Synthesis of a New β-Galactosidase Inhibitor Displaying Pharmacological Chaperone Properties for GM1 Gangliosidosis

GM1 gangliosidosis is a rare lysosomal disease caused by the deficiency of the enzyme β-galactosidase (β-Gal; GLB1; E.C. 3.2.1.23), responsible for the hydrolysis of terminal β-galactosyl residues from GM1 ganglioside, glycoproteins, and glycosaminoglycans, such as keratan-sulfate. With the aim of i...

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Detalles Bibliográficos
Autores principales: Clemente, Francesca, Martínez-Bailén, Macarena, Matassini, Camilla, Morrone, Amelia, Falliano, Silvia, Caciotti, Anna, Paoli, Paolo, Goti, Andrea, Cardona, Francesca
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9268699/
https://www.ncbi.nlm.nih.gov/pubmed/35807262
http://dx.doi.org/10.3390/molecules27134008

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9268699/
https://www.ncbi.nlm.nih.gov/pubmed/35807262
http://dx.doi.org/10.3390/molecules27134008

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