Table 3 Experimental groups

[16]

9

Npc1^nih: 6

Npc1^nmf164: 3

Npc1^nih

AAV9-GAPDH: 6

AAV9-PGK: 6

AAV9-NPC1: 6

AAV9-CBA: 6

AAV9-SYN: 6

AAV9-SYN-S: 6

AAV9-SYN-D: 6

AAV9-CBA: 6

AAV9-CAG: 6

Npc1^nmf164

AAV9-SYN: 3

AAV9-NPC1: 3

78

[11]

9 weeks: 3

End-stage: 5

9 weeks: 2

End-stage: 3

9 weeks: 1

End-stage: 6

9 weeks: 4

End-stage: 2

9 weeks

AAV9: 4

AAV-PHP.B: 2

End-stage:

AAV9: 4

AAV-PHP.B: 5

9 weeks

AAV9: 3

AAV-PHP.B: 2

End-stage:

AAV9: 5

AAV-PHP.B: 4

55

[22]

n.s.

8

10

8

10

36

[12]

11

Saline-treated, gel-food: 11

Untreated, gel-food:6

Untreated, standard food: 6

12

46

[15]

6

8

Low dose: 8

High dose: 8

Miglustat: 8

38

[14]

14–29

11–21

6–13

5–12

3–11

6–14

n.s.

[13]

5

7–17

8–16

n.s.

[10]

0

Untreated: 16

AAV9-GFP: 6

Neonates:

AAV9-CamKII: 6

Juvenile

AAV9-CamKII: 9

AAV9-EF1α: 7

44