Enzyme replacement therapy in a feline model of MPS VI: Modification of enzyme structure and dose frequency

Pediatric Research

Volumn 47, Issue 6, 2000, Pages 743-749

  Byers, Sharon ^a   Crawley, Allison C ^a   Brumfield, Leanne K ^a   Nuttall, Jacqueline D ^a   Hopwood, John J ^a  

^a WOMEN S AND CHILDREN S HOSPITAL   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

1 (3 DIMETHYLAMINOPROPYL) 3 ETHYLCARBODIIMIDE; ETHYLENEDIAMINE; MANNOSE 6 PHOSPHATE; N ACETYLGALACTOSAMINE 4 SULFATASE; POLYLYSINE; RECOMBINANT ENZYME; SUCCINIMIDE DERIVATIVE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ARTICULAR CARTILAGE; CARTILAGE CELL; CAT; CELL VACUOLE; CONTROLLED STUDY; DOSE RESPONSE; DRUG DIFFUSION; DRUG DISTRIBUTION; DRUG HALF LIFE; DRUG UPTAKE; ENZYME MODIFICATION; ENZYME REPLACEMENT; ENZYME STRUCTURE; LYSOSOME; MAROTEAUX LAMY SYNDROME; NONHUMAN; PARTITION COEFFICIENT; PRIORITY JOURNAL;

EID: 0034121656     PISSN: 00313998     EISSN: None     Source Type: Journal    
DOI: 10.1203/00006450-200006000-00010     Document Type: Article

References (59)

