Article
A double-hit in vivo model of GBA viral microRNA-mediated downregulation and human alpha-synuclein overexpression demonstrates nigrostriatal degeneration.
Neurobiology of disease - 1 Feb 2022
Polissidis Alexia, Koronaiou Effrosyni, Nikolopoulou Georgia, Viel Catherine, Nikatou Maria, Bogiongko Marios, Sardi S Pablo, Xilouri Maria, Vekrellis Kostas, Stefanis Leonidas
Abstract excerpt
Preclinical and clinical studies support a strong association between mutations in the GBA1 gene that encodes beta-glucocerebrosidase (GCase) (EC 3.2.1.45; glucosylceramidase beta) and Parkinson's disease (PD). Alpha-synuclein (AS), a key player in PD pathogenesis, and GBA1 mutations may independently and synergistically cause lysosomal dysfunction and thus, embody clinically well-validated targets of the...
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