Tailoring the Specificity and Reactivity of a Mechanism??Based Inactivator of Glucocerebrosidase for Potential Therapeutic Applications

BP Rempel, MB Tropak, DJ Mahuran…

文献索引：Rempel, Brian P.; Tropak, Michael B.; Mahuran, Don J.; Withers, Stephen G. Angewandte Chemie - International Edition, 2011 , vol. 50, # 44 p. 10381 - 10383

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被引用次数: 19

摘要

Gaucher s disease [1, 2] is a lysosomal storage disorder that most commonly arises from mutations in lysosomal β glucocerebrosidase (GCase). Mutant GCase is not trafficked to the lysosome correctly, thus leading to an accumulation of unprocessed substrate. While enzyme replacement therapies exist, a potentially less expensive and systemic treatment for Gaucher s disease lies in pharmacological chaperones.[1–3] These small molecules ...