Table 1.

Current status of FRDA iPSC research. Some studies utilized FRDA iPSCs as well as iPSC-derived neurons and/or cardiomyocytes.

Cell type	Study purpose	References
FRDA iPSCs	- Generation of FRDA GAA instability model	[38]
	- Investigation of the role of mismatch repair enzymes in GAA repeats expansion	[39]
	- In vitro study of FXN dynamic change in expression during differentiation	[47]
	- Reversing transcriptional repression caused by elongated GAA tracks by altering specific epigenetic modifications	[45]
	- Studies on DNA replication at the FXN locus	[44]
FRDA iPSC-derived cardiomyocytes	- Generation of patient specific cells affected in FRDA	[46]
	- Investigation of mitochondrial damage in FRDA	[48]
	- Modeling iron-overload induced cardiomyopathy	[49]
	- Drug screening platform for studying iron-overload cardiomyopathy in FRDA	[50]
	- Cardiac-specific electrophysiological profile of FRDA	[51]
	- Activating FXN expression by synthetic transcription elongation factors	[52]
FRDA iPSC-derived peripheral sensory neurons	- Generation of patient specific cells affected in FRDA	[46]
	- In vitro study of FXN dynamic change in expression	[47]
	- Reversing oxidative stress and loss of Fe-S proteins with 2-Aminobenzamide HDAC inhibitors	[53]
FRDA iPSC-derived neural stem cells	- Investigation of the role of mismatch repair enzymes in GAA repeats expansion	[39]
	- Proteomic study of targets and pathways of 2-Aminobenzamide HDAC inhibitors	[54]
FRDA iPSC - derived neurons	- Investigation of mitochondrial damage in FRDA	[48]
	- Studying mechanism of apoptosis in FRDA	[55]
	- FXN expression upregulation via zinc finger nuclease-mediated GAA tract excision	[56]
	- FXN expression upregulation via 2-Aminobenzamide HDAC inhibitors	[57]
	- FXN deficiency alleviation via HDACi treatment	[58]
	- Activating FXN expression by synthetic transcription elongation factors	[52]
	- FXN expression upregulation via 2-Aminobenzamide HDAC inhibitors	[59]
FRDA iPSC-derived neural progenitors	- Functional characterization and brain integration	[60]
FRDA iPSC-derived  retinal pigment  epithelium cells	- Function and characterization of retinal pigment epithelium in FRDA patients	[61]