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Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy

Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative disease characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD is caused by an expansion in the number of CAG repeats in the huntingtin gene on chromosome 4. To date, no effe...

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Detalles Bibliográficos
Autores principales: Csobonyeiova, Maria, Polak, Stefan, Danisovic, Lubos
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7139425/
https://www.ncbi.nlm.nih.gov/pubmed/32213859
http://dx.doi.org/10.3390/ijms21062239
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author Csobonyeiova, Maria
Polak, Stefan
Danisovic, Lubos
author_facet Csobonyeiova, Maria
Polak, Stefan
Danisovic, Lubos
author_sort Csobonyeiova, Maria
collection PubMed
description Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative disease characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD is caused by an expansion in the number of CAG repeats in the huntingtin gene on chromosome 4. To date, no effective therapy for preventing the onset or progression of the disease has been found, and many symptoms do not respond to pharmacologic treatment. However, recent results of pre-clinical trials suggest a beneficial effect of stem-cell-based therapy. Induced pluripotent stem cells (iPSCs) represent an unlimited cell source and are the most suitable among the various types of autologous stem cells due to their patient specificity and ability to differentiate into a variety of cell types both in vitro and in vivo. Furthermore, the cultivation of iPSC-derived neural cells offers the possibility of studying the etiopathology of neurodegenerative diseases, such as HD. Moreover, differentiated neural cells can organize into three-dimensional (3D) organoids, mimicking the complex architecture of the brain. In this article, we present a comprehensive review of recent HD models, the methods for differentiating HD–iPSCs into the desired neural cell types, and the progress in gene editing techniques leading toward stem-cell-based therapy.
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spelling pubmed-71394252020-04-10 Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy Csobonyeiova, Maria Polak, Stefan Danisovic, Lubos Int J Mol Sci Review Huntington’s disease (HD) is an inherited, autosomal dominant, degenerative disease characterized by involuntary movements, cognitive decline, and behavioral impairment ending in death. HD is caused by an expansion in the number of CAG repeats in the huntingtin gene on chromosome 4. To date, no effective therapy for preventing the onset or progression of the disease has been found, and many symptoms do not respond to pharmacologic treatment. However, recent results of pre-clinical trials suggest a beneficial effect of stem-cell-based therapy. Induced pluripotent stem cells (iPSCs) represent an unlimited cell source and are the most suitable among the various types of autologous stem cells due to their patient specificity and ability to differentiate into a variety of cell types both in vitro and in vivo. Furthermore, the cultivation of iPSC-derived neural cells offers the possibility of studying the etiopathology of neurodegenerative diseases, such as HD. Moreover, differentiated neural cells can organize into three-dimensional (3D) organoids, mimicking the complex architecture of the brain. In this article, we present a comprehensive review of recent HD models, the methods for differentiating HD–iPSCs into the desired neural cell types, and the progress in gene editing techniques leading toward stem-cell-based therapy. MDPI 2020-03-24 /pmc/articles/PMC7139425/ /pubmed/32213859 http://dx.doi.org/10.3390/ijms21062239 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Csobonyeiova, Maria
Polak, Stefan
Danisovic, Lubos
Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title_full Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title_fullStr Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title_full_unstemmed Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title_short Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy
title_sort recent overview of the use of ipscs huntington’s disease modeling and therapy
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7139425/
https://www.ncbi.nlm.nih.gov/pubmed/32213859
http://dx.doi.org/10.3390/ijms21062239
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