ALS Centre, Department of Neurology, Eastern Piedmont University, "Maggiore della Carità" Hospital, Novara, Italy.
Prog Brain Res. 2012;201:333-59. doi: 10.1016/B978-0-444-59544-7.00016-0.
Amyotrophic lateral sclerosis (ALS) is a devastating incurable, neurodegenerative disease that targets motor neurons (MNs) in the primary motor cortex, brainstem, and spinal cord, leading to muscle atrophy, paralysis, and death due to respiratory failure within 2-5 years. Currently, there is no cure for ALS. The development of a therapy that can support or restore MN function and attenuate toxicity in the spinal cord provides the most comprehensive approach for treating ALS. Mesenchymal stem cells might be suitable for cell therapy in ALS because of their immunomodulatory and protective properties. In this review, the authors discuss the major challenges to the translation of in vitro and animal studies of MSCs therapy in the clinical setting.
肌萎缩侧索硬化症（ALS）是一种毁灭性的不可治愈的神经退行性疾病，它靶向初级运动皮层、脑干和脊髓中的运动神经元（MNs），导致肌肉萎缩、瘫痪，并在 2-5 年内因呼吸衰竭而死亡。目前，尚无治疗 ALS 的方法。开发一种能够支持或恢复 MN 功能并减轻脊髓中毒性的治疗方法，为治疗 ALS 提供了最全面的方法。间充质干细胞因其免疫调节和保护特性，可能适合用于 ALS 的细胞治疗。在这篇综述中，作者讨论了将间充质干细胞治疗的体外和动物研究转化为临床应用的主要挑战。
