Choi Kyung-Ah, Hong Sunghoi
a School of Biosystem and Biomedical Science , College of Health Science, Korea University , Seongbuk-gu , Republic of Korea.
b Department of Integrated Biomedical and Life Science , College of Health Science, Korea University , Seongbuk-gu , Republic of Korea.
Expert Opin Biol Ther. 2017 Nov;17(11):1333-1343. doi: 10.1080/14712598.2017.1365133. Epub 2017 Aug 9.
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.
亨廷顿舞蹈症（HD）是一种遗传性神经退行性疾病，其特征为舞蹈症、痴呆和抑郁，由进行性神经细胞退化引起，而这种退化是由亨廷顿蛋白（Htt）基因中CAG重复序列的扩增所触发。目前，这种疾病无法治愈，也没有有效的药物可以延缓或改善由其导致的身体、精神和行为方面的严重程度。涵盖领域：在本综述中，作者描述了通过直接转化技术使用诱导神经干细胞（iNSCs），作为一种治疗HD的治疗性细胞类型，其具有很大优势。专家观点：将体细胞转化为所需的干细胞类型是HD最有前景的治疗方法之一，因为它有助于生成患者特异性神经干细胞。诱导多能干细胞（iPSCs）具有分化为神经元的强大潜力，但移植后由于未分化的多能干细胞可能会导致畸胎瘤形成。因此，将体细胞直接转化为iNSCs是再生医学中一种有前景的替代技术，iNSCs可能作为亨廷顿舞蹈症的治疗性细胞来源。
