SCIENCE

GBA PARKINSON'S DISEASE

GBA Parkinson’s disease is arguably the most common genetic form of Parkinson’s disease.  Numerous genetic studies have shown that a single mutation in the GBA1 gene (the gene that causes Gaucher disease) can increase your risk of developing Parkinsons’ disease by up to 30-fold.  In vitro and in vivo preclinical studies suggest that the protein alpha-synuclein can interact with glycolipids and this can lead to aggregation and toxicity.  Studies using GCS inhibitors in mouse models of Parkinson’s disease support that lowering the production of these glycolipids using can reduce synuclein aggregation, slow the progression of neurobehavioral phenotypes, and neurodegeneration.  

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