Establishment of an induced pluripotent stem cell (iPSC) line (INNDSUi004-A) from a patient with Congenital Nemaline Myopathy

从患有先天性线状体肌病的患者体内建立诱导性多能干细胞 (iPSC) 系 (INNDSUi004-A)

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作者:Yingxin Wang, Wenzhu Liu, Yitong Yang, Yu Wang, Yao Tang, Zexin Zhan, Xiaohan Sun, Yichang Jiao, Didi Shan, Rui Zhang, Dongdong Wang, Ping Sun, Xiulian Sun, Chuanzhu Yan, Fuchen Liu

Abstract

We used a non-integrated reprogramming approach to establish a human induced pluripotent stem cell (hiPSC) line (INNDSUi004-A) from the skin fibroblasts of a 13-year-old female individual with Congenital Nemaline Myopath. The cells obtained have typical characteristics of embryonic stem cells, show expression of specific pluripotency markers, and can differentiate into three germ layers in vitro. This iPSC cell line has the genetic information of the patient and is a good model for studying disease mechanisms and developing novel therapies.

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