(5aR)-5a-C-Pentyl-4-epi-isofagomine: A powerful inhibitor of lysosomal β-galactosidase and a remarkable chaperone for mutations associated with GM1-gangliosidosis and Morquio disease type B

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• 作者：Sophie Front^a ; Anna Biela-Banaś^a ; Patricie Burda^b ; Diana Ballhausen^c ; Katsumi Higaki^d ; Anna Caciotti^e ; Amelia Morrone^e ; ^f ; Julie Charollais-Thoenig^g ; Estelle Gallienne^a ; Sté ; phane Demotz^g ; Olivier R. Martin^a ; ^{olivier.martin@univ-orleans.fr}
• 关键词：Galactosidase inhibitors ; GM1-gangliosidosis ; Morquio disease type B ; Iminosugars ; Pharmacological chaperones
• 刊名：European Journal of Medicinal Chemistry
• 出版年：2017
• 出版时间：27 January 2017
• 年：2017
• 卷：126
• 期：Complete
• 页码：160-170
• 全文大小：881 K
• 卷排序：126

文摘

The synthesis of 3 new C-alkylated derivatives of 4-epi-isofagomine is described. Their inhibitory activities and selectivity towards human β-galactosidase were evaluated. Compound 1 behaved as an effective pharmacological chaperone towards several mutations.