Article

Identification and characterization of nanobodies acting as molecular chaperones for glucocerebrosidase through a novel allosteric mechanism

2024-03-28

Abstract excerpt

The enzyme glucocerebrosidase (GCase) catalyses the hydrolysis of glucosylceramide to glucose and ceramide within lysosomes. Homozygous or compound heterozygous mutations in the GCase-sencoding GBA1 gene cause the lysosomal storage disorder Gaucher disease, while heterozygous mutations are the most frequent genetic risk factor for Parkinson’s disease. These mutations commonly affect GCase stability, trafficking o...

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Literature Corpus work: a776e8a2-e349-5e96-af3b-36a925c5d664
DOI: 10.1101/2024.03.25.586126

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