(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 (original ) (raw )4-epi-Isofagomine derivatives as pharmacological chaperones for the treatment of lysosomal diseases linked to β-galactosidase mutations: Improved synthesis and biological investigations
Sophie FRONT
Bioorganic & Medicinal Chemistry, 2018
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Synthesis of a New β-Galactosidase Inhibitor Displaying Pharmacological Chaperone Properties for GM1 Gangliosidosis
Paolo Paoli
Molecules
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Synthesis of 1,5-Dideoxy-1,5-iminoribitol C‑Glycosides through a Nitrone−Olefin Cycloaddition Domino Strategy: Identification of Pharmacological Chaperones of Mutant Human Lysosomal β‑Galactosidase
Sougata Ghosh
2014
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Structural basis of pharmacological chaperoning for human -galactosidase
Hironori Suzuki
Journal of Biological Chemistry, 2014
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Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency
Eiji Nanba , Kaori Adachi
Human Mutation, 2011
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Iminosugar-Based Galactoside Mimics as Inhibitors of Galactocerebrosidase: SAR Studies and Comparison with Other Lysosomal Galactosidases
Sophie FRONT
ChemMedChem, 2014
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Stereoselective synthesis of 1-C-alkyl iminogalactitol derivatives, potential chaperones for galactosidase-linked LSDs: a real challenge
Sophie FRONT
Tetrahedron Letters, 2014
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N-Alkyl-, 1-C-Alkyl-, and 5-C-Alkyl-1,5-dideoxy-1,5-imino-(l)-ribitols as Galactosidase Inhibitors
Sophie FRONT
ChemMedChem, 2016
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Pierre Vogel
Bioorganic & Medicinal Chemistry Letters, 1999
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N-Substituted (β-d-galactopyranosylmethyl)amines as reversible inhibitors of β-d-galactosidase
Madhav Yadav
Carbohydrate Research, 1993
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Novel O-glycosyl amino acid mimetics as building blocks for O-glycopeptides act as inhibitors of galactosidases
Joachim Thiem
Bioorganic & Medicinal Chemistry Letters, 2004
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Noel Southall
Current chemical genomics, 2011
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Galactonojirimycin derivatives restore mutant human β-galactosidase activities expressed in fibroblasts from enzyme-deficient knockout mouse
Eiji Nanba
Brain and Development, 2001
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pH-Responsive Pharmacological Chaperones for Rescuing Mutant Glycosidases
Eiji Nanba
Angewandte Chemie, 2015
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Synthesis of lipophilic 1-deoxygalactonojirimycin derivatives as D-galactosidase inhibitors
Chris Tarling
Beilstein Journal of Organic Chemistry, 2010
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Synthesis of β-d-galactofuranosyl nucleoside analogues. A new type of β-d-galactofuranosidase inhibitor
Carla Marino
Carbohydrate Research, 2001
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Probing the Inhibitor versus Chaperone Properties of sp2-Iminosugars towards Human β-Glucocerebrosidase: A Picomolar Chaperone for Gaucher Disease
Roland Pieters
Molecules, 2018
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Inhibitor screening of pharmacological chaperones for lysosomal β-glucocerebrosidase by capillary electrophoresis
Philip Britz-mckibbin
Analytical and Bioanalytical Chemistry, 2011
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Chemical chaperone therapy: Luciferase assay for screening of beta-galactosidase mutations
Eiji Nanba
2010
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Pharmacological chaperones increase residual β-galactocerebrosidase activity in fibroblasts from Krabbe patients
A. Luddi , Paola Piomboni
Molecular Genetics and Metabolism, 2014
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Sugar-mimic glycosidase inhibitors: natural occurrence, biological activity and prospects for therapeutic application
George Fleet
Tetrahedron: Asymmetry, 2000
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1-Thio-β-D-galactofuranosides: synthesis and evaluation as β-D-galactofuranosidase inhibitors
Luiz Claudio Miletti
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A drug targeting motif for glycosidase inhibitors: an iminosugar–boronate shows unexpectedly selective β-galactosidase inhibition
Todd Houston
Tetrahedron Letters, 2002
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Synthesis of α-Galactosyl Ceramide (KRN7000) and Analogues Thereof via a Common Precursor and Their Preliminary Biological Assessment
Gennaro Libero
The Journal of Organic Chemistry, 2008
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Prediction of the responsiveness to pharmacological chaperones: lysosomal human alpha-galactosidase, a case of study
Marco Cammisa , Maria Cubellis
Orphanet Journal of Rare Diseases, 2010
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Synthesis of Galactose- andN-Acetylglucosamine-Derived Tetrazoles and their evaluation as ?-glycosidase inhibitors
Andrea Vasella
Helvetica Chimica Acta, 1995
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1-Thio- -D-galactofuranosides: synthesis and evaluation as -D-galactofuranosidase inhibitors
Carla Marino , Luiz Claudio Miletti
Glycobiology, 1998
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Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion
Carole Cramer
Molecular Genetics and Metabolism, 2016
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Enzyme enhancement therapy through non-competitive pharmacological chaperones
Marc Martinell
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UDP-Gal: GlcNAc-R β1,4-galactosyltransferase—a target enzyme for drug design. Acceptor specificity and inhibition of the enzyme
Jason Vlahakis , Walter Szarek
Glycoconjugate Journal, 2006
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