Isofagomine In Vivo Effects in a Neuronopathic Gaucher Disease Mouse

The pharmacological chaperone, isofagomine (IFG), enhances acid β-glucosidase (GCase) function by altering folding, trafficking, and activity in wild-type and Gaucher disease fibroblasts. The in vivo effects of IFG on GCase activity, its substrate levels, and phenotype were evaluated using a neurono...

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Detalhes bibliográficos
Main Authors: Sun, Ying, Ran, Huimin, Liou, Benjamin, Quinn, Brian, Zamzow, Matt, Zhang, Wujuan, Bielawski, Jacek, Kitatani, Kazuyuki, Setchell, Kenneth D. R., Hannun, Yusuf A., Grabowski, Gregory A.
Formato: Artigo
Idioma:Inglês
Publicado em: Public Library of Science 2011
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3080394/
https://ncbi.nlm.nih.gov/pubmed/21533102
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1371/journal.pone.0019037
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