2023
DOI: 10.1021/acs.jmedchem.3c00637
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Design and Pharmacological Chaperone Effects of N-(4′-Phenylbutyl)-DAB Derivatives Targeting the Lipophilic Pocket of Lysosomal Acid α-Glucosidase

Abstract: This study provides the first example of a strategy to design a practical ligand toward lysosomal acid α-glucosidase (GAA) focusing on N-alkyl derivatives of 1,4-dideoxy-1,4-imino-Darabinitol (DAB). The optimized N-4′-(p-trifluoromethylphenyl)butyl-DAB (5g) showed a K i value of 0.73 μM, which was 353-fold higher affinity than N-butyl-DAB (3f) without a terminal phenyl group. Docking analysis showed that the phenyl part of 5g was accommodated in a lipophilic pocket. Furthermore, the ptrifluoromethyl group effe… Show more

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