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Journal Abstract Search

204 related items for PubMed ID: 20454522

  • 1. Alpha-galactosidase A-Tat fusion enhances storage reduction in hearts and kidneys of Fabry mice.
    Higuchi K, Yoshimitsu M, Fan X, Guo X, Rasaiah VI, Yen J, Tei C, Takenaka T, Medin JA.
    Mol Med; 2010; 16(5-6):216-21. PubMed ID: 20454522
    [Abstract] [Full Text] [Related]

  • 2. 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; 20(7):1454-61. PubMed ID: 22472949
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  • 3. 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
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  • 4. Correction of cardiac abnormalities in fabry mice by direct intraventricular injection of a recombinant lentiviral vector that engineers expression of alpha-galactosidase A.
    Yoshimitsu M, Higuchi K, Dawood F, Rasaiah VI, Ayach B, Chen M, Liu P, Medin JA.
    Circ J; 2006 Nov 16; 70(11):1503-8. PubMed ID: 17062978
    [Abstract] [Full Text] [Related]

  • 5. 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 Nov 16; 18(2):96-106. PubMed ID: 29618309
    [Abstract] [Full Text] [Related]

  • 6. 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
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  • 8. 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 04; 96(3):91-6. PubMed ID: 19091614
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  • 9. Promoter-specific lentivectors for long-term, cardiac-directed therapy of Fabry disease.
    Lee CJ, Fan X, Guo X, Medin JA.
    J Cardiol; 2011 Jan 04; 57(1):115-22. PubMed ID: 20846825
    [Abstract] [Full Text] [Related]

  • 10. Expression of genes and their responses to enzyme replacement therapy in a Fabry disease mouse model.
    Park ES, Choi JO, Park JW, Lee MH, Park HY, Jung SC.
    Int J Mol Med; 2009 Sep 04; 24(3):401-7. PubMed ID: 19639234
    [Abstract] [Full Text] [Related]

  • 11. 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
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  • 14. 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]

  • 15. 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]

  • 16. 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 Nov 03; 143(1-2):108545. PubMed ID: 39068683
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  • 18. 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
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  • 19. Therapeutic advances in Fabry disease: The future awaits.
    Kant S, Atta MG.
    Biomed Pharmacother; 2020 Nov 15; 131():110779. PubMed ID: 33152937
    [Abstract] [Full Text] [Related]

  • 20. Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.
    Marshall J, Ashe KM, Bangari D, McEachern K, Chuang WL, Pacheco J, Copeland DP, Desnick RJ, Shayman JA, Scheule RK, Cheng SH.
    PLoS One; 2010 Nov 24; 5(11):e15033. PubMed ID: 21124789
    [Abstract] [Full Text] [Related]

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