Huntington's disease (HD) is an autosomal-dominant neurodegenerative disorder encoding a mutant form of the huntingtin protein (HTT). HD is pathologically characterized by loss of neurons in the striatum and cortex, which leads to progressive motor dysfunction, cognitive decline and behavioral symptoms. Stem cell-based therapy has emerged as a feasible therapeutic approach for the treatment of neurodegenerative diseases and may be effective in alleviating and/or halting the pathophysiological mechanisms underlying HD. Several pre-clinical studies have used stem cells in animal models of HD. Here, we performed a systematic review of preclinical studies to estimate the treatment efficacy of stem cells in animal models of HD. Based on our systematic review, treatment with stem cells significantly improves neurological and behavioral outcomes in animal models of HD. Although promising results were found, the design of animal studies, the types of transplanted cells and the route of administration are poorly standardized and this greatly complicates comparative analysis.

亨廷顿舞蹈病（HD）是常染色体显性遗传的神经退行性疾病，编码亨廷顿蛋白（HTT）的突变形式。HD的病理特征是纹状体和皮质中神经元的丢失，这导致进行性运动功能障碍，认知能力下降和行为症状。基于干细胞的治疗已成为治疗神经退行性疾病的可行治疗方法，并且可能有效缓解和/或中止了HD的病理生理机制。一些临床前研究已经在HD动物模型中使用了干细胞。在这里，我们进行了临床前研究的系统评价，以评估干细胞在HD动物模型中的治疗效果。根据我们的系统审查，干细胞治疗可显着改善HD动物模型的神经和行为结果。尽管发现了令人鼓舞的结果，但动物研究的设计，移植细胞的类型和给药途径的标准化程度很低，这大大增加了比较分析的难度。