Human stem cell models of polyglutamine diseases: Sources for disease models and cell therapy

He, Lang; Zhao, Chen; Peng, Linliu; Tang, Beisha; Jiang, Hong

doi:10.1016/j.expneurol.2020.113573

Cited by 7 publications

(5 citation statements)

References 186 publications

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“…Spinocerebellar ataxias (SCA) are a group of hereditary disorders impacting the nervous system that lead to progressive loss of coordination and balance control, and eventually, affect the individuals' cognitive functions (104).Currently, there is no cure for Spinocerebellar ataxias (SCA), and the current management strategies only focus on alleviating the symptoms (105).However, there is hope for the development of Neural Stem Cell Therapy as a potential treatment for Spinocerebellar ataxias (SCA) (106).…”

Section: Neural Stem Cell Therapy For Spinocerebellar Ataxiamentioning

confidence: 99%

Neural Stem Cell Therapies: Promising Treatments for Neurodegenerative Diseases

Gholamzad,

Sadeghi,

Azizabadi Farahani

et al. 2023

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Neurodegenerative diseases are a group of disorders that progressively damage the nervous system, leading to loss of function and disability. Currently, there are limited treatment options available for patients suffering from these disorders. However, Neural Stem Cell Therapy is a promising new approach that has shown great potential in treating various neurodegenerative diseases such as Alzheimer's disease, Parkinson's disease, Huntington's disease, multiple sclerosis, Amyotrophic Lateral Sclerosis, spinocerebellar ataxia, frontotemporal dementia, Creutzfeldt-Jakob disease, and Lewy body disease. Through extensive research and clinical trials, Neural Stem Cell Therapy has been shown to reduce inflammation, enhance neuronal regeneration, and promote functional recovery in various neurological disorders. In Alzheimer's disease, Neural Stem Cell Therapy has been shown to improve cognitive function and reduce amyloid plaques. In Parkinson's disease, Neural Stem Cells have been shown to differentiate into dopaminergic neurons and improve motor function. In multiple sclerosis, Neural Stem Cells have been shown to remyelinate damaged axons and reduce inflammation.Although more research is still needed, the ongoing progress in this field holds significant implications for developing new treatment options for patients with neurodegenerative disorders. Neural Stem Cell Therapy has the potential to offer new hope to millions of individuals around the world who suffer from these debilitating diseases. As the field of Neural Stem Cell Therapy continues to evolve, it is hoped that this promising new approach will ultimately lead to better outcomes and improved quality of life for patients with neurodegenerative diseases.

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Section: Neural Stem Cell Therapy For Spinocerebellar Ataxiamentioning

confidence: 99%

Neural Stem Cell Therapies: Promising Treatments for Neurodegenerative Diseases

Gholamzad,

Sadeghi,

Azizabadi Farahani

et al. 2023

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“…Park et al were the first to generate the iPSCs model of polyQ disease by reprograming fibroblasts from HD patients [122]. Until now, all polyQ diseases except for SCA17 have been modeled in iPSC lines [6,19,154]. However, no phenotypic studies have been carried out yet for DRPLA, SCA1 and SCA7 iPSCs models [6].…”

Section: Induced Pluripotent Stem Cellsmentioning

confidence: 99%

“…Until now, all polyQ diseases except for SCA17 have been modeled in iPSC lines [6,19,154]. However, no phenotypic studies have been carried out yet for DRPLA, SCA1 and SCA7 iPSCs models [6].…”

Section: Induced Pluripotent Stem Cellsmentioning

confidence: 99%

“…It remains under discussion whether the abnormal protein structure leads to neurodegeneration by the toxic gain of function, by the loss of function or by a combination of these two mechanisms [4,5]. Recently numerous studies have proven that the gain of function contributes to the selfassembly of the protein into toxic aggregates and the generation of neuronal intranuclear inclusions (NIIs), leading to neuronal death [6][7][8][9][10][11][12][13]. However, the neurodevelopmental significance of mutated genes supports the loss-of-function theory as the altered protein products may contribute to improper neural functioning [5].…”

Section: Introductionmentioning

confidence: 99%

“…Despite having similar features, polyQ diseases differ regarding their altered genes and protein products, the number of pathogenic CAG repeats, the preferentially degenerated neuronal subpopulations and the affected brain regions [1,2,6,19] (summarized in Table 1). Cerebellum, cortex, substantia nigra, striatum [38,39] Modern, nuclease-based gene-editing methods such as meganucleases, zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs) and clustered, regularly interspaced, short palindromic repeats (CRISPR)-Cas9 technology have significantly contributed to advances in disease modeling and augmented a variety of scientific research.…”

Section: Introductionmentioning

confidence: 99%

See 2 more Smart Citations

Advances in Modeling Polyglutamine Diseases Using Genome Editing Tools

Karwacka

Olejniczak

2022

Cells

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Polyglutamine (polyQ) diseases, including Huntington’s disease, are a group of late-onset progressive neurological disorders caused by CAG repeat expansions. Although recently, many studies have investigated the pathological features and development of polyQ diseases, many questions remain unanswered. The advancement of new gene-editing technologies, especially the CRISPR-Cas9 technique, has undeniable value for the generation of relevant polyQ models, which substantially support the research process. Here, we review how these tools have been used to correct disease-causing mutations or create isogenic cell lines with different numbers of CAG repeats. We characterize various cellular models such as HEK 293 cells, patient-derived fibroblasts, human embryonic stem cells (hESCs), induced pluripotent stem cells (iPSCs) and animal models generated with the use of genome-editing technology.

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