TY - JOUR
T1 - A new type of pharmacological chaperone for GM1-gangliosidosis related human lysosomal β-galactosidase: N-Substituted 5-amino-1-hydroxymethyl-cyclopentanetriols.
AU - Schalli, Michael
AU - Weber, Patrick
AU - Tysoe, Christina
AU - Pabst, Bettina M.
AU - Thonhofer, Martin
AU - Paschke, Eduard
AU - Stütz, Arnold
AU - Tschernutter, Marion
AU - Windischhofer, Werner
AU - Withers, Stephen G
PY - 2017
Y1 - 2017
N2 - N-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.
AB - N-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.
U2 - 10.1016/j.bmcl.2017.05.086
DO - 10.1016/j.bmcl.2017.05.086
M3 - Article
SN - 0960-894X
VL - 27
SP - 3431
EP - 3435
JO - Bioorganic & Medicinal Chemistry Letters
JF - Bioorganic & Medicinal Chemistry Letters
IS - 15
ER -