Induction by plTALEVP64s of the FXN Gene in FRDA4078 Fibroblasts
(A) Number of FXN mRNA copies per microgram of total RNA extracted from cells treated with 14 different plTALEVP64s compared to negative controls. 2×plTALEs is for the result of plTALE6-15 with plTALE8-15; 3×plTALEs for plTALE6-15 + plTALE8-15 + plTALEF4-15; 4×plTALEs for plTALE6-15 + plTALE8-15 + plTALEF4-15 + plTALE10-15. (B) Increased transcriptional activity of the FXN gene normalized to GAPDH gene transcription in treated FRDA4078 cells compared to negative controls. (C) Induction of the FXN gene by the 3 best plTALEVP64s normalized to GAPDH gene and compared to the controls (n = 10). (D and E) Induction of expression of the frataxin protein in FRDA4078 cells treated with the best plTALEVP64s (alone or in pairs). Western blots for frataxin protein (D) were quantified densitometry and normalized with the GAPDH band (E). *p < 0.05, **p < 0.003, ***p < 0.0003, and ****p < 0.0001. (A and B) Results are the average ± SEM.