Developing nanobodies as allosteric molecular chaperones of glucocerebrosidase function

Developing nanobodies as allosteric molecular chaperones of glucocerebrosidase function

Abstract The enzyme glucocerebrosidase (GCase) catalyses the hydrolysis of glucosylceramide to glucose and ceramide within lysosomes. Homozygous or compound heterozygous mutations in the GCase-encoding GBA1 gene cause the lysosomal storage disorder Gaucher disease, while heterozygous and homozygous...

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Main Authors: Thomas Dal Maso, Chiara Sinisgalli, Gianluca Zilio, Elisa Franzin, Isabella Tessari, Els Pardon, Jan Steyaert, Steven Ballet, Elisa Greggio, Wim Versées, Nicoletta Plotegher
Format: Article
Language:English
Published: Nature Portfolio 2025-05-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-025-60134-4
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