September 22-26, 2014
Building 10
- General Schedule of Events
- Opening Plenary Session
- Concurrent Symposia Sessions
- Poster Sessions
- FARE Award Ceremony
- Special Exhibits on Resources for Intramural Research
- TSA Research Festival Exhibit Show
- Clinical Center Tours
- Research Festival Committees
- Past Research Festivals
2014 program
Download the 2014 Research Festival Schedule Overview (6 pages)
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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.