1. Hopwood JJ 1991 Lysosomal function and disease. Todays Life Sci 34:24-33
2. Neufeld EF, Muenzer J 1995. The mucopolysaccharidoses. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic and Molecular Basis of Inherited Disease. McGraw-Hill, New York, pp 2465-2494
3. Byers S, Rozaklis T, Brumfield LK, Ranieri E, Hopwood JJ 1998 Glycosaminoglycan accumulation and excretion in the mucopolysaccharidoses: characterization and basis of a diagnostic test for MPS. Mol Genet Metab 65:282-290
4. Barton NW, Brady RO, Dambrosia JM, Di Bisceglie AM, Doppelt SH, Hill SC, Mankin HJ, Murray GJ, Parker RI, Argoff CE, Grewal RP, Yu KT 1991 Replacement therapy for inherited enzyme deficiency: macrophage-targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 324:1464-1470
5. Hershfield MS, Buckley RH, Greenberg ML, Melton AL, Schiff R, Hatem C, Kurtzberg J, Markert ML, Kobayashi RH, Kobayashi AL, Abuchowski A 1987 Treatment of adenosine deaminase deficiency with polyethylene glycol-modified adenosine deaminase. N Engl J Med 316:589-596
6. Levy Y, Hershfield MS, Fernandez-Mejia C, Polmar SH, Scudiery D, Berger M, Sorenson RU 1988 Adenosine deaminase deficiency with late onset of recurrent infections: response to treatment with polyethylene glycol-modified adenosine deaminase. J Pediatr 113:312-317
7. r. values. Biochem J 248:755-764
8. Freeman C, Hopwood JJ 1989 Sanfilippo D syndrome: estimation of N-acetylglucosamine-6-sulphatase activity with a radiolabelled monosulfated disaccharide substrate. Anal Biochem 176:244-248
9. Freeman C, Hopwood JJ 1992 Human α-L-iduronidase: catalytic properties and an integrated role in the lysosomal degradation of heparan sulphate. Biochem J 282:899-908
10. Bielicki J, Freeman C, Clements PR, Hopwood JJ 1990 Human liver iduronate-2-sulphatase: purification, characterization and catalytic properties. Biochem J 271:75-86
11. Bielicki J, Hopwood JJ 1991 Human liver N-acetylgalactosamine-6-sulphatase: purification, characterization and catalytic properties. Biochem J 279:515-520
12. Mahuran D, Clements P, Hopwood JJ 1983 A rapid four column purification of 2-deoxy-D-glucoside-2-sulphamate sulphohydrolase from human liver. Biochim Biophys Acta 757:359-365
13. Weber B, Blanch L, Clements PR, Scott HS, Hopwood JJ 1996 Cloning and expression of the gene involved in Sanfilippo B syndrome (mucopolysaccharidosis type IIIB). Hum Mol Genet 5:771-777
14. Sasaki T, Sukegawa K, Masue M, Fukuda S, Tomatsu S, Orii T 1991 Purification and partial characterization of α-N-acetylglucosaminidase from human liver. J Biochem 110:842-846
15. Meikle PJ, Whittle AM, Hopwood JJ 1995 Human acetyl-coenzyme A:α-glucosaminide N-acetyltransferase. Kinetic characterization and mechanistic interpretation. Biochem J 308:327-333
16. M1 ganglioside β-galactosidase A: Purification and studies of the enzyme from human liver. J Biol Chem 249:7969-7976
17. Musa BU, Doe RP, Seal US 1965 Purification and properties of human liver β-glucuronidase. J Biol Chem 240:2811-2816
18. Wilson PJ, Morris CP, Anson DS, Occhiodoro T, Bielicki J, Clements PR, Hopwood JJ 1990 Hunter syndrome: isolation of an iduronate-2-sulphatase cDNA clone and analysis of patient DNA. Proc Natl Acad Sci USA 87:8531-8535
19. Scott HS, Anson DS, Orsborn AM, Nelson PV, Clements PR, Morris CP, Hopwood JJ 1991 Human α-:-iduronidase: cDNA isolation, expression and alternatively spliced transcripts. Proc Natl Acad Sci USA 88:9695-9699
20. Schuchman EH, Jackson CE, Desnick RJ 1990 Human arylsulfatase B: MOPAC cloning, nucleotide sequence of a full-length cDNA and regions of aminoacid identity with arylsulfatases A and C. Genomics 6:149-158
21. Peters C, Schmidt B, Rommerskirch W, Rupp K, Zuhlsdorf M, Vingron M, Meyer HE, Pohlmann R, von Figura K 1990 Phylogenetic conservation of arylsulfatases: cDNA cloning and expression of human arylsulfatase B. J Biol Chem 265:3374-3381
22. Scott HS, Blanch L, Guo X-H, Freeman C, Orsborn A, Baker E, Sutherland GR, Morris CP, Hopwood JJ 1995 Cloning of the sulphamidase gene and identification of mutations in Sanfilippo A syndrome. Nat Genet 11:465-467
23. Robertson DA, Freeman C, Morris CP, Hopwood JJ 1992 A cDNA clone for human glucosamine-6-sulphatase reveals differences between arylsulphatases and nonarylsulphatases. Biochem J 288:539-544
24. Tomatsu S, Fukuda S, Masue M, Sukegawa K, Fukao T, Yamagishi A, Hori T, Iwata H, Ogawa T, Nakashima Y, Hanyu Y, Hashimoto T, Titani K, Oyama R, Suzuki M, Yagi K, Hayashi Y, Orii T 1991 Morquio disease: isolation, characterization and expression of full length cDNA for human N-acetylgalactosamine-6-sulphate sulphatase. Biochem Biophys Res Commun 181:677-683
25. Oshima A, Kyle JW, Miller RD, Hoffman JW, Powell PP, Grubb JH, Sly WS, Tropak M, Guise KS, Gravel RA 1987 Cloning, sequencing and expression of cDNA for human β-glucuronidase. Proc Natl Acad Sci USA 84:685-689
26. Oshima A, Tsuji A, Nagao Y, Sakuraba H, Suzuki Y 1988 Cloning, sequencing and expression of cDNA for human β-galactosidase. Biochem Biophys Res Commun 157:238-244
27. Anson DS, Taylor JA, Bielicki J, Harper GS, Peters C, Gibson GJ, Hopwood JJ 1992 Correction of human mucopolysaccharidosis type-VI fibroblasts with recombinant N-acetylgalactosamine-4-sulphatase. Biochem J 284:789-794
28. Bielicki J, Hopwood JJ, Wilson PJ, Anson DS 1993 Recombinant human iduronate-2-sulphatase: correction of mucopolysaccharidosis-type II fibroblasts and characterization of the purified enzyme. Biochem J 289:241-246
