Article
Tuning glycosidase inhibition through aglycone interactions: pharmacological chaperones for Fabry disease and GM1 gangliosidosis.
Chemical communications (Cambridge, England) - 4 Jul 2012
Aguilar-Moncayo M, Takai T, Higaki K, Mena-Barragán T, Hirano Y, Yura K, Li L, Yu Y, Ninomiya H, García-Moreno M I, Ishii S, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, García Fernández J M, Suzuki Y
Abstract excerpt
Competitive inhibitors of either α-galactosidase (α-Gal) or β-galactosidase (β-Gal) with high affinity and selectivity have been accessed by exploiting aglycone interactions with conformationally locked sp(2)-iminosugars. Selected compounds were profiled as potent pharmacological chaperones for mutant lysosomal α- and β-Gal associated with Fabry disease and GM(1) gangliosidosis.
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