iPSC-derived neural precursor cells engineering GBA1 recovers acid β-glucosidase deficiency and diminishes α-synuclein and neuropathology

iPSC-derived neural precursor cells engineering GBA1 recovers acid β-glucosidase deficiency and diminishes α-synuclein and neuropathology

Mutations in GBA1, encoding the lysosomal acid β-glucosidase (GCase), cause neuronopathic Gaucher disease (nGD) and promote Parkinson disease (PD). The mutations on GBA1 include deletion and missense mutations that are pathological and lead to GCase deficiency in Gaucher disease. Both nGD and PD lac...

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Bibliographic Details
Main Authors: Yanyan Peng, Benjamin Liou, Yi Lin, Christopher N. Mayhew, Sheila M. Fleming, Ying Sun
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
cell therapy
neuronopathic Gaucher disease
Parkinson disease
neurodegeneration
neural precursor cell
acid β-glucosidase
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123000438

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http://www.sciencedirect.com/science/article/pii/S2329050123000438

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