Figure 7.
In Vivo Induction of the FXN Gene by AAV9-plTALEVP64s
The FXN gene was induced in mice treated with AAV9, which expressed either the plTALE6-15, plTALE8-15, or plTALEF4-15. The FXN gene expression was analyzed in four organs (muscle, heart, liver, and brain). The negative controls YG8R mice were either untreated or treated with AAV9 (AAV9-ctrl), which did not express plTALEVP64. (A) The transcriptional activity of the FXN gene. (B) The expression of the frataxin protein in YG8R mice treated with plTALEVP64 compared with negative control YG8R mice. *p < 0.05, **p < 0.003, and ****p < 0.0001. (A and B) Results are the average ± SEM.