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Review

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

1
Institute of Histology and Embryology, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia
2
Institute of Medical Biology, Genetics and Clinical Genetics, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia
3
Regenmed Ltd., Medena 29, 811 01 Bratislava, Slovakia
*
Author to whom correspondence should be addressed.
Int. J. Mol. Sci. 2020, 21(6), 2239; https://doi.org/10.3390/ijms21062239
Received: 4 March 2020 / Revised: 18 March 2020 / Accepted: 22 March 2020 / Published: 24 March 2020
(This article belongs to the Special Issue Disease Modeling Using Human Induced Pluripotent Stem Cells 2.0)
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. View Full-Text
Keywords: induced pluripotent stem cells; Huntington’s disease; disease modeling; regeneration induced pluripotent stem cells; Huntington’s disease; disease modeling; regeneration
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MDPI and ACS Style

Csobonyeiova, M.; Polak, S.; Danisovic, L. Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy. Int. J. Mol. Sci. 2020, 21, 2239. https://doi.org/10.3390/ijms21062239

AMA Style

Csobonyeiova M, Polak S, Danisovic L. Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy. International Journal of Molecular Sciences. 2020; 21(6):2239. https://doi.org/10.3390/ijms21062239

Chicago/Turabian Style

Csobonyeiova, Maria, Stefan Polak, and Lubos Danisovic. 2020. "Recent Overview of the Use of iPSCs Huntington’s Disease Modeling and Therapy" International Journal of Molecular Sciences 21, no. 6: 2239. https://doi.org/10.3390/ijms21062239

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