Synthetic mRNA‐based differentiation method enables early detection of Parkinson's phenotypes in neurons derived from Gaucher disease‐induced pluripotent stem cells

Abstract 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 accompa...

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Bibliographic Details
Main Authors: Tomohiko Akiyama, Saeko Sato, Shigeru B. H. Ko, Osamu Sano, Sho Sato, Masayo Saito, Hiroaki Nagai, Minoru S. H. Ko, Hidehisa Iwata
Format: Article
Language:English
Published: Wiley 2021-04-01
Series:Stem Cells Translational Medicine
Subjects:
Online Access:https://doi.org/10.1002/sctm.20-0302