Enzyme Replacement with Recombinant β-Glucuronidase in Murine Mucopolysaccharidosis Type VII: Impact of Therapy during the First Six Weeks of Life on Subsequent Lysosomal Storage, Growth, and Survival

Pediatric Research

Volumn 39, Issue 6, 1996, Pages 1050-1054

  Vogler, Carole ^a,d   Sands, Mark S ^b   Levy, Beth ^a   Galvin, Nancy ^a   Birkenmeier, Edward H ^c   Sly, William S ^a  

^a SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c JACKSON LABORATORY   (United States)

^d Cardinal Glennon Hospital   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA GALACTOSIDASE; BETA GLUCURONIDASE; BETA N ACETYLHEXOSAMINIDASE;

ANIMAL CELL; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOACCUMULATION; BODY WEIGHT; BRAIN; CELL TYPE; CELL VACUOLE; CONTROLLED STUDY; ENZYME REPLACEMENT; FEMALE; INTRAVENOUS DRUG ADMINISTRATION; KIDNEY; LIVER; LONG BONE; LYSOSOME; MACROPHAGE; MALE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NEWBORN; NONHUMAN; PRIORITY JOURNAL; SPLEEN; SURVIVAL;

EID: 0029954652     PISSN: 00313998     EISSN: None     Source Type: Journal    
DOI: 10.1203/00006450-199606000-00019     Document Type: Article

References (26)

1. Birkenmeier EH, Davisson MT, Beamer WG, Ganschow RE, Vogler CA, Gwynn B, Lyford KA, Maltais LM, Wawrzyniak CJ 1989 Murine mucopolysaccharidosis type VII: Characterization of a mouse with β-glucuronidase deficiency. J Clin Invest 83:1258-1266
2. Vogler C, Birkenmeier EH, Sly WS, Levy B, Pegors C, Kyle JW, Beamer WG 1990 A murine model of mucopolysaccharidosis VII: gross and microscopic findings in β-glucuronidase-deficient mice. Am J Pathol 136:207-217
3. Sands MS, Birkenmeier EH 1993 A single-base-pair deletion in the β-glucuronidase gene accounts for the phenotype of murine mucopolysaccharidosis type VII. Proc Natl Acad Sci USA 90:6567-6571
4. Chang PL, Lambert DT, Pisa MA 1993 Behavioural abnormalities in a murine model of a human lysosomal storage disease. Neuroreport 4:507-510
5. Birkenmeier EH, Barker JE, Vogler CA, Kyle JW, Sly WS, Gwynn B, Levy B, Pegors C 1991 Increased life span and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation. Blood 78:3081-3092
6. Sands MS, Barker JE, Vogler C, Levy B, Gwynn B, Galvin N, Sly WS, Birkenmeier E 1993 Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab Invest 68:676-686
7. Sands MS, Erway LC, Vogler C, Sly WS, Birkenmeier EH 1995 Syngeneic bone marrow transplantation reduces hearing loss associated with murine mucopolysaccharidosis type VII. Blood 86:2033-2040
8. 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
9. Wolfe JH, Sands MS, Barker JE, Gwynn B, Rowe LB, Vogler CA, Birkenmeier EH 1992 Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 360:749-753
10. Barton NW, Brady RO, Dambrosia JM, DiBisceglie AM, Doppelt SH, Hill SC, Mankin HJ, Murray GJ, Parker RI, Argoff CE, Grewal RP, Yu KT, and collaborators 1991 Replacement therapy for inherited enzyme deficiency - macrophage targeted glucocerebrosidase for Gaucher's disease. N Engl J Med 324:1464-1470
11. Beutler E 1993 Modern diagnosis and treatment of Gaucher's disease. Am J Dis Child 147:1175-1183
12. Zimran A, Elstein D, Kannai R, Zevin S, Hadas-Halpern I, Levy-Lahad E, Cohen Y, Horowitz M, Abrahamov A 1994 Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype and clinical features on response to treatment. Am J Med 97:3-13
13. Grabowski GA, Barton NW, Pastores G, Dambrosia JM, Banerjee TK, McKee MA, Parker C, Schiffmann R, Hill SC, Brady RO 1995 Enzyme therpay in type I Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources. Ann Intern Med 122:33-39
14. Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, Sly WS 1993 Enzyme replacement with recombinant β-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediatr Res 34:837-840
15. Grubb JH, Kyle JW, Cody LB, Sly WS 1993 Large scale purification of phosphorylated recombinant human β-glucuronidase from overexpressing mouse L cells. FASEB J 7:A1255(abstr)
16. Wolfe JH, Sands MS 1995 Murine mucopolysaccharidosis type VII: A model system for somatic gene therapy of the central nervous system. In: Lowenstein P, Enquist L (eds) Gene Transfer into Neurones, Towards Gene Therapy of Neurological Disorders. J Wiley & Sons, Essex, England (in press)
17. Neufeld EF, Muenzer J 1995 The mucopolysaccharidosis. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic Basis of Inherited Disease, 7th Ed. McGraw-Hill, New York, pp 2465-2494
18. Risau W, Hallmann R, Albrecht U 1986 Differentiation-dependent expression of proteins in brain endothelium during development of the blood-brain barrier. Dev Biol 117:537-545
19. Stewart PA, Hayakawa EM 1987 Interendothelial junctional changes underlie the developmental 'tightening' of the blood-brain barrier. Dev Brain Res 32:271-281
20. Oohira A, Katoh-Semba, R, Watanabe E, Matsui F 1994 Brain development and multiple molecular species of proteoglycan. Neurosci Res 20:195-207
21. Galvin NJ, Levy B, Vogler C 1994 Brain morphology in murine mucopolysaccharidosis type VII. Mol Biol Cell 5:360a(abstr)
22. Natowicz M, Baenziger JU, Sly WS 1982 Structural studies of the phosphorylated high mannose-type oligosaccharides on human β-glucuronidase. J Biol Chem 257:4412-4420
23. Shipley JM, Grubb JH, Sly WS 1993 The role of glycosylation and phosphorylation in the expression of active human β-glucuronidase. J Biol Chem 268:12193-12198
24. Achord D, Brot F, Gonzalez-Noriega A, Sly WS, Stahl P 1977 Human β-glucuronidase II. Fate of infused human placental β-glucuronidase in the rat. Pediatr Res 11:816-822
25. Achord DT, Brot FE, Bell CE, Sly WS 1978 Human β-glucuronidase: in vivo clearance and in vitro uptake by a glycoprotein recognition system on reticuloendothelial cells. Cell 15:269-278
26. Stahl PD, Rodman JS, Miller MJ, Schlesinger PH 1978 Evidence for receptor-mediated binding of glycoproteins, glycoconjugates, and lysosomal glycosidases by alveolar machrophages. Proc Natl Acad Sci USA 75:1399-1403