ArticleCell death & disease2020
Human iNSC-derived brain organoid model of lysosomal storage disorder in Niemann-Pick disease type C.
Article in Cell death & disease, 2020. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers, 1 of them a synthesis that pooled it.
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Who cites it
23 citing papers in PubMed, 1 synthesis or guideline pooled it, 34 citations in OpenAlex.
- Mapping the Cerebral Organoid Landscape: A Systematic Review of Preclinical 3D Models in Neuroscience.Advanced healthcare materials · 2026Pooled it
- Short-lived Niemann-Pick type C mice with accelerated brain aging as a novel model for Alzheimer's disease research.Neural regeneration research · 2026Article
- Organoids for Metabolic Disease Modeling.Journal of inherited metabolic disease · 2026Review
- Neural organoids as advanced tools for neurotoxicity modeling.Current research in toxicology · 2025Review
- Differential gene expression patterns in Niemann-Pick Type C and Tay-Sachs diseases: Implications for neurodegenerative mechanisms.PloS one · 2025Article
- Autophagy-NADFrontiers in molecular biosciences · 2025Review
- Endo-lysosomal dysfunction and neuronal-glial crosstalk in Niemann-Pick type C disease.Philosophical transactions of the Royal Society of London. Series B, Biological sciences · 2024Review
- Brain Organoids: A Game-Changer for Drug Testing.Pharmaceutics · 2024Review
- Differently increased volumes of multiple brain areas inFrontiers in neuroanatomy · 2024Article
- Open and remotely accessible Neuroplatform for research in wetware computing.Frontiers in artificial intelligence · 2024Article
- Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.Advanced drug delivery reviews · 2023Review
- Zebra-Sphinx: Modeling Sphingolipidoses in Zebrafish.International journal of molecular sciences · 2023Review
- The Effect of a Novel Mica Nanoparticle, STB-MP, on an Alzheimer's Disease Patient-Induced PSC-Derived Cortical Brain Organoid Model.Nanomaterials (Basel, Switzerland) · 2023Article
- The Cerebellum in Niemann-Pick C1 Disease: Mouse Versus Man.Cerebellum (London, England) · 2023Review
- Severe neurometabolic phenotype inFrontiers in molecular neuroscience · 2023Article
- Fountain of youth-Targeting autophagy in aging.Frontiers in aging neuroscience · 2023Review
- Zika virus infection accelerates Alzheimer's disease phenotypes in brain organoids.Cell death discovery · 2022Article
- Upregulation of SNAP25 by HDAC inhibition ameliorates Niemann-Pick Type C disease phenotypes via autophagy induction.Clinical and translational medicine · 2022Article
- Vascularization of iNSC spheroid in a 3D spheroid-on-a-chip platform enhances neural maturation.Biotechnology and bioengineering · 2022Article
- Accumulation of APP-CTF induces mitophagy dysfunction in the iNSCs model of Alzheimer's disease.Cell death discovery · 2022Article
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Authors and funding
7 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Recent studies on developing three-dimensional (3D) brain organoids from stem cells have allowed the generation of in vitro models of neural disease and have enabled the screening of drugs because these organoids mimic the complexity of neural tissue. Niemann-Pick disease, type C (NPC) is a neurodegenerative lysosomal storage disorder caused by mutations in the NPC1 or NPC2. The pathological features underlying NPC are characterized by the abnormal accumulation of cholesterol in acidic compartments, including late endosomes and lysosomes. Due to the inaccessibility of brain tissues from human NPC patients, we developed NPC brain organoids with induced neural stem cells from NPC patient-derived fibroblasts. NPC organoids exhibit significantly reduced size and proliferative ability, which are accompanied by accumulation of cholesterol, impairment in neuronal differentiation, and autophagic flux and dysfunction of lysosomes; therefore, NPC organoids can recapitulate the main phenotypes of NPC patients. Furthermore, these pathological phenotypes observed in NPC organoids were reversed by treatment with valproic acid and HPBCD, which are known to be an effective treatment for several neurodegenerative diseases. Our data present patient-specific phenotypes in 3D organoid-based models of NPC and highlight the application of this model to drug screening in vitro.
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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.