A Bicyclic 1-Deoxygalactonojirimycin Derivative as a Novel Pharmacological Chaperone for GM(1) Gangliosidosis

Lysosomal β-galactosidase (β-Gal) deficiency causes a group of disorders that include neuronopathic GM(1) gangliosidosis and non-neuronopathic Morquio B disease. We have previously proposed the use of small molecule ligands of β-Gal as pharmacological chaperones (PCs) for the treatment of GM(1) gang...

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Detalhes bibliográficos
Main Authors: Takai, Tomoko, Higaki, Katsumi, Aguilar-Moncayo, Matilde, Mena-Barragán, Teresa, Hirano, Yuki, Yura, Kei, Yu, Liang, Ninomiya, Haruaki, García-Moreno, M. Isabel, Sakakibara, Yasubumi, Ohno, Kousaku, Nanba, Eiji, Ortiz Mellet, Carmen, García Fernández, José M., Suzuki, Yoshiyuki
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2013
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3589148/
https://ncbi.nlm.nih.gov/pubmed/23337983
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2012.263
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