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Journal Abstract Search

285 related items for PubMed ID: 15233947

  • 21. Enzyme replacement with recombinant beta-glucuronidase in murine mucopolysaccharidosis type VII: impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth, and survival.
    Vogler C, Sands MS, Levy B, Galvin N, Birkenmeier EH, Sly WS.
    Pediatr Res; 1996 Jun; 39(6):1050-4. PubMed ID: 8725268
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  • 22. Photoreceptor degeneration and altered distribution of interphotoreceptor matrix proteoglycans in the mucopolysaccharidosis VII mouse.
    Lazarus HS, Sly WS, Kyle JW, Hageman GS.
    Exp Eye Res; 1993 May; 56(5):531-41. PubMed ID: 8500564
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  • 23. Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII.
    Macsai CE, Derrick-Roberts AL, Ding X, Zarrinkalam KH, McIntyre C, Anderson PH, Anson DS, Byers S.
    Mol Genet Metab; 2012 Jun; 106(2):202-13. PubMed ID: 22525091
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  • 25. Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII.
    Sleeper MM, Fornasari B, Ellinwood NM, Weil MA, Melniczek J, O'Malley TM, Sammarco CD, Xu L, Ponder KP, Haskins ME.
    Circulation; 2004 Aug 17; 110(7):815-20. PubMed ID: 15289379
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  • 30. Vestronidase alfa: Recombinant human β-glucuronidase as an enzyme replacement therapy for MPS VII.
    Cadaoas J, Boyle G, Jungles S, Cullen S, Vellard M, Grubb JH, Jurecka A, Sly W, Kakkis E.
    Mol Genet Metab; 2020 May 17; 130(1):65-76. PubMed ID: 32192868
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  • 31. Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.
    Narfström K, Katz ML, Bragadottir R, Seeliger M, Boulanger A, Redmond TM, Caro L, Lai CM, Rakoczy PE.
    Invest Ophthalmol Vis Sci; 2003 Apr 17; 44(4):1663-72. PubMed ID: 12657607
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  • 32. Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of beta-glucuronidase.
    Taylor RM, Wolfe JH.
    Nat Med; 1997 Jul 17; 3(7):771-4. PubMed ID: 9212105
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  • 33. Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates.
    Sands MS, Barker JE, Vogler C, Levy B, Gwynn B, Galvin N, Sly WS, Birkenmeier E.
    Lab Invest; 1993 Jun 17; 68(6):676-86. PubMed ID: 8515654
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  • 34. Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.
    Vogler C, Levy B, Galvin NJ, Thorpe C, Sands MS, Barker JE, Baty J, Birkenmeier EH, Sly WS.
    Pediatr Res; 1999 Jun 17; 45(6):838-44. PubMed ID: 10367775
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  • 35. Adeno-associated virus type 5 reduces learning deficits and restores glutamate receptor subunit levels in MPS VII mice CNS.
    Liu G, Chen YH, He X, Martins I, Heth JA, Chiorini JA, Davidson BL.
    Mol Ther; 2007 Feb 17; 15(2):242-7. PubMed ID: 17235300
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  • 36. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease.
    Daly TM, Vogler C, Levy B, Haskins ME, Sands MS.
    Proc Natl Acad Sci U S A; 1999 Mar 02; 96(5):2296-300. PubMed ID: 10051635
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  • 37. Delivery of a retroviral vector expressing human beta-glucuronidase to the liver and spleen decreases lysosomal storage in mucopolysaccharidosis VII mice.
    Gao C, Sands MS, Haskins ME, Ponder KP.
    Mol Ther; 2000 Sep 02; 2(3):233-44. PubMed ID: 10985954
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  • 38. Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.
    Skorupa AF, Fisher KJ, Wilson JM, Parente MK, Wolfe JH.
    Exp Neurol; 1999 Nov 02; 160(1):17-27. PubMed ID: 10630187
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  • 39. Glycosaminoglycan storage in cultured neonatal murine mucopolysaccharidosis type VII neuroglial cells and correction by beta-glucuronidase gene transfer.
    Taylor RM, Wolfe JH.
    J Neurochem; 1997 May 02; 68(5):2079-85. PubMed ID: 9109535
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  • 40. Treatment of a lysosomal storage disease, mucopolysaccharidosis VII, with microencapsulated recombinant cells.
    Ross CJ, Bastedo L, Maier SA, Sands MS, Chang PL.
    Hum Gene Ther; 2000 Oct 10; 11(15):2117-27. PubMed ID: 11044913
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