Figure 1.

Visualization of Gene Therapy Efficacy by Transcriptome Profiling in XLMTM Dogs

(A) Experimental timeline in weeks and details of the 5 groups of dogs. The rAAV vector was injected at 10 weeks of age, and dogs were monitored until the age of 39 to 41 weeks, unless euthanasia criteria were reached prematurely, as encountered in untreated XLMTM and AAVLow dogs. Additional details are provided in Table 1. (B) Heatmap representing the degree of corrections of key XLMTM phenotypes achieved in the three groups of dogs, as measured in our previous study.⁵ (C) Principal component analysis (PCA) on RNA-seq data in the Biceps femoris after selection of the top 500 most informative genes across samples. (D) PCA on RNA-seq data in the Vastus lateralis after selection of the top 500 most informative genes across samples. For (C) and (D), PC1 and PC2 were projected in a two-dimensional plot, with each dog represented as a color-coded dot.