FIGURE 5:

Vector transduction and expression efficiency. Panel (A) shows vector genomes in Tk2^KI mice under different treatments. Values are expressed as vector genomes per nucleus. Panel (B) shows gene expression of TK2 and Tk2 from Tk2^KI-AAV9-TK2 and Tk2^WT-AAV9-TK2 in different tissues and time points. Values are expressed as ddCt values using mouse Gapdh as reference gene. Panel (C) shows mtDNA levels in Tk2^WT-AAV9 at age 2 months (postnatal day 60). Values are expressed as percentages of mtDNA in Tk2^WT untreated mice. Panel (D) shows Tk2 expression in muscle from Tk2^WT-AAV9 at age 2 months (postnatal day 60). Values are expressed as percentages of Tk2 mRNA in Tk2^WT untreated mice. In all panels, Tk2^WT-AAV9 and TK2^KI-AAV9 refer to the high-dose group (4.2 × 10¹¹ vg).