Figure 4.

Transduction efficiency and specificity of AAV vector variants in vivo based on GFP expression

AAV vectors (AAV2, AAV9, AAV2-THGTPAD, AAV2-NLPGSGD, and AAV2-LPSRPSL, expressing GFP) were injected in sham- and TAC-operated mice 6 weeks after sham or TAC surgery (note that each experimental group started with four mice each, but the final group size differs owing to the loss of individual mice; n as the number of mice is depicted for each group). GFP expression in cardiac cell types and different organs was analyzed by qRT-PCR 2 weeks after vector injection. (A) GFP expression analysis (separately for all different groups: sham or TAC with respective AAV vector variants) and (B) analysis based on pooled groups (sham + TAC mice, in case of AAV2, AAV9, AAV-THGTPAD, and AAV2-NLPGSGD—vectors that did not show noticeable differences between sham and TAC according to (B) (n = 3–7). (C) CMC to liver expression ratio normalized to AAV9 (n = 3–7). Data are means ± standard deviation. The p values were determined by one-way ANOVA with Dunnett’s multiple comparison with the current gold standard AAV9. GFP, self-complementary vector genome conformation encoding for enhanced green fluorescence protein; CF, cardiac fibroblasts; EC, cardiac endothelial cells; sk. muscle, skeletal muscle.