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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 acid alpha-glucosidase (GAA) gene. Acid alpha-glucosidase deficiency leads to abnormal glycogen accumulation in patient cells. Given the increasing evidence of central nervous system (CNS) involvement in cla...

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Detalles Bibliográficos
Autores principales:	Cheng, Yu-Shan, Yang, Shu, Hong, Junjie, Li, Rong, Beers, Jeanette, Zou, Jizhong, Huang, Wenwei, Zheng, Wei
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7822217/ https://www.ncbi.nlm.nih.gov/pubmed/33375166 http://dx.doi.org/10.3390/cells10010008

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