Niemann-Pick disease type C: induced pluripotent stem cell-derived neuronal cells for modeling neural disease and evaluating drug efficacy

Niemann-Pick disease type C (NPC) is a rare neurodegenerative disorder caused by recessive mutations in NPC1 or NPC2 gene that result in lysosomal accumulation of unesterified cholesterol in patient cells. Patient fibroblasts have been used for evaluation of compound efficacy although neuronal degen...

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Gepubliceerd in:J Biomol Screen
Hoofdauteurs: Yu, Daozhan, Swaroop, Manju, Wang, Mengqiao, Baxa, Ulrich, Yang, Rongze, Yan, Yiping, Coksaygan, Turhan, DeTolla, Louis, Marugan, Juan J., Austin, Christopher P., McKew, John C., Gong, Da-Wei, Zheng, Wei
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: 2014
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Article
Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4529815/
https://ncbi.nlm.nih.gov/pubmed/24907126
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1177/1087057114537378
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