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Induced neural stem cells as a means of treatment in Huntington's disease

机译:诱导神经干细胞作为亨廷顿疾病治疗手段

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Introduction: Huntington's disease (HD) is an inherited neurodegenerative disease characterized by chorea, dementia, and depression caused by progressive nerve cell degeneration, which is triggered by expanded CAG repeats in the huntingtin (Htt) gene. Currently, there is no cure for this disease, nor is there an effective medicine available to delay or improve the physical, mental, and behavioral severities caused by it.Areas covered: In this review, the authors describe the use of induced neural stem cells (iNSCs) by direct conversion technology, which offers great advantages as a therapeutic cell type to treat HD.Expert opinion: Cell conversion of somatic cells into a desired stem cell type is one of the most promising treatments for HD because it could be facilitated for the generation of patient-specific neural stem cells. The induced pluripotent stem cells (iPSCs) have a powerful potential for differentiation into neurons, but they may cause teratoma formation due to an undifferentiated pluripotent stem cell after transplantation Therefore, direct conversion of somatic cells into iNSCs is a promising alternative technology in regenerative medicine and the iNSCs may be provided as a therapeutic cell source for Huntington's disease.
机译:简介:亨廷顿的疾病(HD)是一种遗传性神经退行性疾病,其特征在于伴随着神经细胞变性引起的冠心膜,痴呆和抑郁症,其被亨廷顿(HTT)基因的扩张CAG重复引发。目前,这种疾病没有治愈,也没有有效的药物可用于延迟或改善它由IT引起的身体,精神和行为剧性。覆盖:在本次审查中,作者描述了诱导的神经干细胞的使用(INSCS)通过直接转换技术,作为治疗HD的治疗细胞类型提供了很大的优势患者特异性神经干细胞的产生。诱导的多能干细胞(IPSC)具有分化为神经元的强大潜力,但由于移植后未分化的多能干细胞,它们可能导致畸胎瘤形成,因此,体细胞进入INCES的直接转化是再生医学中的有希望的替代技术。 INSCS可以作为亨廷顿疾病的治疗细胞来源提供。

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