Concepedia

α-<scp>d</scp>-Gal-cyclophellitol cyclosulfamidate is a Michaelis complex analog that stabilizes therapeutic lysosomal α-galactosidase A in Fabry disease

Marta Artola, Christinne Hedberg, R.J. Rowland, Lluı́s Raich, Kassiani Kytidou, Liang Wu, Amanda L. Schaaf, Gijsbert A. van der Marel, Jeroen D. C. Codée, Carme Rovira,

Chemical Science · 2019 · 12 citations · 44 references

DOIFull text

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Concepts

Abstract

α-<sc>d</sc>-Gal-cyclophellitol cyclosulfamidate is a new class of neutral, conformationally-constrained competitive glycosidase inhibitor that stabilizes α-gal A and prevents its degradation both <italic>in vitro</italic> and <italic>in cellulo</italic> by mimicry of the Michaelis complex conformation.

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