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Modeling Niemann Pick type C1 using human embryonic and induced pluripotent stem cells

M Paulina Ordoñez1, John W Steele2

  • 1Sanford Consortium for Regenerative Medicine, University of California, San Diego, La Jolla, CA 92037, United States; Department of Pediatric Gastroenterology, Hepatology, and Nutrition, University of California, San Diego, La Jolla, CA 92037, United States.

Brain Research
|March 15, 2016
PubMed
Summary

Niemann Pick type C1 (NPC1) disease shows neuronal defects in human stem cell-derived neurons. These models are crucial for discovering drugs to treat NPC1, complementing animal studies.

Keywords:
AutophagyDrug screeningHuman embryonic stem cellsHuman induced pluripotent stem cellsModels of diseaseNeurodegenerationNiemann Pick type C1

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