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Isofagomine- and 2,5-Anhydro-2,5-Imino-D-Glucitol-Based Glucocerebrosidase Pharmacological Chaperones for Gaucher Disease Intervention

Gaucher disease, resulting from deficient lysosomal glucocerebrosidase (GC) activity, is the most common lysosomal storage disorder. Clinically important GC mutant enzymes typically have reduced specific activity and reduced lysosomal concentration, the latter due to compromised folding and traffick...

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Detalhes bibliográficos
Main Authors: Yu, Zhanqian, Sawkar, Anu R., Whalen, Lisa J., Wong, Chi-Huey, Kelly, Jeffery W.
Formato: Artigo
Idioma:Inglês
Publicado em: 2007
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2543937/
https://ncbi.nlm.nih.gov/pubmed/17201413
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/jm060677i
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