Article
Synthetic mRNA-based differentiation method enables early detection of Parkinson's phenotypes in neurons derived from Gaucher disease-induced pluripotent stem cells.
Stem cells translational medicine - 1 Apr 2021
Akiyama Tomohiko, Sato Saeko, Ko Shigeru B H, Sano Osamu, Sato Sho, Saito Masayo, Nagai Hiroaki, Ko Minoru S H, Iwata Hidehisa
Abstract excerpt
Gaucher disease, the most prevalent metabolic storage disorder, is caused by mutations in the glucocerebrosidase gene GBA1, which lead to the accumulation of glucosylceramide (GlcCer) in affected cells. Gaucher disease type 1 (GD1), although defined as a nonneuronopathic subtype, is accompanied by an increased risk of Parkinson's disease. To gain insights into the association of progressive accumulation of GlcCer...
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