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The translational potential of human induced pluripotent stem cells for clinical neurology: The translational potential of hiPSCs in neurology

Devine, H; Patani, R; (2016) The translational potential of human induced pluripotent stem cells for clinical neurology: The translational potential of hiPSCs in neurology. Cell Biology and Toxicology , 33 (2) pp. 129-144. 10.1007/s10565-016-9372-7. Green open access

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Abstract

The induced pluripotent state represents a decade-old Nobel prize-winning discovery. Human-induced pluripotent stem cells (hiPSCs) are generated by the nuclear reprogramming of any somatic cell using a variety of established but evolving methods. This approach offers medical science unparalleled experimental opportunity to model an individual patient's disease "in a dish." HiPSCs permit developmentally rationalized directed differentiation into any cell type, which express donor cell mutation(s) at pathophysiological levels and thus hold considerable potential for disease modeling, drug discovery, and potentially cell-based therapies. This review will focus on the translational potential of hiPSCs in clinical neurology and the importance of integrating this approach with complementary model systems to increase the translational yield of preclinical testing for the benefit of patients. This strategy is particularly important given the expected increase in prevalence of neurodegenerative disease, which poses a major burden to global health over the coming decades.

Type: Article
Title: The translational potential of human induced pluripotent stem cells for clinical neurology: The translational potential of hiPSCs in neurology
Location: Netherlands
Open access status: An open access version is available from UCL Discovery
DOI: 10.1007/s10565-016-9372-7
Publisher version: http://dx.doi.org/10.1007/s10565-016-9372-7
Language: English
Additional information: © The Author(s) 2016. This article is distributed under the terms of the Creative Commons Attribution 4.0 International License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided you give appropriate credit to the original author(s) and the source, provide a link to the Creative Commons license, and indicate if changes were made.
Keywords: Cellular therapy, Disease modeling, Drug discovery, Human induced pluripotent stem cells (hiPSCs), Neurology, Translational medicine
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 Brain Sciences
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology
UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases
URI: https://discovery.ucl.ac.uk/id/eprint/1532773
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