ArticlePloS one2013
Impaired neural differentiation of induced pluripotent stem cells generated from a mouse model of Sandhoff disease.
Article in PloS one, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 9 papers.
What it found
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Who cites it
9 citing papers in PubMed, 19 citations in OpenAlex.
- Gene expression changes in Tay-Sachs disease begin early in fetal brain development.Journal of inherited metabolic disease · 2023Article
- Neural Differentiation of Human-Induced Pluripotent Stem Cells (hiPSc) on Surface-Modified Nanofibrous Scaffolds Coated with Platelet-Rich Plasma.Neurochemical research · 2022Article
- Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation.Journal of lipid research · 2018Article
- Induced pluripotent stem cell models of lysosomal storage disorders.Disease models & mechanisms · 2017Review
- Abnormal differentiation of Sandhoff disease model mouse-derived multipotent stem cells toward a neural lineage.PloS one · 2017Article
- TFEB overexpression promotes glycogen clearance of Pompe disease iPSC-derived skeletal muscle.Molecular therapy. Methods & clinical development · 2016Article
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- Comparison of different protocols for neural differentiation of human induced pluripotent stem cells.Molecular biology reports · 2014Article
Corrections and comments
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Authors and funding
9 authors at 2 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sandhoff disease (SD) is a glycosphingolipid storage disease that arises from mutations in the Hexb gene and the resultant deficiency in β-hexosaminidase activity. This deficiency results in aberrant lysosomal accumulation of the ganglioside GM2 and related glycolipids, and progressive deterioration of the central nervous system. Dysfunctional glycolipid storage causes severe neurodegeneration through a poorly understood pathogenic mechanism. Induced pluripotent stem cell (iPSC) technology offers new opportunities for both elucidation of the pathogenesis of diseases and the development of stem cell-based therapies. Here, we report the generation of disease-specific iPSCs from a mouse model of SD. These mouse model-derived iPSCs (SD-iPSCs) exhibited pluripotent stem cell properties and significant accumulation of GM2 ganglioside. In lineage-directed differentiation studies using the stromal cell-derived inducing activity method, SD-iPSCs showed an impaired ability to differentiate into early stage neural precursors. Moreover, fewer neurons differentiated from neural precursors in SD-iPSCs than in the case of the wild type. Recovery of the Hexb gene in SD-iPSCs improved this impairment of neuronal differentiation. These results provide new insights as to understanding the complex pathogenic mechanisms of SD.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.