Johns Hopkins University, 600 N. Wolfe St., Meyer 6-119, Baltimore, MD 21287, USA.
Curr Opin Biotechnol. 2009 Oct;20(5):545-51. doi: 10.1016/j.copbio.2009.09.003. Epub 2009 Oct 12.
Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disorder characterized primarily by motor neuron loss in the motor cortex and spinal cord leading to progressive disability and death. Despite the relative selectivity of motor neuron loss, recent studies have implicated other cell types including astrocytes and microglia as contributors to this cell death. This understanding has resulted in stem-cell-replacement strategies of these cell types, which may result in neuroprotection. In addition to cell-replacement strategies, the development of induced pluripotent stem cell (iPSC) technologies has resulted in the establishment of motor neuron cell lines from patients with ALS. The use of iPSCs from ALS patients will allow for potential autologous cell transplantation, drug discovery, and an increased understanding of ALS pathobiology.
肌萎缩侧索硬化症(ALS)是一种神经退行性疾病,主要表现为运动皮层和脊髓中的运动神经元丧失,导致进行性残疾和死亡。尽管运动神经元的丧失具有相对选择性,但最近的研究表明,其他细胞类型,包括星形胶质细胞和小胶质细胞,也是导致这种细胞死亡的因素。这种认识导致了针对这些细胞类型的干细胞替代策略,这可能会带来神经保护作用。除了细胞替代策略外,诱导多能干细胞(iPSC)技术的发展已经从 ALS 患者中建立了运动神经元细胞系。使用 ALS 患者的 iPSCs 将允许进行潜在的自体细胞移植、药物发现,并增加对 ALS 病理生物学的理解。