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.

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

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.