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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Udgivet i:Int J Mol Sci
Main Authors: 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.
Format: Artigo
Sprog:Inglês
Udgivet: MDPI 2021
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Article
Online adgang: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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