Figure 6.
Induction of the FXN Gene by the 2 Best plTALEST10X and Their Synergistic Effect in Fibroblasts of 4 Different FRDA Patients Compared with Those of 4 Normal Subjects
(A) Number of copies of FXN mRNA in 4 different normal fibroblasts (NED) and in 4 FRDA cells (FRDA66 cells, with 240/640 GAA repeats; FRDA162 cells, with 355/805 GAA repeats; FRDA4675 cells, with 255/1,140 GAA repeats; and FRDA4743 cells, with 470/970 GAA repeats) in negative control fibroblasts and after induction of the endogenous FXN gene with the 2 best plTALESTs (i.e., plTALEST10X-6-15 and plTALEST10X-8-15). (B and C) FXN mRNA normalized with GAPDH (B) or HPRT (C) mRNAs in various FRDA cells not treated or treated with plTALEST10X-6-15, plTALEST10X-8-15 alone or in pair. (D and E) Induction of the expression of the frataxin protein in cells from these 4 FRDA patients treated with 1 or 2 plTALEST10X with respect to the negative controls. Western blots for frataxin protein (D) were quantified by densitometry and normalized with the GAPDH band (E). *p < 0.05, **p < 0.003, ***p < 0.0003, and ****p < 0.0001. (A–C and E) Results are the average ± SEM.