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

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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Bibliographic Details
Main Authors: Diego Sucunza, Alberto J. Rico, Elvira Roda, María Collantes, Gloria González-Aseguinolaza, Ana I. Rodríguez-Pérez, Iván Peñuelas, Alfonso Vázquez, José L. Labandeira-García, Vania Broccoli, José L. Lanciego
Format: Article
Language:English
Published: MDPI AG 2021-05-01
Series:International Journal of Molecular Sciences
Subjects:
GBA1
adeno-associated viral vectors
synucleinopathies
neuroprotection
Parkinson’s disease
Online Access:https://www.mdpi.com/1422-0067/22/9/4825

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