ArticleMovement disorders : official journal of the Movement Disorder Society2021
Modeling Brain Pathology of Niemann-Pick Disease Type C Using Patient-Derived Neurons.
Article in Movement disorders : official journal of the Movement Disorder Society, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 5 papers.
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Who cites it
5 citing papers in PubMed, 7 citations in OpenAlex.
- Dysregulation of Extracellular Vesicle Concentration, MicroRNAs, and Surface Proteins in Patients With Niemann-Pick Disease Type C.Journal of extracellular biology · 2026Article
- Differently increased volumes of multiple brain areas inFrontiers in neuroanatomy · 2024Article
- Self-Regulatory Neuronal Mechanisms and Long-Term Challenges in Schizophrenia Treatment.Brain sciences · 2023Review
- Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1.Frontiers in pharmacology · 2021Article
- Impaired Retrograde Transport Due to Lack of TBC1D5 Contributes to the Trafficking Defect of Lysosomal Cathepsins in Ischemic/Hypoxic Cardiomyocytes.Frontiers in cardiovascular medicine · 2021Article
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
backgroundNiemann-Pick disease type C (NPC) is a rare autosomal-recessive lysosomal storage disease that is also associated with progressive neurodegeneration. NPC shares many pathological features with Alzheimer's disease, including neurofibrillary tangles, axonal spheroids, β-amyloid deposition, and dystrophic neurites. Here, we examined if these pathological features could be detected in induced pluripotent stem cell (iPSC)-derived neurons from NPC patients.
methodsBrain tissues from 8 NPC patients and 5 controls were analyzed for histopathological and biochemical markers of pathology. To model disease in culture, iPSCs from NPC patients and controls were differentiated into cortical neurons.
resultsWe found hyperphosphorylated tau, altered processing of amyloid precursor protein, and increased Aβ42 in NPC postmortem brains and in iPSC-derived cortical neurons from NPC patients.
conclusionOur findings demonstrated that the main pathogenic phenotypes typically found in NPC brains were also observed in patient-derived neurons, providing a useful model for further mechanistic and therapeutic studies of NPC. © 2021 International Parkinson and Movement Disorder Society.
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