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

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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.