Table 1.

Histology evaluation for efficacy and safety of AAV treatment in the FXN-MCK mouse model

Group	1	4	7	10	13
Genotype	Wild type	FXN-MCK	FXN-MCK	FXN-MCK	FXN-MCK
Treatment	Vehicle	Vehicle	AAV-mFXN 3E+13 vg/kg	AAV-mFXN 1E+14 vg/kg	AAV-hFXN 1E+14 vg/kg
Males
No. (animals examined)	2	3	3	3	2
Heart (no. examined)	N = 2	N = 3	N = 3	N = 3	N = 2
Degeneration, cardiomyocyte	(0)a	(3)	(0)	(0)	(0)
Minimal	0	1	0	0	0
Moderate	0	2	0	0	0
Thrombus, atrial	(0)	(1)	(0)	(0)	(0)
Mild	0	1	0	0	0
Fibrosis, myocardial	(0)	(2)	(0)	(0)	(0)
Mild	0	2	0	0	0
Infiltration, mononuclear cell	(2)	(3)	(1)	(1)	(2)
Minimal	2	3	1	1	2
Liver (no. examined)	N = 2	N = 3	N = 3	N = 3	N = 2
Vacuolation, centrilobular	(0)	(1)	(0)	(0)	(0)
Minimal	0	1	0	0	0
Females
No. (animals examined)	2	3	3	3	3
Heart (no. examined)	N = 2	N = 2b	N = 3	N = 3	N = 3
Degeneration, cardiomyocyte	(0)a	(2)	(0)	(0)	(0)
Minimal	0	1	0	0	0
Mild	0	1	0	0	0
Thrombus, atrial	(0)	(1)	(0)	(1)	(0)
Mild	0	1	0	1	0
Fibrosis, myocardial	(0)	(1)	(0)	(0)	(0)
Minimal	0	1	0	0	0
Infiltration, mononuclear cell	(0)	(1)	(0)	(1)	(0)
Minimal	0	1	0	1	0
Liver (no. examined)	N = 2	N = 3	N = 3	N = 3	N = 3
Vacuolation, centrilobular	(0)	(2)	(0)	(0)	(0)
Minimal	0	2	0	0	0

Select microscopic findings in the hearts and livers of cohort 1 males and females euthanized 4 weeks postdose.

^aThe numbers in parentheses represent the total counts of animals exhibiting findings.

^bFor animal numbers, see Materials and methods.