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Article

Disease modeling for Mucopolysaccharidosis type IIIB using patient derived induced pluripotent stem cells.

Experimental cell research - 1 Oct 2021

Huang Wei, Cheng Yu-Shan, Yang Shu, Swaroop Manju, Xu Miao, Huang Wenwei, Zheng Wei

Abstract excerpt

Mucopolysaccharidosis type IIIB (MPS IIIB) is a lysosomal disease caused by mutations in the NAGLU gene encoding α-N-acetylglucosaminidase (NAGLU) which degrades heparan sulfate in lysosomes. Deficiency in NAGLU results in lysosomal accumulation of glycosaminoglycans (GAGs) and neurological symptoms. Currently, there is no effective treatment or cure for this disease. In this study, induced pluripotent stem cell...

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Identifiers

PMID: 34411609
DOI: 10.1016/j.yexcr.2021.112785
PMCID: PMC8440473

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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