Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice

Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice

Mucopolysaccharidosis type I (MPS I) is a metabolic disorder characterized by a deficiency in α-l-iduronidase (IDUA), leading to impaired glycosaminoglycan degradation. Current approved treatments seek to restore IDUA levels via enzyme replacement therapy (ERT) and/or hematopoietic stem cell transpl...

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Bibliographic Details
Main Authors: Caitlin C. Calhoun, Shih-Hsin Kan, Alexander E. Stover, Jerry F. Harb, Edwin S. Monuki, Raymond Y. Wang, Philip H. Schwartz
Format: Article
Language:English
Published: Elsevier 2024-12-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
cell- and tissue-based therapy
induced pluripotent stem cell
neural stem cell
NSC
cell differentiation
neurodegenerative diseases
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124001839
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