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Induced pluripotent stem cells derived from the developing striatum as a potential donor source for cell replacement therapy for Huntington disease

Choompoo, Narawadee, Bartley, Oliver J. M., Precious, Sophie V., Vinh, Ngoc-nga, Schnell, Christian, Garcia, Ana, Roberton, Victoria H., Williams, Nigel M., Kemp, Paul J., Kelly, Claire M. and Rosser, Anne E. 2021. Induced pluripotent stem cells derived from the developing striatum as a potential donor source for cell replacement therapy for Huntington disease. Cytotherapy 23 (2) , pp. 111-118. 10.1016/j.jcyt.2020.06.001

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Abstract

Background Cell replacement therapy (CRT) for Huntington disease (HD) requires a source of striatal (STR) progenitors capable of restoring the function lost due to STR degeneration. Authentic STR progenitors can be collected from the fetal putative striatum, or whole ganglionic eminence (WGE), but these tissues remain impractical for widespread clinical application, and alternative donor sources are required. Here we begin exploring the possibility that induced pluripotent stem cells (iPSC) derived from WGE may retain an epigenetic memory of their tissue of origin, which could enhance their ability to differentiate into STR cells. Results We generate four iPSC lines from human WGE (hWGE) and establish that they have a capacity similar to human embryonic stem cells with regard to their ability to differentiate toward an STR phenotype, as measured by expression and demethylation of key STR genes, while maintaining an overall different methylome. Finally, we demonstrate that these STR-differentiated hWGE iPSCs share characteristics with hWGE (i.e., authentic STR tissues) both in vitro and following transplantation into an HD model. Overall, iPSCs derived from human WGE show promise as a donor source for CRT for HD.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Medicine
Biosciences
Additional Information: This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
Publisher: Elsevier
ISSN: 1465-3249
Funders: Wellcome Trust
Date of First Compliant Deposit: 13 January 2021
Date of Acceptance: 16 June 2020
Last Modified: 08 Sep 2021 01:14
URI: http://orca.cardiff.ac.uk/id/eprint/137641

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