sp2-Iminosugars targeting human lysosomal β-hexosaminidase as pharmacological chaperone candidates for late-onset Tay-Sachs disease

sp2-Iminosugars targeting human lysosomal β-hexosaminidase as pharmacological chaperone candidates for late-onset Tay-Sachs disease

The late-onset form of Tay-Sachs disease displays when the activity levels of human β-hexosaminidase A (HexA) fall below 10% of normal, due to mutations that destabilise the native folded form of the enzyme and impair its trafficking to the lysosome. Competitive inhibitors of HexA can rescue disease...

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Bibliographic Details
Main Authors: Manuel González-Cuesta, Irene Herrera-González, M. Isabel García-Moreno, Roger A. Ashmus, David J. Vocadlo, José M. García Fernández, Eiji Nanba, Katsumi Higaki, Carmen Ortiz Mellet
Format: Article
Language:English
Published: Taylor & Francis Group 2022-12-01
Series:Journal of Enzyme Inhibition and Medicinal Chemistry
Subjects:
Iminosugar
pharmacological chaperone
thiourea
thiazolidine
Tay-Sachs
Online Access:https://www.tandfonline.com/doi/10.1080/14756366.2022.2073444

Internet

https://www.tandfonline.com/doi/10.1080/14756366.2022.2073444

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