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Evaluation of drug efficacy in a cell based Niemann Pick type C disease model using cells derived from patient iPS cells

Wednesday, September 24, 2014 — Poster Session IV

10:00 a.m. –12:00 p.m.

FAES Academic Center

NCATS

STEMCELL-2

* FARE Award Winner

Authors

  • Y. Long
  • R. Li
  • M. Wang
  • S. Dai
  • M. Swaroop
  • J.C. McKew
  • W. Zheng

Abstract

Many genetic diseases do not have an appropriate disease model for evaluation of drug efficacy for drug development. We have developed a cell based Niemann Pick type C disease model using the patient iPSC differentiated neuronal cells. This model exhibits the cholesterol accumulation phenotype in lysosomes of patient neuronal cells. We have evaluated several known drugs which have been previously reported to have benefits in Niemann Pick type C patients. The results demonstrate this disease model is useful for drug development to treat the Niemann Pick type C disease.

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