肌萎缩侧索硬化症(ALS)是一种运动神经元的进行性变性疾病。其发病机制未明,目前尚无十分满意的治疗方法。因此针对该病的治疗研究进展,我们将近年基因治疗(转入神经营养因子、基因沉默抑制突变SOD1表达等)和干细胞治疗的研究结果予以综述。 Amyotrophic lateral sclerosis (ALS) is a progressive degenerative disease of motor neurons. Its pathogenesis is unknown, there is no satisfactory treatment. Therefore, in view of the progress of the treatment of the disease, we reviewed the research results of gene therapy (such as neurotrophic factor, gene silencing inhibitor of mutant SOD1 expression) and stem cell therapy in recent years.