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Journal Abstract Search

133 related items for PubMed ID: 39068683

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  • 2. 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; 35(17-18):680-694. PubMed ID: 38970423
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  • 4. 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 Sep; 18(2):96-106. PubMed ID: 29618309
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  • 8. 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; 25(12):e3560. PubMed ID: 37392007
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  • 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; 96(3):91-6. PubMed ID: 19091614
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  • 10. 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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  • 14. 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
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  • 15. 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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  • 17. 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
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