Molecular Basis of 1-Deoxygalactonojirimycin Arylthiourea Binding to Human α-Galactosidase A: Pharmacological Chaperoning Efficacy on Fabry Disease Mutants

Author:

Yu Yi12,Mena-Barragán Teresa3,Higaki Katsumi1,Johnson Jennifer L.4,Drury Jason E.4,Lieberman Raquel L.4,Nakasone Naoe5,Ninomiya Haruaki5,Tsukimura Takahiro6,Sakuraba Hitoshi7,Suzuki Yoshiyuki8,Nanba Eiji1,Mellet Carmen Ortiz3,García Fernández José M.9,Ohno Kousaku2

Affiliation:

1. Division of Functional Genomics, Research Center for Bioscience and Technology, Tottori University, Yonago 683-8503, Japan

2. Division of Child Neurology, Institute of Neurological Sciences, Tottori University Faculty of Medicine, Yonago 683-8504, Japan

3. Departamento de Química Orgánica, Facultad de Química, Universidad de Sevilla, Sevilla 41012, Spain

4. School of Chemistry & Biochemistry, Georgia Institute of Technology, Atlanta 30332-0400, Georgia United States

5. Department of Biomedical Regulation, School of Health Science, Tottori University Faculty of Medicine, Yonago 683-8503, Japan

6. Department of Functional Bioanalysis, Meiji Pharmaceutical University, Tokyo 204-8588, Japan

7. Department of Clinical Genetics, Meiji Pharmaceutical University, Tokyo 204-8588, Japan

8. Tokyo Metropolitan Institute of Medical Science, Tokyo 156-0057, Japan

9. Instituto de Investigaciones Químicas (IIQ), CSIC-Universidad de Sevilla, Sevilla 41092, Spain

Publisher

American Chemical Society (ACS)

Subject

Molecular Medicine,General Medicine,Biochemistry

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