Generation of ORIONi001-A induced pluripotent stem cell line for in vitro modeling of sporadic form of amyotrophic lateral sclerosis

生成 ORIONi001-A 诱导多能干细胞系,用于体外模拟散发性肌萎缩侧索硬化症

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作者:Jan Strnadel, Romana Zahumenska, Vladimir Nosal, Marek Smolar, Juraj Marcinek, Michal Kalman, Stefan Juhas, Jana Juhasova, Hana Studenovska, Hugo Dumortier, Tomas Chromec, Henrieta Skovierova, Barbora Mitruskova, Ivan Kapralik, Sandra Mersakova, Dusan Brany, Erika Halasova

Abstract

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.

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