Xu Keli, Yang Minlin, Guan Lihong, Yang Ciqing, Qiao Liang, Li Yonghai, Lin Juntang, Li Xiaoying
Stem Cells and Biotherapy Engineering Research Center of Henan, National Joint Engineering Laboratory of Stem Cells and Biotherapy, School of Life Science and Technology, Xinxiang Medical University, Xinxiang, 453003, Henan, China.
Henan Key Laboratory of Medical Tissue Regeneration, Xinxiang Medical University, Xinxiang, 453003, Henan, China.
Mol Biotechnol. 2025 Apr 25. doi: 10.1007/s12033-025-01435-3.
Niemann-Pick disease (NPD) is a rare autosomal recessive neurodegenerative disease characterized by hepatosplenomegaly, neuropathy, and a significantly shortened lifespan. Lipid metabolism disorder is the main pathological feature of NPD. Currently, the exact pathogenesis of NPD remains unclear, and drug therapy is largely palliative, focusing on symptom management, but it has side effects. Mesenchymal stem cells (MSCs) possess several advantageous properties, including their differentiation potential, wide availability, low immunogenicity, and the ability to secrete regulatory factors, which have led to their extensive application in basic research targeting neurodegenerative diseases. Studies have demonstrated that transplantation of MSCs from different sources into animal models of NPD can delay the loss of Purkinje cells in the cerebellum, reduce lipid deposition, improve motor coordination, slow the rate of weight loss, and extend lifespan. This review explores the therapeutic potential of MSCs in the treatment of NPD, highlighting their emerging role in addressing this challenging condition.
尼曼-匹克病（NPD）是一种罕见的常染色体隐性神经退行性疾病，其特征为肝脾肿大、神经病变以及寿命显著缩短。脂质代谢紊乱是NPD的主要病理特征。目前，NPD的确切发病机制仍不清楚，药物治疗在很大程度上只是姑息性的，侧重于症状管理，但存在副作用。间充质干细胞（MSCs）具有多种优势特性，包括其分化潜能、广泛可得性、低免疫原性以及分泌调节因子的能力，这些特性使得它们在针对神经退行性疾病的基础研究中得到广泛应用。研究表明，将不同来源的MSCs移植到NPD动物模型中，可以延缓小脑浦肯野细胞的丧失，减少脂质沉积，改善运动协调性，减缓体重减轻速度，并延长寿命。本综述探讨了MSCs在治疗NPD方面的治疗潜力，强调了它们在应对这一具有挑战性的病症中日益重要的作用。
