Generation of Induced Pluripotent Stem Cell Lines from Friedreich Ataxia Patients

Friedreich ataxia (FRDA) is an autosomal recessive disorder characterised by neurodegeneration and cardiomyopathy. It is caused by a trinucleotide (GAA) repeat expansion in the first intron of the FXN gene that results in reduced synthesis of FXN mRNA and its protein product, frataxin.

Generation of Induced Pluripotent Stem Cell Lines from Friedreich Ataxia Patients