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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, N; Bartley, OJM; Precious, SV; Vinh, NN; Schnell, C; Garcia, A; Roberton, VH; ... Rosser, AE; + view all (2020) Induced pluripotent stem cells derived from the developing striatum as a potential donor source for cell replacement therapy for Huntington disease. Cytotherapy 10.1016/j.jcyt.2020.06.001. (In press). Green open access

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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.

Type: Article
Title: Induced pluripotent stem cells derived from the developing striatum as a potential donor source for cell replacement therapy for Huntington disease
Open access status: An open access version is available from UCL Discovery
DOI: 10.1016/j.jcyt.2020.06.001
Publisher version: https://doi.org/10.1016/j.jcyt.2020.06.001
Language: English
Additional information: Copyright © 2020 International Society for Cell & Gene Therapy. This is an open access article under the CC BY license (http://creativecommons.org/licenses/by/4.0/)
Keywords: cell therapy, epigenetic memory, fetal tissue, Huntington disease, iPSC, striatal differentiation
UCL classification: UCL
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Life Sciences > UCL School of Pharmacy > Pharmacology
URI: https://discovery.ucl.ac.uk/id/eprint/10117421
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