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536 related items for PubMed ID: 16528760

  • 1. AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.
    McEachern KA, Nietupski JB, Chuang WL, Armentano D, Johnson J, Hutto E, Grabowski GA, Cheng SH, Marshall J.
    J Gene Med; 2006 Jun; 8(6):719-29. PubMed ID: 16528760
    [Abstract] [Full Text] [Related]

  • 2. AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann-Pick disease.
    Barbon CM, Ziegler RJ, Li C, Armentano D, Cherry M, Desnick RJ, Schuchman EH, Cheng SH.
    Mol Ther; 2005 Sep; 12(3):431-40. PubMed ID: 16099409
    [Abstract] [Full Text] [Related]

  • 3. Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: implications for gene therapy of Gaucher disease.
    Kim EY, Hong YB, Lai Z, Kim HJ, Cho YH, Brady RO, Jung SC.
    Biochem Biophys Res Commun; 2004 May 28; 318(2):381-90. PubMed ID: 15120612
    [Abstract] [Full Text] [Related]

  • 4. Intracerebroventricular delivery of glucocerebrosidase reduces substrates and increases lifespan in a mouse model of neuronopathic Gaucher disease.
    Cabrera-Salazar MA, Bercury SD, Ziegler RJ, Marshall J, Hodges BL, Chuang WL, Pacheco J, Li L, Cheng SH, Scheule RK.
    Exp Neurol; 2010 Oct 28; 225(2):436-44. PubMed ID: 20673762
    [Abstract] [Full Text] [Related]

  • 5. Systemic inflammation in glucocerebrosidase-deficient mice with minimal glucosylceramide storage.
    Mizukami H, Mi Y, Wada R, Kono M, Yamashita T, Liu Y, Werth N, Sandhoff R, Sandhoff K, Proia RL.
    J Clin Invest; 2002 May 28; 109(9):1215-21. PubMed ID: 11994410
    [Abstract] [Full Text] [Related]

  • 6. Murine MHC class II locus control region drives expression of human beta-glucocerebrosidase in antigen presenting cells of transgenic mice.
    Guy J, Willemsen R, Langeveld A, Grosveld F, Drabek D.
    Gene Ther; 1999 Apr 28; 6(4):498-507. PubMed ID: 10476209
    [Abstract] [Full Text] [Related]

  • 7. Generation of a conditional knockout of murine glucocerebrosidase: utility for the study of Gaucher disease.
    Sinclair GB, Jevon G, Colobong KE, Randall DR, Choy FY, Clarke LA.
    Mol Genet Metab; 2007 Feb 28; 90(2):148-56. PubMed ID: 17079175
    [Abstract] [Full Text] [Related]

  • 8. [Gene therapy of Gaucher's and Fabry's diseases: current status and prospects].
    Fabrega S, Lehn P.
    J Soc Biol; 2002 Feb 28; 196(2):175-81. PubMed ID: 12360746
    [Abstract] [Full Text] [Related]

  • 9. Long-term expression of the human glucocerebrosidase gene in vivo after transplantation of bone-marrow-derived cells transformed with a lentivirus vector.
    Kim EY, Hong YB, Lai Z, Cho YH, Brady RO, Jung SC.
    J Gene Med; 2005 Jul 28; 7(7):878-87. PubMed ID: 15712335
    [Abstract] [Full Text] [Related]

  • 10. Effective cell and gene therapy in a murine model of Gaucher disease.
    Enquist IB, Nilsson E, Ooka A, Månsson JE, Olsson K, Ehinger M, Brady RO, Richter J, Karlsson S.
    Proc Natl Acad Sci U S A; 2006 Sep 12; 103(37):13819-24. PubMed ID: 16954197
    [Abstract] [Full Text] [Related]

  • 11. [Molecular diagnosis and gene therapy for Gaucher disease].
    Ohashi T.
    Nihon Rinsho; 1993 Sep 12; 51(9):2300-7. PubMed ID: 8411706
    [Abstract] [Full Text] [Related]

  • 12. Adeno-associated viral vector-mediated gene transfer results in long-term enzymatic and functional correction in multiple organs of Fabry mice.
    Jung SC, Han IP, Limaye A, Xu R, Gelderman MP, Zerfas P, Tirumalai K, Murray GJ, During MJ, Brady RO, Qasba P.
    Proc Natl Acad Sci U S A; 2001 Feb 27; 98(5):2676-81. PubMed ID: 11226298
    [Abstract] [Full Text] [Related]

  • 13. Expression of the human glucocerebrosidase and arylsulfatase A genes in murine and patient primary fibroblasts transduced by an adeno-associated virus vector.
    Wei JF, Wei FS, Samulski RJ, Barranger JA.
    Gene Ther; 1994 Jul 27; 1(4):261-8. PubMed ID: 7584090
    [Abstract] [Full Text] [Related]

  • 14. Retrovirus-mediated transfer of the human glucocerebrosidase gene to Gaucher fibroblasts.
    Choudary PV, Barranger JA, Tsuji S, Mayor J, LaMarca ME, Cepko CL, Mulligan RC, Ginns EI.
    Mol Biol Med; 1986 Jun 27; 3(3):293-9. PubMed ID: 3736391
    [Abstract] [Full Text] [Related]

  • 15. Improved management of lysosomal glucosylceramide levels in a mouse model of type 1 Gaucher disease using enzyme and substrate reduction therapy.
    Marshall J, McEachern KA, Chuang WL, Hutto E, Siegel CS, Shayman JA, Grabowski GA, Scheule RK, Copeland DP, Cheng SH.
    J Inherit Metab Dis; 2010 Jun 27; 33(3):281-9. PubMed ID: 20336375
    [Abstract] [Full Text] [Related]

  • 16. Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
    Przybylska M, Wu IH, Zhao H, Ziegler RJ, Tousignant JD, Desnick RJ, Scheule RK, Cheng SH, Yew NS.
    J Gene Med; 2004 Jan 27; 6(1):85-92. PubMed ID: 14716680
    [Abstract] [Full Text] [Related]

  • 17. [Enzyme replacement therapy in Gaucher disease: monitoring visceral and bone changes with MRI].
    Tóth J, Szücs FZ, Benkö K, Maródi L.
    Orv Hetil; 2003 Apr 20; 144(16):749-55. PubMed ID: 12778625
    [Abstract] [Full Text] [Related]

  • 18. Chemical chaperones and permissive temperatures alter localization of Gaucher disease associated glucocerebrosidase variants.
    Sawkar AR, Schmitz M, Zimmer KP, Reczek D, Edmunds T, Balch WE, Kelly JW.
    ACS Chem Biol; 2006 May 23; 1(4):235-51. PubMed ID: 17163678
    [Abstract] [Full Text] [Related]

  • 19. Animal model of Gaucher's disease from targeted disruption of the mouse glucocerebrosidase gene.
    Tybulewicz VL, Tremblay ML, LaMarca ME, Willemsen R, Stubblefield BK, Winfield S, Zablocka B, Sidransky E, Martin BM, Huang SP.
    Nature; 1992 Jun 04; 357(6377):407-10. PubMed ID: 1594045
    [Abstract] [Full Text] [Related]

  • 20. Measurement of lysosomal glucocerebrosidase activity in mouse liver using a fluorescence-activated cell sorter assay.
    Chan KW, Waire J, Simons B, Karey K, Fung J, Copeland D, Andrews L.
    Anal Biochem; 2004 Nov 15; 334(2):227-33. PubMed ID: 15494128
    [Abstract] [Full Text] [Related]

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