Fig. 2

Comparison of transduction efficiency between AAV9 and myotropic AAVs in several organs from wild-type adult mice. Mice were systemically injected at 6-week-old and organs collected 5 weeks later. (A) Quantification of vector copy number per diploid genome of AAVMYO-, MyoAAV2A-, MyoAAV4A- and AAV9-CMV-luc-IRES-eGFP in WT CD1 and C57BL/6 mice in tibialis anterior leg muscle, diaphragm, heart and liver. Data are presented as mean values +/- SEM (n = 5–6). One-way ANOVA with Tukey correction. *p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001 versus AAV9 ; #p < 0.05, ##p < 0.01, ###p < 0.001, ####p < 0.0001 versus AAVMYO ; $p < 0.05, $$$$<0.0001 versus MyoAAV2A ; §p < 0.05, §§p < 0.01, §§§§p < 0.05 versus MyoAAV4A. (B) Quantification of eGFP mRNA fold change expression of AAVMYO-, MyoAAV2A-, MyoAAV4A- and AAV9-CMV-luc-IRES-eGFP in WT CD1 and C57BL/6 mice in different organs. Data are presented as mean values +/- SEM (n = 5 for C57BL/6 mice, n = 9–12 for CD1 mice). One-way ANOVA with Tukey correction. *p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001 versus AAV9; #p < 0.05, ##p < 0.01, ####p < 0.0001 versus AAVMYO; §p < 0.05, §§p < 0.01 §§§§p < 0.0001 versus MyoAAV4A. (C) Quantification of luciferase activity of AAVMYO-, MyoAAV2A-, MyoAAV4A- and AAV9-CMV-luc-IRES-eGFP in WT CD1 and C57BL/6 mice in different organs. Data are presented as mean values +/- SEM (n = 5 for C57BL/6 mice, n = 9–13 for CD1 mice). One-way ANOVA with Tukey correction. *p < 0.05, **p < 0.01, ***p < 0.001, ****p < 0.0001 versus AAV9; ##p < 0.01, ####p < 0.0001 versus AAVMYO ; $p < 0.05, $$p < 0.01 versus MyoAAV2A ; §p < 0.05 versus MyoAAV4A