Figure 1. Generation of Dmd-mutated rats via the CRISPR/Cas9 system.
Scheme for targeting the rat Dmd gene at exons 3 (a) and 16 (b) using CRISPR/Cas9. The gRNA sequences (magenta) and protospacer adjacent motif (PAM; green) are labeled. Representative sequences and their waveform data are presented below. (c) RT-PCR for Dmd and Hprt in wild type (WT) and Dmd-mutated F0 rats. These images were cropped and full-length gels are shown in Supplementary Figure 8a and b. (d) Immunoblotting for Dystrophin and α-tubulin in tibialis anterior (TA) muscles of WT and Dmd-mutated F0 rats. These images were cropped and full-length blots are shown in Supplementary Figure 8c and d. (e) Immunostaining for Dmd in WT and Dmd-mutated F0 rats. Scale bar = 100 μm.