A Human Neural Crest Stem Cell-Derived Dopaminergic Neuronal Model Recapitulates Biochemical Abnormalities in GBA1 Mutation Carriers

A Human Neural Crest Stem Cell-Derived Dopaminergic Neuronal Model Recapitulates Biochemical Abnormalities in GBA1 Mutation Carriers

Numerically the most important risk factor for the development of Parkinson's disease (PD) is the presence of mutations in the glucocerebrosidase GBA1 gene. In vitro and in vivo studies show that GBA1 mutations reduce glucocerebrosidase (GCase) activity and are associated with increased α-synuc...

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Bibliographic Details
Main Authors: Shi-Yu Yang, Michelle Beavan, Kai-Yin Chau, Jan-Willem Taanman, Anthony H.V. Schapira
Format: Article
Language:English
Published: Elsevier 2017-03-01
Series:Stem Cell Reports
Subjects:
neural crest stem cells
Parkinson disease
glucocerebrosidase
α-synuclein
chaperone
PD modeling
dopaminergic neurons
GBA1 mutation
ambroxol
GBA1-associated PD
Online Access:http://www.sciencedirect.com/science/article/pii/S2213671117300267

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http://www.sciencedirect.com/science/article/pii/S2213671117300267

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