Bohl Delphine
Biol Aujourdhui. 2016;210(1):27-36. doi: 10.1051/jbio/2016004. Epub 2016 Jun 10.
Among motor neuron diseases, spinal muscular atrophy type 1 and amyotrophic lateral sclerosis are very aggressive diseases with no cure. With the breakthrough of human induced pluripotent stem cells, iPS, researchers have now at their disposal a powerful tool to generate human motor neurons in culture and study the pathological defects in patient cells. In this review, we will see which tools for the study of patients motoneurons were developed from iPS cells and the different cellular models that were generated. We will also see how these models were validated and current research to identify new therapeutic leads.
在运动神经元疾病中,1型脊髓性肌萎缩症和肌萎缩侧索硬化症是非常严重且无法治愈的疾病。随着人类诱导多能干细胞(iPS)技术的突破,研究人员现在有了一种强大的工具,可以在培养物中生成人类运动神经元,并研究患者细胞中的病理缺陷。在这篇综述中,我们将了解从iPS细胞开发出了哪些用于研究患者运动神经元的工具以及所生成的不同细胞模型。我们还将了解这些模型是如何得到验证的,以及目前为确定新的治疗线索所开展的研究。