FIG. 1.

Study design and human MCT8 expression in the liver and brain regions after IV AAV9-MCT8 injection of P30 dKO mice. (A) Schematic of experimental design. dKO mice were treated at postnatal day 30 (P30) by tail vein (IV) delivery of AAV9-MCT8 at a dose of 50 × 10¹⁰ vp/g. (B) Quantification of MCT8 mRNA levels by qRT-PCR showed MCT8 re-expression relative to the three housekeeping genes Polr2a, Actb, and Gapdh in the liver and different brain regions of dKO treated animals. One-way ANOVA with Tukey's multiple comparisons. The data are presented as mean, and error bars represent SEM. (***p < 0.001, ****p < 0.0001). AAV9, adeno-associated virus serotype 9; ANOVA, analysis of variance; dKO, double knockout; IV, intravenous; MCT8, monocarboxylate transporter 8; mRNA, messenger RNA; qRT-PCR, quantitative real-time polymerase chain reaction; SEM, standard error of the mean; vp, viral particles.