Figure 4.

The Synergistic Effect of plTALE_ST10X on the Transcription of Endogenous FXN Gene and on Aconitase Activity

(A and B) The synergistic effect of 2 plTALE_ST10Xs (targeting sequences 6 and 8) containing either 13 or 15 RVDs on the activation of FXN gene transcription in FRDA4078 cells. The number of copies of FXN mRNAs was normalized with GAPDH (A) or HPRT (B) mRNAs (n = 5). (C) Increased aconitase activity in FRDA4078 cells treated with plTALE_ST10X-6-15 + plTALE_ST10X-8-15 plasmids. This increase is relative to negative controls (untreated FRDA4078 cells) and negative cells (control−) treated with pAAV-scFV-sfGFP-VP64-GB1-NLS and with pCR3.1 empty plasmid (n = 3). *p < 0.05 and ****p < 0.0001. (A–C) Results are the average ± SEM.