Glucocerebrosidase Gene Therapy Induces Alpha-Synuclein Clearance and Neuroprotection of Midbrain Dopaminergic Neurons in Mice and Macaques

Mutations in the GBA1 gene coding for glucocerebrosidase (GCase) are the main genetic risk factor for Parkinson’s disease (PD). Indeed, identifying reduced GCase activity as a common feature underlying the typical neuropathological signatures of PD—even when considering idiopathic forms of PD—has re...

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Pubblicato in:Int J Mol Sci
Autori principali: Sucunza, Diego, Rico, Alberto J., Roda, Elvira, Collantes, María, González-Aseguinolaza, Gloria, Rodríguez-Pérez, Ana I., Peñuelas, Iván, Vázquez, Alfonso, Labandeira-García, José L., Broccoli, Vania, Lanciego, José L.
Natura: Artigo
Lingua:Inglês
Pubblicazione: MDPI 2021
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC8125775/
https://ncbi.nlm.nih.gov/pubmed/34062940
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms22094825
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