Figure 2.

Behavioral manifestations after varying dosages of AAV-Slc25a46 vector treatment in Slc25a46^−/− mutants. (A) A Kaplan–Meier survival curve of injected versus uninjected mice shows that the majority of untreated Slc25a46^−/− mutants died prematurely. In contrast to untreated mutants, mutants treated with 1 × 10¹¹ GC/g AAV–Slc25a46 vector gained a longer lifespan, but not at a statistically significant level (P = 0.1425). However, the mutants treated with 2 × 10¹¹ GC/g AAV–Slc25a46 vector did show a significantly extended life-span (P = 0.0144) (WT, n = 16; Slc25a46^−/− mutants, n = 16; mutants treated with 1 × 10¹¹ GC/g, n = 8; mutants treated with 2 × 10¹¹ GC/g, n = 15.) (B) Bodyweight monitoring starting at P10. The bodyweights of 2 × 10¹¹ GC/g AAV-Slc25a46 vector-treated male mutants were notably and significantly increased relative to untreated Slc25a46^−/− male mutants and the male mutants treated with 1 × 10¹¹ GC/g (P = 0.0018 and 0.0070, respectively, with n = 6 for each group). (C) The growth defect was alleviated after treatment with a 2 × 10¹¹ GC/g dose of the AAV–Slc25a46 vector, and the body size of the 2 × 10¹¹ GC/g AAV-Slc25a46 treated mutants was distinctly larger than untreated mutants.