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AAV delivery of <i>GBA1</i> suppresses α-synuclein accumulation in Parkinson’s disease models and restores motor dysfunction in a Gaucher’s disease model

2024-06-26

Abstract excerpt

Biallelic mutations in the glucosylceramidase beta 1 ( GBA1 ) gene are the underlying genetic cause of Gaucher’s disease (GD), resulting in a deficient lysosomal hydrolase and subsequent accumulation of glycosphingolipids. More recently, GBA1 mutations have been identified as the most prevalent genetic risk factor for Parkinson’s disease (PD), associated with more pronounced symptoms characterized by earlier on...

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Literature Corpus work
7b6ade76-6928-5c9e-b4eb-ddb258b1dbcf
DOI
10.1101/2024.06.25.599664
Open publication

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AAV delivery of <i>GBA1</i> suppresses α-synuclein accumulation in Parkinson’s disease models and restores motor dysfunction in a Gaucher’s disease modelDOI 10.1101/2024.06.25.599664
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