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目的：探索腰椎穿刺鞘内注射移植间充质干细胞（MSCs）或经脑脊液诱导分化的神经样干细胞（NSCs）治疗神经变性疾病（NDD）的临床疗效和安全性。方法根据骨髓细胞学分析结果是否正常，将14例符合入选标准的NDD患者分为：骨髓源干细胞治疗组（A组，骨髓细胞正常），脐血源干细胞治疗组（B组）。采用分离培养、脑脊液诱导分化方法获得MSCs和NSCs，通过腰椎穿刺鞘内注射进行细胞移植治疗；于移植治疗前、首次移植后3个月、6个月，采用特定神经功能量表、生活活动能力量表（BI）及生活质量量表（EuroQOL）对患者神经功能状况进行评估；同期，观察记录移植相关不良反应及并发症。结果14例NDD患者中A组8例、B组6例，性别构成、平均年龄差异无统计学意义。细胞移植后A组和B组患者的神经功能有不同程度的改善；治疗后BI分值和EuroQOL分值优于治疗前，差异有统计学意义；而2组间差异无统计学意义。移植术后有3例出现过短暂性低热，A组2例，B组1例，对症处理可控制；随访期间A组有1例患者发生心源性猝死，B组有3例患者死于继发性肺部感染。最长随访时间已超过2年，未出现继发性肿瘤等细胞移植严重并发症。结论腰穿鞘内注射移植骨髓源或脐血源MSCs及其经脑脊液诱导获得的NSCs能显著改善NDD患者的神经功能，无严重不良发应和并发症；当NDD患者自身骨髓细胞分析异常，可用脐血源干细胞替代自体骨髓源干细胞进行细胞移植治疗。“,”Objective To explore the clinical efficacy and safety of intrathecal transplantation of human mesenchymal stem cells (MSCs) or cerebrospinal fluid (CSF) induced neural stem cells (NSCs) by lumbar puncture to treat patients with neurodegenerative diseases (NDD). Methods A total of 14 eligible patients with NDD were divided into a bone marrow stem cell (MSC) treatment group (Group A, normal bone marrow cells) and a umbilical cord blood stem cell (NSC) treatment group (Group B, with abnormal bone marrow cytology), according to their cytological examination of the bone marrow. Then, MSCs and NSCs obtained through isolation and differentiation inducement by CSF were intrathecally injected by lumbar puncture for transplantation therapy. The neurological function of all patients were evaluated using a specific neurological scale, the life events scale (BI) and the quality of life scale (EuroQOL) before, three months and six months after transplantation. Meanwhile, adverse reactions and complications related transplantation were recorded. Results There were eight patients Group A and six in Group B, with no statistical difference in gender and average age. After cell transplantation, patients presented improved nervous functions in both Groups A and B. The scores of BI and EuroQOL were better than those prior to treatment. For adverse reactions, three patients reported a transient fever (2 cases in Group A and 1 case in Group B) which were controlled after symptomatic treatment. During follow-up visits, one patients in Group A died of sudden cardiac death, while three patients in Group B died due to secondary pulmonary infection. The longest follow-up period was more than 2 years, without secondarytumor and other serious complications. Conclusion Intrathecal injection of MSCs and CSF-induced NSCs by lumbar puncture can remarkably improve the neurological function of patients with NDD, without serious adverse reaction and complications. When NDD patients manifested abnormal bone marrow cytological results, umbilical cord blood stem cells can replace autologous bone marrow stem cells for transplantation therapy.