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A modulator of wild-type glucocerebrosidase improves pathogenic phenotypes in dopaminergic neuronal models of Parkinson’s disease
Mutations in the GBA1 gene encoding the lysosomal enzyme β-glucocerebrosidase (GCase) represent the most common risk factor for Parkinson’s disease (PD). GCase has been identified as a potential therapeutic target for PD and current efforts are focused on using chemical chaperones to translocate mut...
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| Pubblicato in: | Sci Transl Med |
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| Autori principali: | , , , , , |
| Natura: | Artigo |
| Lingua: | Inglês |
| Pubblicazione: |
2019
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| Soggetti: | |
| Accesso online: | https://ncbi.nlm.nih.gov/pmc/articles/PMC7359409/ https://ncbi.nlm.nih.gov/pubmed/31619543 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1126/scitranslmed.aau6870 |
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