Kim Changsung, Lee Hee Chul, Sung Jung-Joon
Department of Bioscience and Biotechnology, Sejong University, Seoul 143-747, Korea.
Department of Neurology, Seoul National University Hospital, Seoul 110-774, Korea.
Exp Neurobiol. 2014 Sep;23(3):207-14. doi: 10.5607/en.2014.23.3.207. Epub 2014 Sep 18.
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease, characterized by the predominant loss of motor neurons (MNs) in primary motor cortex, the brainstem, and the spinal cord, causing premature death in most cases. Minimal delay of pathological development by available medicine has prompted the search for novel therapeutic treatments to cure ALS. Cell-based therapy has been proposed as an ultimate source for regeneration of MNs. Recent completion of non-autologous fetal spinal stem cell transplant to ALS patients brought renewed hope for further human trials to cure the disease. Autologous somatic stem cell-based human trials are now in track to reveal the outcome of the ongoing trials. Furthermore, induced pluripotent stem cell (iPSC)-based ALS disease drug screen and autologous cell transplant options will broaden therapeutic options. In this review paper, we discuss recent accomplishments in cell transplant treatment for ALS and future options with iPSC technology.
肌萎缩侧索硬化症（ALS）是一种神经退行性疾病，其特征是原发性运动皮层、脑干和脊髓中的运动神经元（MNs）大量丧失，在大多数情况下会导致过早死亡。现有药物对病理发展的延缓作用微乎其微，这促使人们寻找治疗ALS的新型疗法。基于细胞的疗法已被提议作为运动神经元再生的最终来源。最近对ALS患者进行的非自体胎儿脊髓干细胞移植的完成，为进一步的人体试验治愈该疾病带来了新的希望。基于自体体细胞干细胞的人体试验目前正在进行，以揭示正在进行的试验的结果。此外，基于诱导多能干细胞（iPSC）的ALS疾病药物筛选和自体细胞移植选择将拓宽治疗选择。在这篇综述文章中，我们讨论了ALS细胞移植治疗的最新成果以及iPSC技术的未来选择。
