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The molecular basis of pharmacological chaperoning in human α-galactosidase
Fabry disease patients show a deficiency in the activity of the lysosomal enzyme α-galactosidase (α-GAL or α-Gal A). One proposed treatment for Fabry disease is pharmacological chaperone therapy, where a small molecule stabilizes the α-GAL protein, leading to increased enzymatic activity. Using enzy...
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| Hauptverfasser: | , , , |
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
2011
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3246215/ https://ncbi.nlm.nih.gov/pubmed/22195554 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.chembiol.2011.10.012 |
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