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Journal Abstract Search

259 related items for PubMed ID: 37670350

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  • 2. 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
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  • 4. 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
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  • 9. 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 03; 30(6):487-502. PubMed ID: 36631545
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  • 10. Nicotiana benthamiana α-galactosidase A1.1 can functionally complement human α-galactosidase A deficiency associated with Fabry disease.
    Kytidou K, Beekwilder J, Artola M, van Meel E, Wilbers RHP, Moolenaar GF, Goosen N, Ferraz MJ, Katzy R, Voskamp P, Florea BI, Hokke CH, Overkleeft HS, Schots A, Bosch D, Pannu N, Aerts JMFG.
    J Biol Chem; 2018 Jun 29; 293(26):10042-10058. PubMed ID: 29674318
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  • 14. 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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  • 15. 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 04; 130(3):209-214. PubMed ID: 32418857
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  • 16. The Ckd. Qld fabRy Epidemiology (aCQuiRE) study protocol: identifying the prevalence of Fabry disease amongst patients with kidney disease in Queensland, Australia.
    Mallett A, Kearey P, Cameron A, Healy H, Denaro C, Thomas M, Lee VW, Stark S, Fuller M, Hoy WE.
    BMC Nephrol; 2020 Feb 22; 21(1):58. PubMed ID: 32087678
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  • 17. 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 22; 35(17-18):680-694. PubMed ID: 38970423
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  • 19. 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 22; 35(5-6):192-201. PubMed ID: 38386497
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