We generated new in vitro model for sporadic form of amyotrophic lateral sclerosis by reprogramming isolated skin fibroblasts into iPSCs. Fibroblasts were reprogrammed with commercially available synthetic polycistronic, self-replicating RNA vector. As verified by FISH, an early passages of a new iPSC line showed mosaic karyotype (cells with normal and abnormal karyotype 46,XY,t(2;14)(q13;p12) were present), while late passages contained only cells with abnormal karyotype. New iPSCs differentiated into all three germ layers and formed a teratoma in nude mice. Our iPSC line represents a new model for therapy testing and drug development in the field of ALS research.

通过将孤立的皮肤成纤维细胞重编程为iPSC，我们生成了散发形式的肌萎缩性侧索硬化的新体外模型。用可商购的合成多顺反子，自我复制RNA载体对成纤维细胞进行重编程。经FISH验证，新iPSC品系的早期传代显示出花叶核型（存在正常和异常核型46，XY，t（2; 14）（q13; p12）的细胞），而后期传代仅包含异常的细胞核型。新的iPSC分化为所有三个胚层，并在裸鼠中形成畸胎瘤。我们的iPSC系列代表了ALS研究领域中用于治疗测试和药物开发的新模型。