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Journal Abstract Search

196 related items for PubMed ID: 12624185

  • 1. Long-term correction of globotriaosylceramide storage in Fabry mice by recombinant adeno-associated virus-mediated gene transfer.
    Park J, Murray GJ, Limaye A, Quirk JM, Gelderman MP, Brady RO, Qasba P.
    Proc Natl Acad Sci U S A; 2003 Mar 18; 100(6):3450-4. PubMed ID: 12624185
    [Abstract] [Full Text] [Related]

  • 2. Characterization of Fabry mice treated with recombinant adeno-associated virus 2/8-mediated gene transfer.
    Choi JO, Lee MH, Park HY, Jung SC.
    J Biomed Sci; 2010 Apr 16; 17(1):26. PubMed ID: 20398385
    [Abstract] [Full Text] [Related]

  • 3. Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period.
    Ogawa K, Hirai Y, Ishizaki M, Takahashi H, Hanawa H, Fukunaga Y, Shimada T.
    Mol Genet Metab; 2009 Mar 16; 96(3):91-6. PubMed ID: 19091614
    [Abstract] [Full Text] [Related]

  • 4. Long-term systemic therapy of Fabry disease in a knockout mouse by adeno-associated virus-mediated muscle-directed gene transfer.
    Takahashi H, Hirai Y, Migita M, Seino Y, Fukuda Y, Sakuraba H, Kase R, Kobayashi T, Hashimoto Y, Shimada T.
    Proc Natl Acad Sci U S A; 2002 Oct 15; 99(21):13777-82. PubMed ID: 12370426
    [Abstract] [Full Text] [Related]

  • 5. 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
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  • 7. 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]

  • 8. Systemic mRNA Therapy for the Treatment of Fabry Disease: Preclinical Studies in Wild-Type Mice, Fabry Mouse Model, and Wild-Type Non-human Primates.
    Zhu X, Yin L, Theisen M, Zhuo J, Siddiqui S, Levy B, Presnyak V, Frassetto A, Milton J, Salerno T, Benenato KE, Milano J, Lynn A, Sabnis S, Burke K, Besin G, Lukacs CM, Guey LT, Finn PF, Martini PGV.
    Am J Hum Genet; 2019 Apr 04; 104(4):625-637. PubMed ID: 30879639
    [Abstract] [Full Text] [Related]

  • 9. Naked plasmid DNA-based alpha-galactosidase A gene transfer partially reduces systemic accumulation of globotriaosylceramide in Fabry mice.
    Nakamura G, Maruyama H, Ishii S, Shimotori M, Kameda S, Kono T, Miyazaki J, Kulkarni AB, Gejyo F.
    Mol Biotechnol; 2008 Feb 04; 38(2):109-19. PubMed ID: 18219591
    [Abstract] [Full Text] [Related]

  • 10. Therapeutic strategy for Fabry disease by intravenous administration of adeno-associated virus 2 or 9 in α-galactosidase A-deficient mice.
    Hayashi Y, Sehara Y, Watano R, Ohba K, Takayanagi Y, Muramatsu K, Sakiyama Y, Mizukami H.
    J Gene Med; 2023 Dec 04; 25(12):e3560. PubMed ID: 37392007
    [Abstract] [Full Text] [Related]

  • 11. Lentivector transduction improves outcomes over transplantation of human HSCs alone in NOD/SCID/Fabry mice.
    Pacienza N, Yoshimitsu M, Mizue N, Au BC, Wang JC, Fan X, Takenaka T, Medin JA.
    Mol Ther; 2012 Jul 04; 20(7):1454-61. PubMed ID: 22472949
    [Abstract] [Full Text] [Related]

  • 12. Transgenic mouse expressing human mutant alpha-galactosidase A in an endogenous enzyme deficient background: a biochemical animal model for studying active-site specific chaperone therapy for Fabry disease.
    Ishii S, Yoshioka H, Mannen K, Kulkarni AB, Fan JQ.
    Biochim Biophys Acta; 2004 Nov 05; 1690(3):250-7. PubMed ID: 15511632
    [Abstract] [Full Text] [Related]

  • 13. Therapeutic Strategy for Fabry Disease by Intravenous Administration of Adeno-Associated Virus 9 in a Symptomatic Mouse Model.
    Hayashi Y, Sehara Y, Watano R, Ohba K, Takayanagi Y, Sakiyama Y, Muramatsu K, Mizukami H.
    Hum Gene Ther; 2024 Mar 05; 35(5-6):192-201. PubMed ID: 38386497
    [Abstract] [Full Text] [Related]

  • 14. ZFN-mediated in vivo gene editing in hepatocytes leads to supraphysiologic α-Gal A activity and effective substrate reduction in Fabry mice.
    Pagant S, Huston MW, Moreira L, Gan L, St Martin S, Sproul S, Holmes MC, Meyer K, Wechsler T, Desnick RJ, Yasuda M.
    Mol Ther; 2021 Nov 03; 29(11):3230-3242. PubMed ID: 33775910
    [Abstract] [Full Text] [Related]

  • 15. Long-term enzyme correction and lipid reduction in multiple organs of primary and secondary transplanted Fabry mice receiving transduced bone marrow cells.
    Takenaka T, Murray GJ, Qin G, Quirk JM, Ohshima T, Qasba P, Clark K, Kulkarni AB, Brady RO, Medin JA.
    Proc Natl Acad Sci U S A; 2000 Jun 20; 97(13):7515-20. PubMed ID: 10840053
    [Abstract] [Full Text] [Related]

  • 16. Cardiac manifestations of Fabry disease in G3Stg/GlaKO and GlaKO mouse models-Translation to Fabry disease patients.
    Kugadas A, Artoni P, Ruangsiriluk W, Zhao M, Boukharov N, Islam R, Volfson D, Derakhchan K.
    PLoS One; 2024 Jun 20; 19(5):e0304415. PubMed ID: 38820517
    [Abstract] [Full Text] [Related]

  • 17. Restoration of peripheral neuropathy in Fabry mice via intrathecal administration of an adeno-associated virus vector encoding mGLA cDNA.
    Higuchi T, Shimada Y, Takahashi Y, Kato F, Ohashi T, Kobayashi H.
    Mol Genet Metab; 2024 Jun 20; 143(1-2):108545. PubMed ID: 39068683
    [Abstract] [Full Text] [Related]

  • 18. Genetics and Gene Therapy of Anderson-Fabry Disease.
    Simonetta I, Tuttolomondo A, Di Chiara T, Miceli S, Vogiatzis D, Corpora F, Pinto A.
    Curr Gene Ther; 2018 Jun 20; 18(2):96-106. PubMed ID: 29618309
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  • 20. Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease.
    Lee CJ, Fan X, Guo X, Medin JA.
    J Cardiol; 2011 Jan 20; 57(1):115-22. PubMed ID: 20846825
    [Abstract] [Full Text] [Related]

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