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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Detalles Bibliográficos
Autores principales: 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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8125775/
https://www.ncbi.nlm.nih.gov/pubmed/34062940
http://dx.doi.org/10.3390/ijms22094825