Pharmacological Screening Using an FXN-EGFP Cellular Genomic Reporter Assay for the Therapy of Friedreich Ataxia
Figure 5
Compound evaluation in Friedreich ataxia cells.
The modulation of FXN gene expression by selected test compounds was evaluated in lymphoblast and fibroblast cell lines derived from individuals with FRDA. All cell lines were homozygous for a GAA repeat expansion in the first intron of the FXN gene. Cultures were incubated for 72 hours. FXN mRNA levels were measured by real-time RT-PCR. Frataxin protein levels were measured by lateral flow immunoassay. (A) FXN mRNA levels measured in FRDA lymphoblast cell lines. (B) FXN mRNA levels measured in FRDA fibroblast cell lines. (C) Frataxin protein levels measured in FRDA lymphoblast cell lines. (D) Frataxin protein levels measured in FRDA fibroblast cell lines. Assays were performed in triplicate on at least three independent occasions. Error bars represent standard error of the mean. *p<0.05, **p<0.01 in comparison to the untreated control.