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Article

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

Molecules (Basel, Switzerland) - 22 Jun 2022

Clemente Francesca, Martínez-Bailén Macarena, Matassini Camilla, Morrone Amelia, Falliano Silvia, Caciotti Anna, Paoli Paolo, Goti Andrea, Cardona Francesca

Abstract excerpt

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 identifying new pharmacological chaperones for GM1 gangliosidosis, the synthesis of five new trihydroxypiperidine...

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Identifiers

PMID: 35807262
DOI: 10.3390/molecules27134008
PMCID: PMC9268699

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