N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols: A new family of activity promotors for a GM1-gangliosidosis related human lysosomal β-galactosidase mutant.

Michael Schalli, Christina Tysoe, Roland Fischer, Bettina M. Pabst, Martin Thonhofer, Eduard Paschke, Tanja Rappitsch, Arnold Stütz*, Marion Tschernutter, Werner Windischhofer, Stephen G Withers

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


From 1,2;3,4-di-O-isopropylidene-α-D-galactopyranose, a series of highly functionalized (hydroxymethyl)cyclopentanes was easily available. In line with reports by Reymond and Jager on similar structures, these amine contg. basic carbasugars are potent inhibitors of β-D-galactosidases and, for the first time, could be shown to act as pharmacol. chaperones for GM1-gangliosidosis-assocd. lysosomal acid β-galactosidase mutant R201C, thus representing a new structural type of pharmacol. chaperones for this lysosomal storage disease. [on SciFinder(R)]
Original languageGerman
Pages (from-to)15-22
Number of pages8
JournalCarbohydrate Research
Publication statusPublished - 2017

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