Exploring substituent diversity on pyrrolidine-aryltriazole iminosugars: Structural basis of β-glucocerebrosidase inhibition

The synthesis of a library of pyrrolidine-aryltriazole hybrids through CuAAC between two epimeric dihydroxylated azidomethylpyrrolidines and differently substituted phenylacetylenes is reported. The evaluation of the new compounds as inhibitors of lysosomal β-glucocerebrosidase showed the importance...

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Detalhes bibliográficos
Autores: Martínez Bailén, Macarena, Carmona Asenjo, Ana Teresa, Patterson-Orazem, Athéna C., Lieberman, Raquel L., Ide, Daisuke, Kubo, Moemi, Kato, Atsushi, Robina Ramírez, Inmaculada, Moreno Vargas, Antonio José
Formato: artículo
Estado:Versión aceptada para publicación
Fecha de publicación:2019
País:España
Recursos:Universidad de Sevilla (US)
Repositorio:idUS. Depósito de Investigación de la Universidad de Sevilla
OAI Identifier:oai:idus.us.es:11441/176977
Acesso em linha:https://hdl.handle.net/11441/176977
https://doi.org/10.1016/j.bioorg.2019.02.025
Access Level:acceso abierto
Palavra-chave:B-glucocerebrosidase inhibitors
Iminosugars
Pyrrolidines
Triazoles
Click chemistry
Protein crystallization
Chaperones
Descrição
Resumo:The synthesis of a library of pyrrolidine-aryltriazole hybrids through CuAAC between two epimeric dihydroxylated azidomethylpyrrolidines and differently substituted phenylacetylenes is reported. The evaluation of the new compounds as inhibitors of lysosomal β-glucocerebrosidase showed the importance of the substitution pattern of the phenyl moiety in the inhibition. Crystallization and docking studies revealed key interactions of the pyrrolidine motif with aminoacid residues of the catalytic site while the aryltriazole moiety extended along a hydrophobic surface groove. Some of these compounds were able to increase the enzyme activity in Gaucher patient fibroblasts, acting as a new type of chemical chaperone for Gaucher disease.