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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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| Huvudupphovsmän: | , , , , |
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| Materialtyp: | Artigo |
| Språk: | Inglês |
| Publicerad: |
2007
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| Ämnen: | |
| Länkar: | 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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