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Journal Abstract Search

278 related items for PubMed ID: 33775910

  • 1. 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]

  • 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 03; 35(17-18):680-694. PubMed ID: 38970423
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  • 3. 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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  • 4. 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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  • 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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  • 6. 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 27; 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 27; 96(3):91-6. PubMed ID: 19091614
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  • 10. Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In Vivo Genome Editing.
    Laoharawee K, DeKelver RC, Podetz-Pedersen KM, Rohde M, Sproul S, Nguyen HO, Nguyen T, St Martin SJ, Ou L, Tom S, Radeke R, Meyer KE, Holmes MC, Whitley CB, Wechsler T, McIvor RS.
    Mol Ther; 2018 Apr 04; 26(4):1127-1136. PubMed ID: 29580682
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  • 11. 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 Apr 04; 143(1-2):108545. PubMed ID: 39068683
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  • 12. 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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  • 13. 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. 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 Mar 18; 19(5):e0304415. PubMed ID: 38820517
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  • 16. Sequencing and characterization of the porcine α-galactosidase A gene: towards the generation of a porcine model for Fabry disease.
    Yoshimitsu M, Higuchi K, Fan X, Takao S, Medin JA, Tei C, Takenaka T.
    Mol Biol Rep; 2011 Jun 18; 38(5):3145-52. PubMed ID: 20131008
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  • 17. 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. Zinc finger nuclease-mediated targeting of multiple transgenes to an endogenous soybean genomic locus via non-homologous end joining.
    Bonawitz ND, Ainley WM, Itaya A, Chennareddy SR, Cicak T, Effinger K, Jiang K, Mall TK, Marri PR, Samuel JP, Sardesai N, Simpson M, Folkerts O, Sarria R, Webb SR, Gonzalez DO, Simmonds DH, Pareddy DR.
    Plant Biotechnol J; 2019 Apr 15; 17(4):750-761. PubMed ID: 30220095
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  • 20. 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
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