A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1

A human iPSC-derived inducible neuronal model of Niemann-Pick disease, type C1

Abstract Background Niemann-Pick disease, type C (NPC) is a childhood-onset, lethal, neurodegenerative disorder caused by autosomal recessive mutations in the genes NPC1 or NPC2 and characterized by impaired cholesterol homeostasis, a lipid essential for cellular function. Cellular cholesterol level...

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Bibliographic Details
Main Authors: Anika V. Prabhu, Insung Kang, Raffaella De Pace, Christopher A. Wassif, Hideji Fujiwara, Pamela Kell, Xuntian Jiang, Daniel S. Ory, Juan S. Bonifacino, Michael E. Ward, Forbes D. Porter
Format: Article
Language:English
Published: BMC 2021-10-01
Series:BMC Biology
Subjects:
Human induced pluripotent stem cells
Human neurons
Niemann-Pick disease, type C1
Neurodegeneration
NPC1
Lysosomal disease
Online Access:https://doi.org/10.1186/s12915-021-01133-x

Internet

https://doi.org/10.1186/s12915-021-01133-x

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