Figure 4.

Restoration of Dystrophin Expression in Edited Myotubes

(A) Myoblasts from a healthy subject (WT) and from 4 DMD patients with different exon deletions were infected with the lentivirus coding for SaCas9, a pair of sgRNAs, and EGPF and allowed to fuse into myotubes for at least 7 days in low-serum medium. EGFP expression allowed us to observe that myoblasts infected with the lentivirus were able to fuse into myotubes. (B) The myotube proteins were then extracted and submitted to a western blot analysis for detection of the dystrophin protein using an anti-dystrophin monoclonal antibody (NCL-Dys2, Novocastra).