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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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Main Authors: Guce, Abigail I., Clark, Nathaniel E., Rogich, Jerome J., Garman, Scott C.
格式: Artigo
語言:Inglês
出版: 2011
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在線閱讀: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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