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Article

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

International journal of molecular sciences - 1 May 2021

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

Abstract excerpt

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 recently paved the way for designing novel strategies focused on enhancing GCase activity to reduce alpha-synuclein...

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Identifiers

PMID: 34062940
DOI: 10.3390/ijms22094825
PMCID: PMC8125775

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