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254 related items for PubMed ID: 36631545

  • 1. Preclinical evaluation of FLT190, a liver-directed AAV gene therapy for Fabry disease.
    Jeyakumar JM, Kia A, Tam LCS, McIntosh J, Spiewak J, Mills K, Heywood W, Chisari E, Castaldo N, Verhoef D, Hosseini P, Kalcheva P, Cocita C, Miranda CJ, Canavese M, Khinder J, Rosales C, Hughes D, Sheridan R, Corbau R, Nathwani A.
    Gene Ther; 2023 Jun; 30(6):487-502. PubMed ID: 36631545
    [Abstract] [Full Text] [Related]

  • 2. 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]

  • 3. 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]

  • 4. Systematic gene therapy derived from an investigative study of AAV2/8 vector gene therapy for Fabry disease.
    Deng M, Zhou H, He S, Qiu H, Wang Y, Zhao AY, Mu Y, Li F, Zhao AZ.
    Orphanet J Rare Dis; 2023 Sep 05; 18(1):275. PubMed ID: 37670350
    [Abstract] [Full Text] [Related]

  • 5. 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 05; 25(12):e3560. PubMed ID: 37392007
    [Abstract] [Full Text] [Related]

  • 6. Developing Gene Therapy for Mitigating Multisystemic Pathology in Fabry Disease: Proof of Concept in an Aggravated Mouse Model.
    Boukharov N, Yuan S, Ruangsirluk W, Ayyadurai S, Rahman A, Rivera-Hernandez M, Sunkara S, Tonini K, Park EYH, Deshpande M, Islam R.
    Hum Gene Ther; 2024 Sep 05; 35(17-18):680-694. PubMed ID: 38970423
    [Abstract] [Full Text] [Related]

  • 7. 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]

  • 8. 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]

  • 9. 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]

  • 10. 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]

  • 11. AAV2/6 Gene Therapy in a Murine Model of Fabry Disease Results in Supraphysiological Enzyme Activity and Effective Substrate Reduction.
    Yasuda M, Huston MW, Pagant S, Gan L, St Martin S, Sproul S, Richards D, Ballaron S, Hettini K, Ledeboer A, Falese L, Cao L, Lu Y, Holmes MC, Meyer K, Desnick RJ, Wechsler T.
    Mol Ther Methods Clin Dev; 2020 Sep 11; 18():607-619. PubMed ID: 32775495
    [Abstract] [Full Text] [Related]

  • 12. 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 Sep 11; 143(1-2):108545. PubMed ID: 39068683
    [Abstract] [Full Text] [Related]

  • 13. Correction of enzymatic and lysosomal storage defects in Fabry mice by adenovirus-mediated gene transfer.
    Ziegler RJ, Yew NS, Li C, Cherry M, Berthelette P, Romanczuk H, Ioannou YA, Zeidner KM, Desnick RJ, Cheng SH.
    Hum Gene Ther; 1999 Jul 01; 10(10):1667-82. PubMed ID: 10428212
    [Abstract] [Full Text] [Related]

  • 14. Distributions of Globotriaosylceramide Isoforms, and Globotriaosylsphingosine and Its Analogues in an α-Galactosidase A Knockout Mouse, a Model of Fabry Disease.
    Sueoka H, Aoki M, Tsukimura T, Togawa T, Sakuraba H.
    PLoS One; 2015 Jul 01; 10(12):e0144958. PubMed ID: 26661087
    [Abstract] [Full Text] [Related]

  • 15. 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]

  • 16. AAV2 vector harboring a liver-restricted promoter facilitates sustained expression of therapeutic levels of alpha-galactosidase A and the induction of immune tolerance in Fabry mice.
    Ziegler RJ, Lonning SM, Armentano D, Li C, Souza DW, Cherry M, Ford C, Barbon CM, Desnick RJ, Gao G, Wilson JM, Peluso R, Godwin S, Carter BJ, Gregory RJ, Wadsworth SC, Cheng SH.
    Mol Ther; 2004 Feb 05; 9(2):231-40. PubMed ID: 14759807
    [Abstract] [Full Text] [Related]

  • 17. 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]

  • 18. Migalastat HCl reduces globotriaosylsphingosine (lyso-Gb3) in Fabry transgenic mice and in the plasma of Fabry patients.
    Young-Gqamana B, Brignol N, Chang HH, Khanna R, Soska R, Fuller M, Sitaraman SA, Germain DP, Giugliani R, Hughes DA, Mehta A, Nicholls K, Boudes P, Lockhart DJ, Valenzano KJ, Benjamin ER.
    PLoS One; 2013 Mar 18; 8(3):e57631. PubMed ID: 23472096
    [Abstract] [Full Text] [Related]

  • 19. Reduced glucosylceramide in the mouse model of Fabry disease: correction by successful enzyme replacement therapy.
    Quinta R, Rodrigues D, Assunção M, Macedo MF, Azevedo O, Cunha D, Oliveira P, Sá Miranda MC.
    Gene; 2014 Feb 15; 536(1):97-104. PubMed ID: 24334116
    [Abstract] [Full Text] [Related]

  • 20. A comprehensive testing algorithm for the diagnosis of Fabry disease in males and females.
    Stiles AR, Zhang H, Dai J, McCaw P, Beasley J, Rehder C, Koeberl DD, McDonald M, Bali DS, Young SP.
    Mol Genet Metab; 2020 Jul 15; 130(3):209-214. PubMed ID: 32418857
    [Abstract] [Full Text] [Related]

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