Table 4 Organ enrichment of NPC1 mRNA, relative to GAPDH mRNA, in THL treated NPC1−/− mice as compared to vehicle treated NPC1−/− mice.

From: Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes

Organ

Treatment

NPC1 Cq

GAPDH Cq

ΔCq

ΔΔCq

NPC1 mRNA fold change

Spleen

Vehicle

35.5 ± 2.6

18.8 ± 1.1

16.9 ± 2.1

–

–

Spleen

THL

22.8 ± 0.7

18.9 ± 0.4

3.9 ± 1.0

13.0

8,192

Liver

Vehicle

33.9 ± 2.8

24.2 ± 1.7

10.0 ± 2.6

–

–

Liver

THL

26.4 ± 0.9

24.3 ± 1.3

2.1 ± 1.0

7.9

238

Brain

Vehicle

37.0 ± 1.4

21.4 ± 0.4

15.9 ± 1.2

–

–

Brain

THL

29.6 ± 0.8

22.1 ± 1.6

7.5 ± 1.8

8.4

338

  1. ΔCq = (NPC1 Cq − GAPDH Cq); ΔΔCq = [ΔCq (vehicle mice) − ΔCq (THL mice)]; NPC1 mRNA fold change between vehicle mice and THL mice computed from base 2 anti-log. Cq values are mean ± SD [8 mice (4 males; 4 females)] in vehicle group; 8 mice [(4 males; 4 females)] in THL group.
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