Department of Neurology, Seoul National University Hospital, Seoul, Korea.
J Mov Disord. 2014 Apr;7(1):1-6. doi: 10.14802/jmd.14001. Epub 2014 Apr 30.
Huntington's disease (HD) is a genetic neurodegenerative disorder. The most common symptom of HD is abnormal involuntary writhing movements, called chorea. Antipsychotics and tetrabenazine are used to alleviate the signs and symptoms of HD. Stem cells have been investigated for use in neurodegenerative disorders to develop cell therapy strategies. Recent evidence indicates that the beneficial effects of stem cell therapies are actually mediated by secretory molecules, as well as cell replacement. Although stem cell studies show that cell transplantation provides cellular improvement around lesions in in vivo models, further work is required to elucidate some issues before the clinical application of stem cells. These issues include the precise mechanism of action, delivery method, toxicity and safety. With a focus on HD, this review summarizes cell therapy strategies and the paracrine effect of stem cells.
亨廷顿病(HD)是一种遗传性神经退行性疾病。HD 的最常见症状是异常的不自主扭动运动,称为舞蹈症。抗精神病药和四苯嗪用于缓解 HD 的体征和症状。干细胞已被用于神经退行性疾病,以开发细胞治疗策略。最近的证据表明,干细胞治疗的有益效果实际上是由分泌分子以及细胞替代介导的。尽管干细胞研究表明细胞移植可提供体内模型中病变周围的细胞改善,但在干细胞的临床应用之前,还需要进一步研究来阐明一些问题。这些问题包括确切的作用机制、递送方法、毒性和安全性。本文重点介绍 HD,总结了细胞治疗策略和干细胞的旁分泌作用。