TABLE 1.

Nomenclature and mice description.

Nomenclature	Mice description
Tg +	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with doxycycline
Tg-	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with no doxycycline
Tg ±	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] mice that were treated with doxycycline food 9 weeks and 9 weeks without doxycycline (rescue)
WT +	Wild-type treated with doxycycline
WT-	Wild-type treated without doxycycline
WT ±	Wild-type treated with doxycycline for 9 weeks and 9 weeks without doxycycline (rescue)
PGK Half	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with doxycycline and at week 10 received one dose of AAV8-PGK-mFXN vector (0.5 E + 14 vg/kg) intravenous injection
PGK Low	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with doxycycline and at week 10 received one dose of AAV8-PGK-mFXN vector (1.00E + 14 vg/kg) intravenous injection
PGK High	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with doxycycline and at week 10 received one dose of AAV8-PGK-mFXN vector (3.00E + 14 vg/kg) intravenous injection
Placebo	Transgenic [harboring the FXN shRNA transgene (Tet-O model)] treated with doxycycline and at week 10 received one dose of vehicle (Lactated Ringer’s Solution w/10% Poloxamer: 1 μl/g) intravenous injection