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Recent Overview of the Use of iPSCs Huntington's Disease Modeling and Therapy
Maria Csobonyeiova1, Stefan Polak1, Lubos Danisovic2,3
1Institute of Histology and Embryology, Faculty of Medicine, Comenius University, Sasinkova 4, 811 08 Bratislava, Slovakia.
Insights
Huntington's disease (HD) research explores stem-cell therapy using induced pluripotent stem cells (iPSCs). These patient-specific cells offer a promising avenue for understanding and potentially treating this neurodegenerative disorder.
Area of Science:
- Neuroscience
- Genetics
- Stem Cell Biology
Background:
- Huntington's disease (HD) is a fatal, inherited neurodegenerative disorder caused by CAG repeat expansion in the huntingtin gene.
- Current treatments for HD are largely symptomatic and ineffective in halting disease progression.
- Stem-cell-based therapies show promise in preclinical studies for neurodegenerative diseases.
Purpose of the Study:
- To review current Huntington's disease models.
- To discuss methods for differentiating patient-derived induced pluripotent stem cells (iPSCs) into neural cells for HD research.
- To explore advancements in gene editing for stem cell-based Huntington's disease therapy.
Main Methods:
- Review of existing Huntington's disease models.
- Analysis of protocols for differentiating induced pluripotent stem cells (iPSCs) into neural cell types.
- Examination of gene editing techniques applicable to stem cell therapy for HD.
Main Results:
- Induced pluripotent stem cells (iPSCs) provide a patient-specific, renewable source for neural cell generation.
- Differentiated iPSC-derived neural cells can form 3D organoids, modeling brain architecture.
- Progress in gene editing offers potential for correcting the genetic defect in HD.
Conclusions:
- Stem cell therapy, particularly using patient-specific induced pluripotent stem cells (iPSCs), holds significant therapeutic potential for Huntington's disease.
- iPSC-derived neural cells and organoids are valuable tools for studying HD pathogenesis.
- Advancements in gene editing complement stem cell approaches for future Huntington's disease treatments.
Abstract:
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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