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288 related items for PubMed ID: 19773742

  • 1. The pharmacological chaperone 1-deoxygalactonojirimycin reduces tissue globotriaosylceramide levels in a mouse model of Fabry disease.
    Khanna R, Soska R, Lun Y, Feng J, Frascella M, Young B, Brignol N, Pellegrino L, Sitaraman SA, Desnick RJ, Benjamin ER, Lockhart DJ, Valenzano KJ.
    Mol Ther; 2010 Jan; 18(1):23-33. PubMed ID: 19773742
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  • 3. The pharmacological chaperone 1-deoxygalactonojirimycin increases alpha-galactosidase A levels in Fabry patient cell lines.
    Benjamin ER, Flanagan JJ, Schilling A, Chang HH, Agarwal L, Katz E, Wu X, Pine C, Wustman B, Desnick RJ, Lockhart DJ, Valenzano KJ.
    J Inherit Metab Dis; 2009 Jun; 32(3):424-40. PubMed ID: 19387866
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  • 4. Preclinical efficacy and safety of 1-deoxygalactonojirimycin in mice for Fabry disease.
    Ishii S, Chang HH, Yoshioka H, Shimada T, Mannen K, Higuchi Y, Taguchi A, Fan JQ.
    J Pharmacol Exp Ther; 2009 Mar; 328(3):723-31. PubMed ID: 19106170
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  • 5. Increased globotriaosylceramide levels in a transgenic mouse expressing human alpha1,4-galactosyltransferase and a mouse model for treating Fabry disease.
    Shiozuka C, Taguchi A, Matsuda J, Noguchi Y, Kunieda T, Uchio-Yamada K, Yoshioka H, Hamanaka R, Yano S, Yokoyama S, Mannen K, Kulkarni AB, Furukawa K, Ishii S.
    J Biochem; 2011 Feb; 149(2):161-70. PubMed ID: 20961863
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  • 6. 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 Feb; 8(3):e57631. PubMed ID: 23472096
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  • 8. Chaperone Therapy in Fabry Disease.
    Weidemann F, Jovanovic A, Herrmann K, Vardarli I.
    Int J Mol Sci; 2022 Feb 08; 23(3):. PubMed ID: 35163813
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  • 9. Mutant alpha-galactosidase A enzymes identified in Fabry disease patients with residual enzyme activity: biochemical characterization and restoration of normal intracellular processing by 1-deoxygalactonojirimycin.
    Ishii S, Chang HH, Kawasaki K, Yasuda K, Wu HL, Garman SC, Fan JQ.
    Biochem J; 2007 Sep 01; 406(2):285-95. PubMed ID: 17555407
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  • 13. Molecular basis of 1-deoxygalactonojirimycin arylthiourea binding to human α-galactosidase a: pharmacological chaperoning efficacy on Fabry disease mutants.
    Yu Y, Mena-Barragán T, Higaki K, Johnson JL, Drury JE, Lieberman RL, Nakasone N, Ninomiya H, Tsukimura T, Sakuraba H, Suzuki Y, Nanba E, Mellet CO, García Fernández JM, Ohno K.
    ACS Chem Biol; 2014 Jul 18; 9(7):1460-9. PubMed ID: 24783948
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  • 14. Safety and pharmacodynamic effects of a pharmacological chaperone on α-galactosidase A activity and globotriaosylceramide clearance in Fabry disease: report from two phase 2 clinical studies.
    Germain DP, Giugliani R, Hughes DA, Mehta A, Nicholls K, Barisoni L, Jennette CJ, Bragat A, Castelli J, Sitaraman S, Lockhart DJ, Boudes PF.
    Orphanet J Rare Dis; 2012 Nov 24; 7():91. PubMed ID: 23176611
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  • 15. 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
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  • 16. Computational and modeling approaches to understand the impact of the Fabry's disease causing mutation (D92Y) on the interaction with pharmacological chaperone 1-deoxygalactonojirimycin (DGJ).
    Thirumal Kumar D, Judith E, Priyadharshini Christy J, Siva R, Tayubi IA, Chakraborty C, George Priya Doss C, Zayed H.
    Adv Protein Chem Struct Biol; 2019 Jul 01; 114():341-407. PubMed ID: 30635085
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  • 18. Partial correction of the alpha-galactosidase A deficiency and reduction of glycolipid storage in Fabry mice using synthetic vectors.
    Przybylska M, Wu IH, Zhao H, Ziegler RJ, Tousignant JD, Desnick RJ, Scheule RK, Cheng SH, Yew NS.
    J Gene Med; 2004 Jan 01; 6(1):85-92. PubMed ID: 14716680
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  • 19. Rescue of mutant alpha-galactosidase A in the endoplasmic reticulum by 1-deoxygalactonojirimycin leads to trafficking to lysosomes.
    Hamanaka R, Shinohara T, Yano S, Nakamura M, Yasuda A, Yokoyama S, Fan JQ, Kawasaki K, Watanabe M, Ishii S.
    Biochim Biophys Acta; 2008 Jun 01; 1782(6):408-13. PubMed ID: 18381081
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