Table 3 Organ concentrations (pg plasmid DNA per mg wet tissue) in female and male NPC1−/− mice at 4 days following the last IV injection of either vehicle or THLs.

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

Sex

Treatment

Brain

Spleen

Liver

Female

Vehicle

 < 0.6

 < 0.6

 < 0.6

Male

Vehicle

 < 0.6

 < 0.6

 < 0.6

Female

THL

10.1 ± 3.1

107.3 ± 40.5

39.9 ± 7.9

Male

THL

8.7 ± 0.6

58.6 ± 12.9

30.0 ± 7.8

  1. Mean ± SEM (N = 4 male or 4 female mice per vehicle or THL treatment group). PCR Cq values were converted to pg plasmid DNA with a pPDGF-NPC1 plasmid DNA standard curve.
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