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J Genet Genomics. 2012 Jun 20;39(6):253-9. doi: 10.1016/j.jgg.2012.04.006. Epub 2012 May 9.

Pluripotent stem cells models for Huntington's disease: prospects and challenges.

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  • 1Yerkes National Primate Research Center, 954 Gatewood Rd., N.E. Atlanta, GA 39329, USA.


Pluripotent cellular models have shown great promise in the study of a number of neurological disorders. Several advantages of using a stem cell model include the potential for cells to derive disease relevant neuronal cell types, providing a system for researchers to monitor disease progression during neurogenesis, along with serving as a platform for drug discovery. A number of stem cell derived models have been employed to establish in vitro research models of Huntington's disease that can be used to investigate cellular pathology and screen for drug and cell-based therapies. Although some progress has been made, there are a number of challenges and limitations that must be overcome before the true potential of this research strategy is achieved. In this article we review current stem cell models that have been reported, as well as discuss the issues that impair these studies. We also highlight the prospective application of Huntington's disease stem cell models in the development of novel therapeutic strategies and advancement of personalized medicine.

Copyright © 2012. Published by Elsevier Ltd.

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