Correction of Friedreich ataxia induced pluripotent stem cells by non-viral gene therapy

Friedreich共济失调(FRDA)是一种遗传性神经系统和心脏进行性疾病。干细胞疗法有可能修复或替代FRDA患者的受损组织和恢复器官功能。从FRDA患者身上获得的干细胞固有的缺陷将通过一种基因治疗方法得到纠正，这种方法将恢复FRDA基因的正常表达，并解决在移植医学中临床使用纠正后的干细胞的主要安全问题。

Friedreich ataxia (FRDA) is an inherited progressive disorder of the nervous system and heart. Stem cell therapy has the potential to repair or replace damaged tissues and restore organ function in FRDA patients. The defect inherent in stem cells obtained from FRDA patients will be corrected by a gene therapy approach that will restore normal FRDA gene expression and addresses major safety concerns for the clinical use of corrected stem cells in transplantation medicine.