Induced Pluripotent Stem Cells in Huntington's Disease: Disease Modeling and the Potential for Cell-Based Therapy.
Induced Pluripotent Stem Cells in Huntington's Disease: Disease Modeling and the Potential for Cell-Based Therapy.
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亨廷顿病的诱导多能干细胞：疾病模型和细胞治疗的潜力
DOI：
10.1007/s12035-015-9601-8
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发表时间：
2016-12
影响因子：
5.1
通讯作者：
Wang T
中科院分区：
文献类型：
--
作者：
Liu L;Huang JS;Han C;Zhang GX;Xu XY;Shen Y;Li J;Jiang HY;Lin ZC;Xiong N;Wang T
亨廷顿氏病（HD）是一种无法治愈的神经退行性疾病，其特征是运动功能障碍、认知障碍和行为异常。它是一种常染色体显性遗传疾病，由亨廷顿基因中的CAG重复扩增引起，导致主要在纹状体和皮质中的进行性神经元损失。尽管在1993年发现了致病基因，但HD发病机制的确切机制尚未阐明。目前还没有减缓或停止疾病进程的治疗方法。诱导多能干细胞（iPSC）技术的最新进展已经改变了我们研究人类神经细胞疾病的能力。在这里，我们首先回顾了使用患者来源的iPSCs体外模拟HD的进展，这些进展揭示了阐明分子机制的独特见解，并为药物发现提供了一种新的基于人类细胞的平台。然后，我们强调了多能干细胞的承诺和挑战，可能被用作细胞替代疗法在HD大脑中丢失的神经元的治疗来源。
Huntington’s disease (HD) is an incurable neurodegenerative disorder that is characterized by motor dysfunction, cognitive impairment, and behavioral abnormalities. It is an autosomal dominant disorder caused by a CAG repeat expansion in the huntingtin gene, resulting in progressive neuronal loss predominately in the striatum and cortex. Despite the discovery of the causative gene in 1993, the exact mechanisms underlying HD pathogenesis have yet to be elucidated. Treatments that slow or halt the disease process are currently unavailable. Recent advances in induced pluripotent stem cell (iPSC) technologies have transformed our ability to study disease in human neural cells. Here, we firstly review the progress made to model HD in vitro using patient-derived iPSCs, which reveal unique insights into illuminating molecular mechanisms and provide a novel human cell-based platform for drug discovery. We then highlight the promises and challenges for pluripotent stem cells that might be used as a therapeutic source for cell replacement therapy of the lost neurons in HD brains.
影响因子：
3.9
作者：
Im W;Kim M
通讯作者：
Kim M