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

*Korrespondierende/r Autor/-in für diese Arbeit

Publikation: Beitrag in einer FachzeitschriftArtikelBegutachtung


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)]
Seiten (von - bis)15-22
FachzeitschriftCarbohydrate Research
PublikationsstatusVeröffentlicht - 2017


  • crystal mol structure dioxolo cyclopenta isoxazolol
  • isopropylidenedioxy cyclopenta isoxazole crystal mol structure
  • amino hydroxymethyl cyclopentanetriol prepn pharmacol chaperone gangliosidosis
  • human lysosomal galactosidase amino hydroxymethyl cyclopentanetriol pharmacol chaperone

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