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Using a neuron and astrocyte co-culture system to model cell interactions in Niemann-Pick type C

Friday, November 08, 2013 — Poster Session III

10:00 a.m. – 12:00 p.m.

FAES Academic Center (Upper-Level Terrace)




  • A Efthymiou
  • MS Rao
  • NS Malik


Niemann-Pick type C (NP-C) is a neurodegenerative disorder that affects approximately 1 in 150,000 people. It is categorized as an inborn error of lipid metabolism, and is characterized by an accumulation of cholesterol in lysosomes. Currently, there is currently no cure or disease-modifying treatment available for NP-C. Patient-derived induced pluripotent stem cells (iPSCs) are a unique source for modeling disease states in vitro. Due to their unlimited differentiation potential, iPSCs derived from a patient with NP-C can be differentiated into affected cell types, such as neurons and astrocytes. We have generated an NP-C specific iPSC line with a mutation in NPC1 at I1061T. Neural stem cells (NSC) were subsequently generated from this line. The NSCs were further differentiated into functional neurons and astrocytes, as characterized by morphological, molecular, and functional criteria. Microarray data obtained from NP-C NSCs and neurons suggest that these cells have a bias toward glial fates. We aim to use these cells in a neuron and astrocyte co-culture with healthy and affected cell types in order to model cell interactions in Niemann-Pick type C and uncover novel information about the nature of this disease to determine possible treatments.

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