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Induced pluripotent stem cells in the study of neurological diseases

Five years after their initial derivation from mouse somatic cells, induced pluripotent stem (iPS) cells are an important tool for the study of neurological diseases. By offering an unlimited source of patient-specific disease-relevant neuronal and glial cells, iPS cell-based disease models hold eno...

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Detalles Bibliográficos
Autores principales: Saporta, Mario A, Grskovic, Marica, Dimos, John T
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2011
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3308034/
https://www.ncbi.nlm.nih.gov/pubmed/21936964
http://dx.doi.org/10.1186/scrt78
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author Saporta, Mario A
Grskovic, Marica
Dimos, John T
author_facet Saporta, Mario A
Grskovic, Marica
Dimos, John T
author_sort Saporta, Mario A
collection PubMed
description Five years after their initial derivation from mouse somatic cells, induced pluripotent stem (iPS) cells are an important tool for the study of neurological diseases. By offering an unlimited source of patient-specific disease-relevant neuronal and glial cells, iPS cell-based disease models hold enormous promise for identification of disease mechanisms, discovery of molecular targets and development of phenotypic screens for drug discovery. The present review focuses on the recent advancements in modeling neurological disorders, including the demonstration of disease-specific phenotypes in iPS cell-derived neurons generated from patients with spinal muscular atrophy, familial dysautonomia, Rett syndrome, schizophrenia and Parkinson disease. The ability of this approach to detect treatment effects from known therapeutic compounds has also been demonstrated, providing proof of principle for the use of iPS cell-derived cells in drug discovery.
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spelling pubmed-33080342012-09-21 Induced pluripotent stem cells in the study of neurological diseases Saporta, Mario A Grskovic, Marica Dimos, John T Stem Cell Res Ther Review Five years after their initial derivation from mouse somatic cells, induced pluripotent stem (iPS) cells are an important tool for the study of neurological diseases. By offering an unlimited source of patient-specific disease-relevant neuronal and glial cells, iPS cell-based disease models hold enormous promise for identification of disease mechanisms, discovery of molecular targets and development of phenotypic screens for drug discovery. The present review focuses on the recent advancements in modeling neurological disorders, including the demonstration of disease-specific phenotypes in iPS cell-derived neurons generated from patients with spinal muscular atrophy, familial dysautonomia, Rett syndrome, schizophrenia and Parkinson disease. The ability of this approach to detect treatment effects from known therapeutic compounds has also been demonstrated, providing proof of principle for the use of iPS cell-derived cells in drug discovery. BioMed Central 2011-09-21 /pmc/articles/PMC3308034/ /pubmed/21936964 http://dx.doi.org/10.1186/scrt78 Text en Copyright ©2011 BioMed Central Ltd
spellingShingle Review
Saporta, Mario A
Grskovic, Marica
Dimos, John T
Induced pluripotent stem cells in the study of neurological diseases
title Induced pluripotent stem cells in the study of neurological diseases
title_full Induced pluripotent stem cells in the study of neurological diseases
title_fullStr Induced pluripotent stem cells in the study of neurological diseases
title_full_unstemmed Induced pluripotent stem cells in the study of neurological diseases
title_short Induced pluripotent stem cells in the study of neurological diseases
title_sort induced pluripotent stem cells in the study of neurological diseases
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3308034/
https://www.ncbi.nlm.nih.gov/pubmed/21936964
http://dx.doi.org/10.1186/scrt78
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