Fig. 3

BTHS mouse gene therapy experimental setup showing A the therapeutic vector design, B the experimental cohorts, and C the experimental timeline. D Graph depicting the fold increase in coTAFAZZIN transcript levels over untreated BTHS controls in heart and gastrocnemius. E Fold increase in coTAFAZZIN transcripts over untreated controls in other tissues. Heart (Ht), gastrocnemius (Ga), soleus (So), brain (Br), liver (Li), spleen (Sp), kidney (Ki), lung (Lu). (t-tests were performed for comparisons between BTHS AAV9 and BTHS Ex + AAV9 cohorts and data indicate mean ± SEM) *p ≤ 0.05, **p ≤ 0.01, ***p ≤ 0.005