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Functionalized Cyclophellitols Are Selective Glucocerebrosidase Inhibitors and Induce a Bona Fide Neuropathic Gaucher Model in Zebrafish
[Image: see text] Gaucher disease is caused by inherited deficiency in glucocerebrosidase (GBA, a retaining β-glucosidase), and deficiency in GBA constitutes the largest known genetic risk factor for Parkinson’s disease. In the past, animal models of Gaucher disease have been generated by treatment...
Autores principales: | Artola, Marta, Kuo, Chi-Lin, Lelieveld, Lindsey T., Rowland, Rhianna J., van der Marel, Gijsbert A., Codée, Jeroen D. C., Boot, Rolf G., Davies, Gideon J., Aerts, Johannes M. F. G., Overkleeft, Herman S. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
American Chemical
Society
2019
|
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6418866/ https://www.ncbi.nlm.nih.gov/pubmed/30811188 http://dx.doi.org/10.1021/jacs.9b00056 |
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