A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 gangliosidosis

Molecular Therapy

Volumn 21, Issue 3, 2013, Pages 526-532

  Takai, Tomoko ^a   Higaki, Katsumi ^a   Aguilar Moncayo, Matilde ^b   Mena Barragán, Teresa ^b   Hirano, Yuki ^c   Yura, Kei ^c   Yu, Liang ^a,d,h   Ninomiya, Haruaki ^d   García Moreno, M Isabel ^b   Sakakibara, Yasubumi ^e   Ohno, Kousaku ^d   Nanba, Eiji ^a   Ortiz Mellet, Carmen ^b   García Fernández, José M ^f   Suzuki, Yoshiyuki ^g  

^a TOTTORI UNIVERSITY   (Japan)

^b UNIVERSITY OF SEVILLE   (Spain)

^c OCHANOMIZU UNIVERSITY   (Japan)

^d TOTTORI UNIVERSITY   (Japan)

^e KEIO UNIVERSITY   (Japan)

^f INSTITUTO DE INVESTIGACIONES QUÍMICAS   (Spain)

^g INTERNATIONAL UNIVERSITY OF HEALTH AND WELFARE   (Japan)

^h QINGDAO UNIVERSITY MEDICAL COLLEGE   (China)

Author keywords

[No Author keywords available]

Indexed keywords

5N (N' BUTYLIMINOMETHYLIDENE) 6 THIO 1 DEOXYGALACTONOJIRIMYCIN; ALPHA GALACTOSIDASE; ALPHA GLUCOSIDASE; BETA GALACTOSIDASE; BETA GLUCOSIDASE; BICYCLIC 1 DEOXYGALACTONOJIRIMYCIN DERIVATIVE; CHAPERONE; ENZYME INHIBITOR; IMINOSUGAR; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; AUTOPHAGY; BRAIN DISEASE; CLINICAL ARTICLE; COMPETITIVE INHIBITION; COMPUTER MODEL; CONCENTRATION RESPONSE; CONTROLLED STUDY; DRUG EFFECT; DRUG MECHANISM; DRUG MEGADOSE; DRUG STRUCTURE; ENZYMATIC DEGRADATION; ENZYME ACTIVE SITE; ENZYME BINDING; ENZYME CONFORMATION; ENZYME INHIBITION; GM1 GANGLIOSIDOSIS; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; LOW DRUG DOSE; MOUSE; NONHUMAN; NUCLEOTIDE SEQUENCE; STRUCTURE ACTIVITY RELATION;

MUS;

EID: 84875223589     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2012.263     Document Type: Article

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