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Journal Abstract Search


227 related items for PubMed ID: 23337983

  • 1. A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis.
    Takai T, Higaki K, Aguilar-Moncayo M, Mena-Barragán T, Hirano Y, Yura K, Yu L, Ninomiya H, García-Moreno MI, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y.
    Mol Ther; 2013 Mar; 21(3):526-32. PubMed ID: 23337983
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  • 4. Fluorous iminoalditols act as effective pharmacological chaperones against gene products from GLB₁ alleles causing GM1-gangliosidosis and Morquio B disease.
    Fantur KM, Wrodnigg TM, Stütz AE, Pabst BM, Paschke E.
    J Inherit Metab Dis; 2012 May; 35(3):495-503. PubMed ID: 22033734
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  • 5. Structural basis of pharmacological chaperoning for human β-galactosidase.
    Suzuki H, Ohto U, Higaki K, Mena-Barragán T, Aguilar-Moncayo M, Ortiz Mellet C, Nanba E, Garcia Fernandez JM, Suzuki Y, Shimizu T.
    J Biol Chem; 2014 May 23; 289(21):14560-8. PubMed ID: 24737316
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  • 6. N-butyldeoxygalactonojirimycin reduces neonatal brain ganglioside content in a mouse model of GM1 gangliosidosis.
    Kasperzyk JL, El-Abbadi MM, Hauser EC, D'Azzo A, Platt FM, Seyfried TN.
    J Neurochem; 2004 May 23; 89(3):645-53. PubMed ID: 15086521
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  • 7. Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis.
    Aguilar-Moncayo M, Takai T, Higaki K, Mena-Barragán T, Hirano Y, Yura K, Li L, Yu Y, Ninomiya H, García-Moreno MI, Ishii S, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández JM, Suzuki Y.
    Chem Commun (Camb); 2012 Jul 04; 48(52):6514-6. PubMed ID: 22618082
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  • 9. Evaluation of N-nonyl-deoxygalactonojirimycin as a pharmacological chaperone for human GM1 gangliosidosis leads to identification of a feline model suitable for testing enzyme enhancement therapy.
    Rigat BA, Tropak MB, Buttner J, Crushell E, Benedict D, Callahan JW, Martin DR, Mahuran DJ.
    Mol Genet Metab; 2012 Sep 04; 107(1-2):203-12. PubMed ID: 22784478
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  • 11. (5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B.
    Front S, Biela-Banaś A, Burda P, Ballhausen D, Higaki K, Caciotti A, Morrone A, Charollais-Thoenig J, Gallienne E, Demotz S, Martin OR.
    Eur J Med Chem; 2017 Jan 27; 126():160-170. PubMed ID: 27750150
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  • 12. Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion.
    Condori J, Acosta W, Ayala J, Katta V, Flory A, Martin R, Radin J, Cramer CL, Radin DN.
    Mol Genet Metab; 2016 Feb 27; 117(2):199-209. PubMed ID: 26766614
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  • 13. Crystal structure of human β-galactosidase: structural basis of Gm1 gangliosidosis and morquio B diseases.
    Ohto U, Usui K, Ochi T, Yuki K, Satow Y, Shimizu T.
    J Biol Chem; 2012 Jan 13; 287(3):1801-12. PubMed ID: 22128166
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  • 17. Pharmacological chaperone corrects lysosomal storage in Fabry disease caused by trafficking-incompetent variants.
    Yam GH, Bosshard N, Zuber C, Steinmann B, Roth J.
    Am J Physiol Cell Physiol; 2006 Apr 13; 290(4):C1076-82. PubMed ID: 16531566
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  • 18. Substrate reduction reduces gangliosides in postnatal cerebrum-brainstem and cerebellum in GM1 gangliosidosis mice.
    Kasperzyk JL, d'Azzo A, Platt FM, Alroy J, Seyfried TN.
    J Lipid Res; 2005 Apr 13; 46(4):744-51. PubMed ID: 15687347
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