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Issues for the further development of ambroxol in ALS/MND (Poster Hall at the Palais de Congres Montreal; 517b/c) - Nov 7, 2024 - Abstract #ALSMND2024ALS_MND_589; P2, P3 Ambroxol inhibits non-lysosomal glucosylceramidase (GBA2) while being a chaperone for lysosomal GBA1. GBA2 activity was specifically increased 8
- |||||||||| Journal: Selective labelling of GBA2 in cells with fluorescent ?-d-arabinofuranosyl cyclitol aziridines. (Pubmed Central) - Sep 9, 2024
Co-staining with an antibody selective for the lysosomal ?-glucosylceramidase GBA1, shows distinct subcellular localization of the two enzymes. We proffer our ABP technology for further delineating the role and functioning of GBA2 in disease and propose the ?-d-Araf aziridine scaffold as a good starting point for the development of GBA2-specific inhibitors for clinical development.
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