Modeling CNS Involvement in Pompe Disease Using Neural Stem Cells Generated from Patient-Derived Induced Pluripotent Stem Cells

Modeling CNS Involvement in Pompe Disease Using Neural Stem Cells Generated from Patient-Derived Induced Pluripotent Stem Cells

Pompe disease is a lysosomal storage disorder caused by autosomal recessive mutations in the <i>acid alpha-glucosidase (GAA)</i> gene. Acid alpha-glucosidase deficiency leads to abnormal glycogen accumulation in patient cells. Given the increasing evidence of central nervous system (CNS)...

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Bibliographic Details
Main Authors: Yu-Shan Cheng, Shu Yang, Junjie Hong, Rong Li, Jeanette Beers, Jizhong Zou, Wenwei Huang, Wei Zheng
Format: Article
Language:English
Published: MDPI AG 2021-12-01
Series:Cells
Subjects:
Pompe disease
lysosomal storage disease
induced pluripotent stem cells
cell-based disease model
neural stem cells
Online Access:https://www.mdpi.com/2073-4409/10/1/8

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