Article
Glucosylceramide synthase inhibition alleviates aberrations in synucleinopathy models.
Proceedings of the National Academy of Sciences of the United States of America - 7 Mar 2017
Sardi S Pablo, Viel Catherine, Clarke Jennifer, Treleaven Christopher M, Richards Amy M, Park Hyejung, Olszewski Maureen A, Dodge James C, Marshall John, Makino Elina, Wang Bing, Sidman Richard L, Cheng Seng H, Shihabuddin Lamya S
Abstract excerpt
Mutations in the glucocerebrosidase gene (GBA) confer a heightened risk of developing Parkinson's disease (PD) and other synucleinopathies, resulting in a lower age of onset and exacerbating disease progression. However, the precise mechanisms by which mutations in GBA increase PD risk and accelerate its progression remain unclear. Here, we investigated the merits of glucosylceramide synthase (GCS) inhibition as...
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