A novel class of allosteric glucosylceramidase beta 1 correctors that reduce cellular stress and enhance lysosomal function
Ilaria Fregno, Natàlia Pérez‐Carmona, Mikhail Rudinskiy, Tatiana Soldà, Timothy Jan Bergmann, Ana Ruano, Aida Delgado, Elena Cubero, Manolo Bellotto, Ana María García-Collazo, Maurizio Molinari
This paper develops two allosteric GCase correctors that improve folding and lysosomal function in GD patient fibroblasts, reducing cellular stress and offering potential therapy for GD, PD, and Dementia with Lewy Bodies.
International Journal of Molecular Sciences · May 6, 2025Peer-reviewedCell Biology