Yang Meimei, Liu Min, Ding Yicheng, Vajda Alice, Ma Jun, Cui Huixian, O'Brien Timothy, Henshall David, Hardiman Orla, Shen Sanbing
Regenerative Medicine Institute (REMEDI), School of Medicine, National University of Ireland (NUI) Galway, Galway, Ireland; FutureNeuro, The SFI Research Centre for Chronic and Rare Neurological Diseases, Royal College of Surgeons in Ireland, D02, Dublin, Ireland.
Department of Physiology, College of Life Science, Hebei Normal University, Shijiazhuang, Hebei 050024, China.
Stem Cell Res. 2020 Apr;44:101752. doi: 10.1016/j.scr.2020.101752. Epub 2020 Mar 17.
The majority of amyotrophic lateral sclerosis are sporadic (sALS) with no familial history or known genetic association, therefore a large cohort of disease models are required to identify common mechanisms or to test therapeutic interventions. Here we generated twelve induced pluripotent stem cell (iPSC) lines from human dermal fibroblasts of two healthy individuals and two sALS patients lacking common ALS mutations, using non-integrational Sendai virus expressing reprogramming factors OCT3/4, KLF4, SOX2 and c-MYC. The iPSC lines highly expressed pluripotency markers could be spontaneously differentiated into three embryonic germ layers, with no gross chromosomal aberrations or specific copy number variations.
大多数肌萎缩侧索硬化症是散发性的（sALS），没有家族病史或已知的遗传关联，因此需要大量的疾病模型队列来确定共同机制或测试治疗干预措施。在这里，我们使用表达重编程因子OCT3/4、KLF4、SOX2和c-MYC的非整合仙台病毒，从两名健康个体和两名缺乏常见ALS突变的sALS患者的人皮肤成纤维细胞中生成了12条诱导多能干细胞（iPSC）系。高度表达多能性标志物的iPSC系可自发分化为三个胚层，没有明显的染色体畸变或特定的拷贝数变异。
