A new type of pharmacological chaperone for G M1 -gangliosidosis related human lysosomal β-galactosidase: N -Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols
2017; Elsevier BV; Volume: 27; Issue: 15 Linguagem: Inglês
10.1016/j.bmcl.2017.05.086
ISSN1464-3405
AutoresMichael Schalli, Patrick Weber, Christina Tysoe, Bettina M. Pabst, Martin Thonhofer, Eduard Paschke, Arnold Stütz, Marion Tschernutter, Werner Windischhofer, Stephen G. Withers,
Tópico(s)Cellular transport and secretion
ResumoN-Functionalized amino(hydroxymethyl)cyclopentanetriols are potent inhibitors of β-d-galactosidases and, for the first time, could be shown to act as pharmacological chaperones for GM1-gangliosidosis-associated lysosomal acid β-galactosidase thus representing a new structural type of pharmacological chaperones for this lysosomal storage disease.
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