1.
Chem Commun (Camb)
; 48(52): 6514-6, 2012 Jul 04.
Artigo
em Inglês
| MEDLINE
| ID: mdl-22618082
RESUMO
Competitive inhibitors of either α-galactosidase (α-Gal) or ß-galactosidase (ß-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 ß-Gal associated with Fabry disease and GM(1) gangliosidosis.