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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Vydáno v:Int J Mol Sci
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: MDPI 2021
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On-line přístup: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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