Lee Gi Beom, Park Se Min, Jung Un Ju, Kim Sang Ryong
School of Life Science and Biotechnology, BK21 FOUR KNU Creative BioResearch Group, Kyungpook National University, Daegu 41566, Republic of Korea.
Department of Food Science and Nutrition, Pukyong National University, Busan 48513, Republic of Korea.
Biomedicines. 2024 Nov 1;12(11):2507. doi: 10.3390/biomedicines12112507.
Spinocerebellar ataxia (SCA) is a heterogeneous disorder characterized by impaired balance and coordination caused by cerebellar dysfunction. The absence of treatments approved by the U.S. Food and Drug Administration for SCA has driven the investigation of alternative therapeutic strategies, including stem cell therapy. Mesenchymal stem cells (MSCs), known for their multipotent capabilities, have demonstrated significant potential in treating SCA. This review examines how MSCs may promote neuronal growth, enhance synaptic connectivity, and modulate brain inflammation. Recent findings from preclinical and clinical studies are also reviewed, emphasizing the promise of MSC therapy in addressing the unmet needs of SCA patients. Furthermore, ongoing clinical trials and future directions are proposed to address the limitations of the current approaches.
脊髓小脑共济失调(SCA)是一种异质性疾病,其特征是由小脑功能障碍导致平衡和协调受损。美国食品药品监督管理局未批准用于治疗SCA的药物,这推动了包括干细胞疗法在内的替代治疗策略的研究。间充质干细胞(MSC)以其多能性而闻名,在治疗SCA方面已显示出巨大潜力。本综述探讨了MSC如何促进神经元生长、增强突触连接并调节脑部炎症。还回顾了临床前和临床研究的最新发现,强调了MSC疗法在满足SCA患者未满足需求方面的前景。此外,针对当前方法的局限性提出了正在进行的临床试验和未来方向。