iPSC-based drug screening for Huntington's disease.

Autor: Zhang N; Buck Institute for Research on Aging, 8001 Redwood Blvd, Novato, CA 94945, United States., Bailus BJ; Buck Institute for Research on Aging, 8001 Redwood Blvd, Novato, CA 94945, United States., Ring KL; Buck Institute for Research on Aging, 8001 Redwood Blvd, Novato, CA 94945, United States., Ellerby LM; Buck Institute for Research on Aging, 8001 Redwood Blvd, Novato, CA 94945, United States. Electronic address: lellerby@buckinstitute.org.
Jazyk: angličtina
Zdroj: Brain research [Brain Res] 2016 May 01; Vol. 1638 (Pt A), pp. 42-56. Date of Electronic Publication: 2015 Sep 30.
DOI: 10.1016/j.brainres.2015.09.020
Abstrakt: Huntington's disease (HD) is an autosomal dominant neurodegenerative disorder, caused by an expansion of the CAG repeat in exon 1 of the huntingtin gene. The disease generally manifests in middle age with both physical and mental symptoms. There are no effective treatments or cures and death usually occurs 10-20 years after initial symptoms. Since the original identification of the Huntington disease associated gene, in 1993, a variety of models have been created and used to advance our understanding of HD. The most recent advances have utilized stem cell models derived from HD-patient induced pluripotent stem cells (iPSCs) offering a variety of screening and model options that were not previously available. The discovery and advancement of technology to make human iPSCs has allowed for a more thorough characterization of human HD on a cellular and developmental level. The interaction between the genome editing and the stem cell fields promises to further expand the variety of HD cellular models available for researchers. In this review, we will discuss the history of Huntington's disease models, common screening assays, currently available models and future directions for modeling HD using iPSCs-derived from HD patients. This article is part of a Special Issue entitled SI: PSC and the brain.
(Copyright © 2015 Elsevier B.V. All rights reserved.)
Databáze: MEDLINE
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