyi, Y.V., Keulemans, J.L., van Diggelen, O.P.: A fluorimetric enzyme assay for the diagnosis of MPS II (Hunter disease). J Inherit Metab Dis 24(6), 675-680 (2001)
    • (2001) J Inherit Metab Dis , vol.24 , Issue.6 , pp. 675-680
    • Voznyi, Y.V.1    Keulemans, J.L.2    Van Diggelen, O.P.3
  • 19
    • 0019954923 scopus 로고
    • Endocytosis of lysosomal alpha-galactosidase A by cultured fibroblasts from patients with Fabry disease
    • Mayes, J.S., Cray, E.L., Dell, V.A., Scheerer, J.B., Sifers, R.N.: Endocytosis of lysosomal alpha-galactosidase A by cultured fibroblasts from patients with Fabry disease. Am J Hum Genet 34(4), 602-610 (1982)
    • (1982) Am J Hum Genet , vol.34 , Issue.4 , pp. 602-610
    • Mayes, J.S.1    Cray, E.L.2    Dell, V.A.3    Scheerer, J.B.4    Sifers, R.N.5
  • 20
    • 0029130352 scopus 로고
    • A novel amino acid modification in sulfatases that is defective in multiple sulfatase deficiency
    • Schmidt, B., Selmer, T., Ingendoh, A., von Figura, K.:A novel amino acid modification in sulfatases that is defective in multiple sulfatase deficiency. Cell 82(2), 271-278 (1995)
    • (1995) Cell , vol.82 , Issue.2 , pp. 271-278
    • Schmidt, B.1    Selmer, T.2    Ingendoh, A.3    Von Figura, K.4
  • 21
    • 0030711751 scopus 로고    scopus 로고
    • Conversion of cysteine to formylglycine: A protein modification in the endoplasmic reticulum
    • Dierks, T., Schmidt, B., von Figura, K.: Conversion of cysteine to formylglycine: a protein modification in the endoplasmic reticulum. Proc Natl Acad Sci U S A 94(22), 11963-11968 (1997)
    • (1997) Proc Natl Acad Sci U S A , vol.94 , Issue.22 , pp. 11963-11968
    • Dierks, T.1    Schmidt, B.2    Von Figura, K.3
  • 22
    • 79951811108 scopus 로고    scopus 로고
    • SUMF1 mutations affecting stability and activity of formylglycine generating enzyme predict clinical outcome in multiple sulfatase deficiency
    • doi:10.1038/ejhg.2010.219
    • Schlotawa, L., Ennemann, E.C., Radhakrishnan, K., Schmidt, B., Chakrapani, A., Christen, H.J., Moser, H., Steinmann, B., Dierks, T., Gartner, J.: SUMF1 mutations affecting stability and activity of formylglycine generating enzyme predict clinical outcome in multiple sulfatase deficiency. Eur J Hum Genet: EJHG 19(3), 253-261 (2011). doi:10.1038/ejhg.2010.219
    • (2011) Eur J Hum Genet: EJHG , vol.19 , Issue.3 , pp. 253-261
    • Schlotawa, L.1    Ennemann, E.C.2    Radhakrishnan, K.3    Schmidt, B.4    Chakrapani, A.5    Christen, H.J.6    Moser, H.7    Steinmann, B.8    Dierks, T.9    Gartner, J.10
  • 23
    • 0032512813 scopus 로고    scopus 로고
    • Conversion of cysteine to formylglycine in eukaryotic sulfatases occurs by a common mechanism in the endoplasmic reticulum
    • Dierks, T., Lecca, M.R., Schmidt, B., von Figura, K.: Conversion of cysteine to formylglycine in eukaryotic sulfatases occurs by a common mechanism in the endoplasmic reticulum. FEBS Lett 423(1), 61-65 (1998)
    • (1998) FEBS Lett , vol.423 , Issue.1 , pp. 61-65
    • Dierks, T.1    Lecca, M.R.2    Schmidt, B.3    Von Figura, K.4
  • 25
    • 34447296997 scopus 로고    scopus 로고
    • Selective clearance of glycoforms of a complex glycoprotein pharmaceutical caused by terminal N-acetylglucosamine is similar in humans and cynomolgus monkeys
    • doi:10.1093/glycob/cwm017
    • Jones, A.J., Papac, D.I., Chin, E.H., Keck, R., Baughman, S.A., Lin, Y.S., Kneer, J., Battersby, J.E.: Selective clearance of glycoforms of a complex glycoprotein pharmaceutical caused by terminal N-acetylglucosamine is similar in humans and cynomolgus monkeys. Glycobiology 17(5), 529-540 (2007). doi:10.1093/glycob/cwm017
    • (2007) Glycobiology , vol.17 , Issue.5 , pp. 529-540
    • Jones, A.J.1    Papac, D.I.2    Chin, E.H.3    Keck, R.4    Baughman, S.A.5    Lin, Y.S.6    Kneer, J.7    Battersby, J.E.8
  • 26
    • 0024382077 scopus 로고
    • Ligand interactions of the cation-independent mannose 6-phosphate receptor. The stoichiometry of mannose 6-phosphate binding
    • Tong, P.Y., Gregory, W., Kornfeld, S.: Ligand interactions of the cation-independent mannose 6-phosphate receptor. The stoichiometry of mannose 6-phosphate binding. J Biol Chem 264(14), 7962-7969 (1989)
    • (1989) J Biol Chem , vol.264 , Issue.14 , pp. 7962-7969
    • Tong, P.Y.1    Gregory, W.2    Kornfeld, S.3
  • 27
    • 84860710411 scopus 로고    scopus 로고
    • Immunogenicity to therapeutic proteins: Impact on PK/PD and efficacy
    • doi:10.1208/s12248-012-9340-y
    • Chirmule, N., Jawa, V., Meibohm, B.: Immunogenicity to therapeutic proteins: impact on PK/PD and efficacy. AAPS J 14(2), 296-302 (2012). doi:10.1208/s12248-012-9340-y
    • (2012) AAPS J , vol.14 , Issue.2 , pp. 296-302
    • Chirmule, N.1    Jawa, V.2    Meibohm, B.3


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