Figure 6.

The FSHD-optimized regulatory cassette is active in skeletal muscles

In vivo AAV9-mediated mCherry expression under control of the FSHD-optimized regulatory cassette (A), (Figure 1B) was visualized in the indicated tissues at 12 weeks post-injection with the same exposure time (0.5 s). (B–H and L) For two-tissue panels, tissues from uninjected mice are shown on the left. (I’–K) Single-tissue panels are AAV injected, and (I) is uninjected. All injected tissues are indicated by an asterisk. Expression of mCherry was detected in skeletal muscles (tibialis anterior [TA], gastrocnemius [GA], extensor digitorum longus [EDL], and quadriceps [QUA], as well as pectoral, abdominal, and facial muscles) and was undetectable in soleus (SOL), heart, and testis.