Publication Name	World Journal of Stem Cells
Manuscript ID	120578
DOI	10.4252/wjsc.120578
Country	China

Category	Clinical Neurology
Manuscript Type	Basic Study
Article Title	Integrated patient induced pluripotent stem cell models and GAA-/- mice reveal central nervous system neural pathology in infantile-onset Pompe disease
Manuscript Source	Unsolicited Manuscript
All Author List	Yi-Chang Jiao, Dan-Dan Zhao, Hai-Yan Zhang and Fu-Chen Liu
Funding Agency and Grant Number	Funding Agency Grant Number Natural Science Foundation of Shandong Province ZR2023MH180
Corresponding Author	Fu-Chen Liu, MD, PhD, Professor, Department of Neurology, Qilu Hospital of Shandong University, No. 107 West Wenhua Road, Jinan 250012, Shandong Province, China. fuchen.liu@email.sdu.edu.cn
Key Words	Pompe disease; Lysosomal storage disease; Central nervous system; Blood-brain barrier; Induced pluripotent stem cells
Core Tip	This study integrates patient-derived induced pluripotent stem cell neural lineages and GAA-/- mice to model central nervous system pathology in infantile-onset Pompe disease. Patient-specific cortical neurons, astrocytes, oligodendrocytes, and motor neurons recapitulate key lysosomal pathology seen in vivo. GAA-/- mice exhibit progressive glycogen accumulation, astrogliosis, and neuronal loss. This dual-platform approach faithfully reproduces infantile-onset Pompe disease neural features, providing a valuable system for mechanistic research and testing central nervous system-directed therapies.
Citation	Jiao YC, Zhao DD, Zhang HY, Liu FC. Integrated patient induced pluripotent stem cell models and GAA-/- mice reveal central nervous system neural pathology in infantile-onset Pompe disease. World J Stem Cells 2026; In press DOI: https://doi.org/10.4252/wjsc.120578
PDF	120578-in-press.pdf

Received	2026-03-03 02:31
Peer-Review Started	2026-03-03 02:34
First Decision by Editorial Office Director	2026-03-26 10:14
Return for Revision	2026-03-26 10:14
Revised	2026-04-03 17:15
Publication Fee Transferred	2026-04-09 06:51
Second Decision by Editor	2026-05-29 03:08
Second Decision by Editor-in-Chief
Final Decision by Editorial Office Director	2026-06-05 08:37
Articles in Press	2026-06-05 08:37
Edit the Manuscript by Language Editor
Typeset the Manuscript

ISSN	1948-0210 (online)
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Copyright	©Author(s) (or their employer(s)) 2026. No commercial re-use. See Permissions. Published by Baishideng Publishing Group Inc.
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