Figure 5.

Induction of the FXN Gene in 4 Different Types of FRDA Cells with 3 Best plTALE_VP64

Fibroblasts of 4 different FRDA patients (i.e., FRDA66, with 240 and 640 GAA repeats; FRDA162, 355/805; FRDA4675, 255/1,140; and FRDA4743, 470/970) were treated with plTALE_VP64-6-15, plTALE_VP64-8-15, or plTALE_VP64-F4-15 to increase the expression of the endogenous FXN gene. The FXN expression was compared to untreated cells and cells treated with empty pCR3.1. (A) The expression of the FXN gene in control cells and in treated FRDA cells is presented as the number of FXN mRNA copies per microgram RNA. (B and C) The FXN mRNAs are normalized with either GAPDH (B) or HPRT (C) mRNAs. (D–G) Frataxin protein in cells from these 2 FRDA (D and E in FRDA4675 and F and G in FRDA4743) patients were treated with the best plTALE_VP64s and compared with negative controls (untreated cells or cells treated with empty plasmid). *p < 0.05, **p < 0.003, ***p < 0.0003, and ****p < 0.0001. (A–C) Results are the average ± SEM.