Ferraiuolo Laura
*The Research Institute at Nationwide Children's Hospital, Columbus, OH 43205, U.S.A.
Biochem Soc Trans. 2014 Oct;42(5):1270-4. doi: 10.1042/BST20140168.
Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disorder affecting the motor nerves. At present, there is no effective therapy for this devastating disease and only one Food and Drug Administration (FDA)-approved drug, riluzole, is known to moderately extend survival. In the last decade, the field of ALS has made a remarkable leap forward in understanding some of the genetic causes of this disease and the role that different cell types play in the degenerative mechanism affecting motor neurons. In particular, astrocytes have been implicated in disease progression, and multiple studies suggest that these cells are valuable therapeutic targets. Recent technological advancements have provided new tools to generate astrocytes from ALS patients either from post-mortem biopsies or from skin fibroblasts through genetic reprogramming. The advent of induced pluripotent stem cell (iPSC) technology and the newly developed induced neural progenitor cells (iNPCs) have created unprecedented exciting opportunities to unravel the mechanisms involved in neurodegeneration and initiate high-throughput drug screenings.
肌萎缩侧索硬化症（ALS）是一种影响运动神经的致命性神经退行性疾病。目前，对于这种毁灭性疾病尚无有效的治疗方法，已知只有一种美国食品药品监督管理局（FDA）批准的药物利鲁唑能适度延长患者生存期。在过去十年中，ALS领域在了解该疾病的一些遗传病因以及不同细胞类型在影响运动神经元的退化机制中所起的作用方面取得了显著进展。特别是，星形胶质细胞与疾病进展有关，多项研究表明这些细胞是有价值的治疗靶点。最近的技术进步提供了新工具，可通过遗传重编程从ALS患者的死后活检组织或皮肤成纤维细胞中生成星形胶质细胞。诱导多能干细胞（iPSC）技术的出现以及新开发的诱导神经祖细胞（iNPC）为揭示神经退行性变的机制和开展高通量药物筛选创造了前所未有的令人兴奋的机会。
