Intellectual and Developmental Disabilities Research Center, Semel Institute for Neuroscience and Human Behavior, University of California, Los Angeles, CA, USA.
Institute for Memory Impairment and Neurological Disorders, University of California, Irvine, CA, USA.
CNS Neurosci Ther. 2018 Apr;24(4):329-342. doi: 10.1111/cns.12839. Epub 2018 Mar 6.
The principal symptoms of Huntington's disease (HD), chorea, cognitive deficits, and psychiatric symptoms are associated with the massive loss of striatal and cortical projection neurons. As current drug therapies only partially alleviate symptoms, finding alternative treatments has become peremptory. Cell replacement using stem cells is a rapidly expanding field that offers such an alternative. In this review, we examine recent studies that use mesenchymal cells, as well as pluripotent, cell-derived products in animal models of HD. Additionally, we provide further electrophysiological characterization of a human neural stem cell line, ESI-017, which has already demonstrated disease-modifying properties in two mouse models of HD. Overall, the field of regenerative medicine represents a viable and promising avenue for the treatment of neurodegenerative disorders including HD.
亨廷顿病（HD）的主要症状包括舞蹈病、认知缺陷和精神症状，这些症状与纹状体和皮质投射神经元的大量丧失有关。由于目前的药物治疗只能部分缓解症状，因此寻找替代治疗方法已变得刻不容缓。使用干细胞进行细胞替代是一个迅速发展的领域，提供了这样一种替代方法。在这篇综述中，我们研究了最近使用间充质细胞以及多能细胞衍生产物在 HD 动物模型中的研究。此外，我们还对已在两种 HD 小鼠模型中显示出疾病修饰特性的人神经干细胞系 ESI-017 进行了进一步的电生理特性分析。总的来说，再生医学领域为包括 HD 在内的神经退行性疾病的治疗提供了一条可行而有前途的途径。
