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One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders

Studying Parkinson’s disease (PD) in the laboratory presents many challenges, the main one being the limited availability of human cells and tissue from affected individuals. As PD is characterized by a loss of dopaminergic (DA) neurons in the brain, it is nearly impossible for researchers to access...

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Autores principales: Mohamed, Nguyen-Vi, Larroquette, Frédérique, Beitel, Lenore K., Fon, Edward A., Durcan, Thomas M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: IOS Press 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6597965/
https://www.ncbi.nlm.nih.gov/pubmed/30741685
http://dx.doi.org/10.3233/JPD-181515
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author Mohamed, Nguyen-Vi
Larroquette, Frédérique
Beitel, Lenore K.
Fon, Edward A.
Durcan, Thomas M.
author_facet Mohamed, Nguyen-Vi
Larroquette, Frédérique
Beitel, Lenore K.
Fon, Edward A.
Durcan, Thomas M.
author_sort Mohamed, Nguyen-Vi
collection PubMed
description Studying Parkinson’s disease (PD) in the laboratory presents many challenges, the main one being the limited availability of human cells and tissue from affected individuals. As PD is characterized by a loss of dopaminergic (DA) neurons in the brain, it is nearly impossible for researchers to access and extract these cells from living patients. Thus, in the past PD research has focused on the use of patients’ post-mortem tissues, animal models, or immortalized cell lines to dissect cellular pathways of interest. While these strategies deepened our knowledge of pathological mechanisms in PD, they failed to faithfully capture key mechanisms at play in the human brain. The emergence of induced pluripotent stem cell (iPSC) technology is revolutionizing PD research, as it allows for the differentiation and growth of human DA neurons in vitro, holding immense potential not only for modelling PD, but also for identifying novel therapies. However, to reproduce the complexity of the brain’s environment, researchers are recognizing the need to further develop and refine iPSC-based tools. In this review, we provide an overview of different systems now available for the study of PD, with a particular emphasis on the potential and limitations of iPSC as research tools to generate more relevant models of PD pathophysiology and advance the drug discovery process.
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spelling pubmed-65979652019-07-01 One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders Mohamed, Nguyen-Vi Larroquette, Frédérique Beitel, Lenore K. Fon, Edward A. Durcan, Thomas M. J Parkinsons Dis Review Studying Parkinson’s disease (PD) in the laboratory presents many challenges, the main one being the limited availability of human cells and tissue from affected individuals. As PD is characterized by a loss of dopaminergic (DA) neurons in the brain, it is nearly impossible for researchers to access and extract these cells from living patients. Thus, in the past PD research has focused on the use of patients’ post-mortem tissues, animal models, or immortalized cell lines to dissect cellular pathways of interest. While these strategies deepened our knowledge of pathological mechanisms in PD, they failed to faithfully capture key mechanisms at play in the human brain. The emergence of induced pluripotent stem cell (iPSC) technology is revolutionizing PD research, as it allows for the differentiation and growth of human DA neurons in vitro, holding immense potential not only for modelling PD, but also for identifying novel therapies. However, to reproduce the complexity of the brain’s environment, researchers are recognizing the need to further develop and refine iPSC-based tools. In this review, we provide an overview of different systems now available for the study of PD, with a particular emphasis on the potential and limitations of iPSC as research tools to generate more relevant models of PD pathophysiology and advance the drug discovery process. IOS Press 2019-05-23 /pmc/articles/PMC6597965/ /pubmed/30741685 http://dx.doi.org/10.3233/JPD-181515 Text en © 2019 – IOS Press and the authors. All rights reserved https://creativecommons.org/licenses/by-nc/4.0/ This is an open access article distributed under the terms of the Creative Commons Attribution Non-Commercial (CC BY-NC 4.0) License (https://creativecommons.org/licenses/by-nc/4.0/) , which permits unrestricted non-commercial use, distribution, and reproduction in any medium, provided the original work is properly cited.
spellingShingle Review
Mohamed, Nguyen-Vi
Larroquette, Frédérique
Beitel, Lenore K.
Fon, Edward A.
Durcan, Thomas M.
One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title_full One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title_fullStr One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title_full_unstemmed One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title_short One Step Into the Future: New iPSC Tools to Advance Research in Parkinson’s Disease and Neurological Disorders
title_sort one step into the future: new ipsc tools to advance research in parkinson’s disease and neurological disorders
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6597965/
https://www.ncbi.nlm.nih.gov/pubmed/30741685
http://dx.doi.org/10.3233/JPD-181515
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