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Journal Abstract Search

239 related items for PubMed ID: 32192868

  • 21. First Report of a Patient with MPS Type VII, Due to Novel Mutations in GUSB, Who Underwent Enzyme Replacement and Then Hematopoietic Stem Cell Transplantation.
    Dubot P, Sabourdy F, Plat G, Jubert C, Cancès C, Broué P, Touati G, Levade T.
    Int J Mol Sci; 2019 Oct 28; 20(21):. PubMed ID: 31661765
    [Abstract] [Full Text] [Related]

  • 22. Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors.
    Vogler C, Galvin N, Levy B, Grubb J, Jiang J, Zhou XY, Sly WS.
    Proc Natl Acad Sci U S A; 2003 Mar 04; 100(5):2669-73. PubMed ID: 12591953
    [Abstract] [Full Text] [Related]

  • 23. Enzyme replacement therapy for murine mucopolysaccharidosis type VII.
    Sands MS, Vogler C, Kyle JW, Grubb JH, Levy B, Galvin N, Sly WS, Birkenmeier EH.
    J Clin Invest; 1994 Jun 04; 93(6):2324-31. PubMed ID: 8200966
    [Abstract] [Full Text] [Related]

  • 24. Enzyme replacement therapy improves reproductive performance in mucopolysaccharidosis type VII mice but does not prevent postnatal losses.
    Soper BW, Pung AW, Vogler CA, Grubb JH, Sly WS, Barker JE.
    Pediatr Res; 1999 Feb 04; 45(2):180-6. PubMed ID: 10022587
    [Abstract] [Full Text] [Related]

  • 25. AAV-mediated intravitreal gene therapy reduces lysosomal storage in the retinal pigmented epithelium and improves retinal function in adult MPS VII mice.
    Hennig AK, Ogilvie JM, Ohlemiller KK, Timmers AM, Hauswirth WW, Sands MS.
    Mol Ther; 2004 Jul 04; 10(1):106-16. PubMed ID: 15233947
    [Abstract] [Full Text] [Related]

  • 26. Widespread correction of lysosomal storage following intrahepatic injection of a recombinant adeno-associated virus in the adult MPS VII mouse.
    Sferra TJ, Backstrom K, Wang C, Rennard R, Miller M, Hu Y.
    Mol Ther; 2004 Sep 04; 10(3):478-91. PubMed ID: 15336648
    [Abstract] [Full Text] [Related]

  • 27. Active site mutant transgene confers tolerance to human beta-glucuronidase without affecting the phenotype of MPS VII mice.
    Sly WS, Vogler C, Grubb JH, Zhou M, Jiang J, Zhou XY, Tomatsu S, Bi Y, Snella EM.
    Proc Natl Acad Sci U S A; 2001 Feb 27; 98(5):2205-10. PubMed ID: 11226217
    [Abstract] [Full Text] [Related]

  • 28. Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery.
    Richard M, Arfi A, Seguin J, Gandolphe C, Scherman D.
    Gene Ther; 2009 Jun 27; 16(6):746-56. PubMed ID: 19357715
    [Abstract] [Full Text] [Related]

  • 29. 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
    [Abstract] [Full Text] [Related]

  • 30. Mucopolysaccharidosis type VII (Sly syndrome) - What do we know?
    Grant CL, López-Valdez J, Marsden D, Ezgü F.
    Mol Genet Metab; 2024 Mar 17; 141(3):108145. PubMed ID: 38301529
    [Abstract] [Full Text] [Related]

  • 31. Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse.
    Sferra TJ, Qu G, McNeely D, Rennard R, Clark KR, Lo WD, Johnson PR.
    Hum Gene Ther; 2000 Mar 01; 11(4):507-19. PubMed ID: 10724030
    [Abstract] [Full Text] [Related]

  • 32. Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model.
    Rowan DJ, Tomatsu S, Grubb JH, Haupt B, Montaño AM, Oikawa H, Sosa AC, Chen A, Sly WS.
    Mol Genet Metab; 2012 Sep 01; 107(1-2):161-72. PubMed ID: 22902520
    [Abstract] [Full Text] [Related]

  • 33. Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts.
    Moullier P, Bohl D, Heard JM, Danos O.
    Nat Genet; 1993 Jun 01; 4(2):154-9. PubMed ID: 8348154
    [Abstract] [Full Text] [Related]

  • 34. Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse.
    Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, Sly WS.
    Pediatr Res; 1993 Dec 01; 34(6):837-40. PubMed ID: 8108204
    [Abstract] [Full Text] [Related]

  • 35. Corrective GUSB transfer to the canine mucopolysaccharidosis VII cornea using a helper-dependent canine adenovirus vector.
    Serratrice N, Cubizolle A, Ibanes S, Mestre-Francés N, Bayo-Puxan N, Creyssels S, Gennetier A, Bernex F, Verdier JM, Haskins ME, Couderc G, Malecaze F, Kalatzis V, Kremer EJ.
    J Control Release; 2014 May 10; 181():22-31. PubMed ID: 24607662
    [Abstract] [Full Text] [Related]

  • 36. Dose-dependent effects of enzyme replacement therapy on skeletal disease progression in mucopolysaccharidosis VII dogs.
    Gawri R, Lau YK, Lin G, Shetye SS, Zhang C, Jiang Z, Abdoun K, Scanzello CR, Jo SY, Mai W, Dodge GR, Casal ML, Smith LJ.
    Mol Ther Methods Clin Dev; 2023 Mar 09; 28():12-26. PubMed ID: 36570425
    [Abstract] [Full Text] [Related]

  • 37. Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice.
    LeBowitz JH, Grubb JH, Maga JA, Schmiel DH, Vogler C, Sly WS.
    Proc Natl Acad Sci U S A; 2004 Mar 02; 101(9):3083-8. PubMed ID: 14976248
    [Abstract] [Full Text] [Related]

  • 38. Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus.
    Watson GL, Sayles JN, Chen C, Elliger SS, Elliger CA, Raju NR, Kurtzman GJ, Podsakoff GM.
    Gene Ther; 1998 Dec 02; 5(12):1642-9. PubMed ID: 10023443
    [Abstract] [Full Text] [Related]

  • 39. 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 02; 3(7):771-4. PubMed ID: 9212105
    [Abstract] [Full Text] [Related]

  • 40. Individual heat map assessments demonstrate vestronidase alfa treatment response in a highly heterogeneous mucopolysaccharidosis VII study population.
    Haller C, Song W, Cimms T, Chen CY, Whitley CB, Wang RY, Bauer M, Harmatz P.
    JIMD Rep; 2019 Sep 02; 49(1):53-62. PubMed ID: 31497482
    [Abstract] [Full Text] [Related]

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