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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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Bibliografski detalji
Glavni autori: Yu, Zhanqian, Sawkar, Anu R., Whalen, Lisa J., Wong, Chi-Huey, Kelly, Jeffery W.
Format: Artigo
Jezik:Inglês
Izdano: 2007
Teme:
Online pristup: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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