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...

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Detalhes bibliográficos
Publicado no:J Am Chem Soc
Main Authors: 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.
Formato: Artigo
Idioma:Inglês
Publicado em: American Chemical Society 2019
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6418866/
https://ncbi.nlm.nih.gov/pubmed/30811188
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/jacs.9b00056
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