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- W1833136679 endingPage "56" @default.
- W1833136679 startingPage "42" @default.
- W1833136679 abstract "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." @default.
- W1833136679 created "2016-06-24" @default.
- W1833136679 creator A5010726629 @default.
- W1833136679 creator A5043919479 @default.
- W1833136679 creator A5068708465 @default.
- W1833136679 creator A5083824053 @default.
- W1833136679 date "2016-05-01" @default.
- W1833136679 modified "2023-10-10" @default.
- W1833136679 title "iPSC-based drug screening for Huntington׳s disease" @default.
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