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A novel class of allosteric glucosylceramidase beta 1 correctors that reduce cellular stress and enhance lysosomal function

2024-11-15

Abstract excerpt

Mutations in glucosylceramidase beta 1 (GCase) disrupt the protein’s conformational maturation in the endoplasmic reticulum (ER) and hinder its transport to the lysosome. The intralysosomal accumulation of glucocerebrosides, which are substrates of the GCase enzyme, impairs lysosomal function and is linked to Gaucher disease (GD). GCase mutations also increase the risk of Parkinson’s disease (PD) and Dementia with...

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Literature Corpus work
1c071579-7e2e-5b5f-afae-aee60b5e39cb
DOI
10.1101/2024.11.15.623779
Open publication

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