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A double-hit <i>in vivo</i> model of <i>GBA1</i> viral microRNA-mediated downregulation and human alpha-synuclein overexpression demonstrates nigrostriatal degeneration

2021-06-23

Abstract excerpt

Preclinical and clinical studies support a strong association between mutations in the GBA1 gene that encodes β-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 neurodege...

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Literature Corpus work
17521e21-11d2-5ce4-a2e1-b18169fdeb81
DOI
10.1101/2021.06.23.449545
Open publication

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A double-hit <i>in vivo</i> model of <i>GBA1</i> viral microRNA-mediated downregulation and human alpha-synuclein overexpression demonstrates nigrostriatal degenerationDOI 10.1101/2021.06.23.449545
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