The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine

In recent years several genes have linked amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) as a spectrum disease; however little is known about what triggers their onset. With the ability to generate patient specific stem cell lines from somatic cells, it is possible to model di...

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Main Authors: Erin C. Hedges, Vera J. Mehler, Agnes L. Nishimura
Format: Article
Language:English
Published: Wiley 2016-01-01
Series:Stem Cells International
Online Access:http://dx.doi.org/10.1155/2016/9279516
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author Erin C. Hedges
Vera J. Mehler
Agnes L. Nishimura
author_facet Erin C. Hedges
Vera J. Mehler
Agnes L. Nishimura
author_sort Erin C. Hedges
collection DOAJ
description In recent years several genes have linked amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) as a spectrum disease; however little is known about what triggers their onset. With the ability to generate patient specific stem cell lines from somatic cells, it is possible to model disease without the need to transfect cells with exogenous DNA. These pluripotent stem cells have opened new avenues for identification of disease phenotypes and their relation to specific molecular pathways. Thus, as never before, compounds with potential applications for regenerative medicine can be specifically tailored in patient derived cultures. In this review, we discuss how patient specific induced pluripotent stem cells (iPSCs) have been used to model ALS and FTD and the most recent drug screening targets for these diseases. We also discuss how an iPSC bank would improve the quality of the available cell lines and how it would increase knowledge about the ALS/FTD disease spectrum.
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spelling doaj-art-089417ad9bb54598bd05ce7d55ae258a2025-08-20T02:03:20ZengWileyStem Cells International1687-966X1687-96782016-01-01201610.1155/2016/92795169279516The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative MedicineErin C. Hedges0Vera J. Mehler1Agnes L. Nishimura2Department of Basic and Clinical Neuroscience, Institute of Psychiatry, Psychology and Neuroscience (IOPPN), King’s College London and King’s Health Partners, London SE5 9RX, UKDepartment of Basic and Clinical Neuroscience, Institute of Psychiatry, Psychology and Neuroscience (IOPPN), King’s College London and King’s Health Partners, London SE5 9RX, UKDepartment of Basic and Clinical Neuroscience, Institute of Psychiatry, Psychology and Neuroscience (IOPPN), King’s College London and King’s Health Partners, London SE5 9RX, UKIn recent years several genes have linked amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) as a spectrum disease; however little is known about what triggers their onset. With the ability to generate patient specific stem cell lines from somatic cells, it is possible to model disease without the need to transfect cells with exogenous DNA. These pluripotent stem cells have opened new avenues for identification of disease phenotypes and their relation to specific molecular pathways. Thus, as never before, compounds with potential applications for regenerative medicine can be specifically tailored in patient derived cultures. In this review, we discuss how patient specific induced pluripotent stem cells (iPSCs) have been used to model ALS and FTD and the most recent drug screening targets for these diseases. We also discuss how an iPSC bank would improve the quality of the available cell lines and how it would increase knowledge about the ALS/FTD disease spectrum.http://dx.doi.org/10.1155/2016/9279516
spellingShingle Erin C. Hedges
Vera J. Mehler
Agnes L. Nishimura
The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
Stem Cells International
title The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
title_full The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
title_fullStr The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
title_full_unstemmed The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
title_short The Use of Stem Cells to Model Amyotrophic Lateral Sclerosis and Frontotemporal Dementia: From Basic Research to Regenerative Medicine
title_sort use of stem cells to model amyotrophic lateral sclerosis and frontotemporal dementia from basic research to regenerative medicine
url http://dx.doi.org/10.1155/2016/9279516
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