Journal
STEM CELL REPORTS
Volume 3, Issue 4, Pages 585-593Publisher
CELL PRESS
DOI: 10.1016/j.stemcr.2014.07.011
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Funding
- Office of Research and Infrastructure Program (ORIP)/OD [P51OD11132]
- Royal Golden Jubilee PhD program of Thailand Research Fund
- NIH [5T32MH879774]
- ORIP/NIH [OD010930]
- NINDS/NIH [NS084163]
- ACTSI
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Huntington's disease (HD) is a dominant neurodegenerative disorder caused by the expansion of glutamine residues in the N-terminal region of the huntingtin (HTT) protein. The disease results in progressive neuronal loss, leading to motor, cognitive, and psychiatric impairment. Here, we report the establishment of neural progenitor cell (NPC) lines derived from induced pluripotent stem cells (iPSCs) of transgenic HD monkeys. Upon differentiation to neurons, HD neural cells develop cellular features of HD, including the formation of nuclear inclusions and oligomeric mutant HTT (mHTT) aggregates, as well as increased apoptosis. These phenotypes are rescued by genetic suppression of HTT and pharmacological treatment, demonstrating the ability of our HD cell model to respond to therapeutic treatment. The development and reversal of HD-associated phenotypes in neural cells from HD monkeys provides a unique nonhuman primate (NHP) model for exploring HD pathogenesis and evaluating therapeutics that could be assessed further in HD monkeys.
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