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Journal Abstract Search

285 related items for PubMed ID: 15233947

  • 41. Phenotypic rescue after adeno-associated virus-mediated delivery of 4-sulfatase to the retinal pigment epithelium of feline mucopolysaccharidosis VI.
    Ho TT, Maguire AM, Aguirre GD, Surace EM, Anand V, Zeng Y, Salvetti A, Hopwood JJ, Haskins ME, Bennett J.
    J Gene Med; 2002; 4(6):613-21. PubMed ID: 12439853
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  • 48. Intrathecal AAVrh10 corrects biochemical and histological hallmarks of mucopolysaccharidosis VII mice and improves behavior and survival.
    Pagès G, Giménez-Llort L, García-Lareu B, Ariza L, Navarro M, Casas C, Chillón M, Bosch A.
    Hum Mol Genet; 2019 Nov 01; 28(21):3610-3624. PubMed ID: 31511867
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  • 49. Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function.
    Bush RA, Zeng Y, Colosi P, Kjellstrom S, Hiriyanna S, Vijayasarathy C, Santos M, Li J, Wu Z, Sieving PA.
    Hum Gene Ther; 2016 May 01; 27(5):376-89. PubMed ID: 27036983
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  • 50. 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 01; 16(6):746-56. PubMed ID: 19357715
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  • 51. Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.
    Woloszynek JC, Roberts M, Coleman T, Vogler C, Sly W, Semenkovich CF, Sands MS.
    Biochem J; 2004 Apr 15; 379(Pt 2):461-9. PubMed ID: 14705966
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  • 52. Gene therapy for murine mucopolysaccharidosis type VII.
    Sands MS, Wolfe JH, Birkenmeier EH, Barker JE, Vogler C, Sly WS, Okuyama T, Freeman B, Nicholes A, Muzyczka N, Chang PL, Axelrod HR.
    Neuromuscul Disord; 1997 Jul 15; 7(5):352-60. PubMed ID: 9267850
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  • 55. Syngeneic bone marrow transplantation reduces the hearing loss associated with murine mucopolysaccharidosis type VII.
    Sands MS, Erway LC, Vogler C, Sly WS, Birkenmeier EH.
    Blood; 1995 Sep 01; 86(5):2033-40. PubMed ID: 7655032
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  • 57. Gene therapeutic prospects in early onset of severe retinal dystrophy: restoration of vision in RPE65 Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium.
    Rolling F, Le Meur G, Stieger K, Smith AJ, Weber M, Deschamps JY, Nivard D, Mendes-Madeira A, Provost N, Péréon Y, Cherel Y, Ali RR, Hamel C, Moullier P, Rolling F.
    Bull Mem Acad R Med Belg; 2006 Sep 01; 161(10-12):497-508; discussion 508-9. PubMed ID: 17503728
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  • 58. Inner ear pathology in the mucopolysaccharidosis VII mouse.
    Ohlemiller KK, Hennig AK, Lett JM, Heidbreder AF, Sands MS.
    Hear Res; 2002 Jul 01; 169(1-2):69-84. PubMed ID: 12121741
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  • 59. 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 01; 93(6):2324-31. PubMed ID: 8200966
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  • 60. Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function.
    O'Connor LH, Erway LC, Vogler CA, Sly WS, Nicholes A, Grubb J, Holmberg SW, Levy B, Sands MS.
    J Clin Invest; 1998 Apr 01; 101(7):1394-400. PubMed ID: 9525982
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