Quantifiers of Transcriptome Remodeling and Gene Therapy Efficacy in XLMTM Dogs
(A) Venn diagram representing the intersections of differentially expressed gene populations obtained after comparison of untreated XLMTM, rAAV-treated XLMTM, and healthy dogs. Focus is made on the Rescued and the Resistant two-circle domains in specific insets and on the central domain containing Partially Rescued and Worsened genes. Exemplar gene-expression levels are plotted on a line for the three groups: healthy, XLMTM, and rAAV-treated (AAV) dogs. n.s., non-significant gene expression difference; *, significant difference. (B and C) Evolution of the described metrics in the Sub-therapeutic and the Therapeutic groups of dogs—as defined in the text—in the Biceps (B) and the Vastus (C) muscles. The initial DE gene set in XLMTM dogs is represented as a blue ring, and its counterpart in healthy controls as a green ring. In rAAV-treated dogs, the areas corresponding to the above-described metrics have an arc length directly proportional to the proportion of initial DE genes presenting the corresponding expression profile.