29. Kakkis ED, Matynia A, Jonas AJ, Neufeld EF 1994 Overexpression of the human lysosomal enzyme α-L-iduronidase in chinese hamster ovary cells. Protein Expr Purif 5:225-232
30. Unger EG, Durrant J, Anson DS, Hopwood JJ 1994 Recombinant α-L-iduronidase: characterization of the purified enzyme and correction of mucopolysaccharidosis type I fibroblasts. Biochem J 303:43-49
31. Bielicki J, Hopwood JJ, Melville EL, Anson DS 1998 Recombinant human sulphamidase: expression, amplification, purification and characterization. Biochem J 329:145-150
32. Litjens T, Bielicki J, Anson DS, Friderici K, Jones MZ, Hopwood JJ 1997 Expression, purification and characterization of recombinant caprine N-acetylglucosamine-6-sulphatase. Biochem J 327:89-94
33. Bielicki J, Fuller M, Guo X-H, Morris CP, Hopwood JJ, Anson DS 1995 Expression, purification and characterization of recombinant human N-acetylgalactosamine-6-sulphatase. Biochem J 311:333-339
34. Gehrmann MC, Opper M, Sedlacek HH, Bosslet K, Czech J 1994 Biochemical properties of recombinant human β-glucuronidase synthesized in baby hamster kidney cells. Biochem J 301:821-828
35. Shull RM, Kakkis ED, McEntee MF, Kania SA, Jonas AJ, Neufield E 1995 Enzyme replacement therapy in a canine model of Hurler syndrome. Proc Natl Acad Sci USA 91:12937-12941
36. Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, Birkenmeier EH 1994 Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest 93:2324-2331
37. Crawley AC, Brooks DA, Muller VJ, Petersen BA, Isaac EL, Bielicki J, King BM, Boulter CD, Moore AJ, Fazzalari NL, Anson DS, Byers S, Hopwood JJ 1996 Enzyme replacement therapy in a feline model of Maroteaux-Lamy syndrome. J Clin Invest 97:1864-1873
38. Crawley AC, Niedzielski KH, Isaac EL, Davey RCA, Byers S, Hopwood JJ 1997 Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI. J Clin Invest 99:651-662
39. Byers S, Nuttall JD, Crawley AC, Hopwood JJ, Smith K, Fazzalari NL 1997 Effect of enzyme replacement therapy on bone formation in a feline model of mucopolysaccharidosis type VI. Bone 21:425-431
40. Hopwood JJ, Muller V, Harrison JR, Carey WF, Elliott H, Robertson EF, Pollard AC 1982 Enzymatic diagnosis of the mucopolysaccharidoses: experience of 96 cases diagnosed in a 5-year period. Med J Aust 1:257-260
41. Bradford MM 1976 A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal Biochem 72:248-254
42. Hoare DG, Koshland DE 1967 A method for the quantitative modification and estimation of carboxylic acid groups in proteins. J Biol Chem 242:2447-2453
43. Griffen DE, Giffels J 1982 Study of protein characteristics that influence entry into the cerebrospinal fluid of normal mice and mice with encephalitis. J Clin Invest 70:289-295
44. Staros JV, Wright RW, Swingle DM 1986 Enhancement by N-hydroxysulfosuccinimide of water-soluble carbodiimide-mediated coupling reactions. Anal Biochem 156:220-222
45. Maroudas A 1976 Transport of solutes through cartilage: permeability to large molecules. J Anat 122:335-347
46. Kirk RW, Bistner SI 1985 Handbook of Veterinary Procedures and Emergency Treatment. WB Saunders, Philadelphia, p 592
47. Parfitt AM, Drezner MK, Glorieux FH, Kanis JA, Malluche H, Meunier PJ, Ott SM, Recker RR 1987 Bone histomorphometry: standardization of nomenclature, symbols and units. J Bone Miner Res 2:595-610
48. 3H-[N-acetylgalactosamine-4-sulphatase]: plasma clearance, tissue distribution and cellular uptake in the rat. J Mol Neurosci 11:223-232
49. Fillat C, Simonaro CM, Yeyati PL, Abkowitz JL, Haskins ME, Schuchman EH 1996 Arylsulfatase B activities and glycosaminoglycan levels in retrovirally transduced mucopolysaccharidosis type VI cells. Prospects for gene therapy. J Clin Invest 98:497-502
50. Dunstone JR 1960 Ion-exchange reactions between cartilage and various cations. Biochem J 77:164-170
51. Comper WD, Laurent TC 1978 Physiological function of connective tissue polysaccharides. Physiol Rev 58:255-315
52. Triguero D, Buciak JD, Yang J, Pardridge WM 1989 Blood-brain barrier transport of cationized immunoglobulin G: Enhanced delivery compared to native protein. Proc Natl Acad Sci USA 86:4761-4765
53. Pardridge WM, Triguero D, Buciak J, Yang J 1990 Evaluation of cationized rat albumin as a potential blood-brain barrier drug transport vector. J Pharmacol Exp Ther 255:893-899
54. Shimon-Hophy M, Wadhwani KC, Chandrasekaran K, Larson D, Smith QR, Rapoport SI 1991 Regional blood-brain barrier transport of cationized bovine serum albumin in awake rats. Am J Physiol 30:R478-R483
55. dosSantos WLC, Rahman J, Klein N, Male DK 1995 Distribution and analysis of surface charge on brain endothelium in vitro and in situ. Acta Neuropathol 90:305-311
56. Triguero D, Biciak J, Pardridge W 1990 Capillary depletion method for quantification of blood-brain barrier transport of circulating peptides and plasma proteins. J Neurochem 54:1882-1885
57. Poduslo JF, Curran GL 1996 Polyamine modification increases the permeability of proteins at the blood-nerve and blood-brain barriers. J Neurochem 66:1599-1609
58. Brooks DA, King BM, Crawley AC, Byers S, Hopwood JJ 1997 Enzyme replacement therapy in mucopolysaccharidosis type VI: evidence for immune responses and altered efficacy of treatment in animal models. Biochim Biophys Acta 1363:203-216
59. Kushner I, Somerville JA 1971 Permeability of human synovial membrane to plasma proteins. Relationship to molecular size and inflammation. Arthritis Rheum 14:560-570