Figure 1.

Repair of dystrophin in DMD-iPSC-CMs using CRISPR/Cas9. (A) Strategy of genome editing using CRISPR/Cas9. Exon 55 in DMD-iPSCs was deleted using each sgRNAs designed upstream and downstream of exon 55 to modify from “out of frame” to “in frame.” (B) Exon size from exon 47 to exon 56 in Con-iPSCs, DMD-iPSCs, and Ed-DMD-iPSCs determined using PCR with complementary DNA (cDNA) as the template. (C) Sanger sequencing of DMD-iPSCs (Δexon 48–54) and Ed-DMD-iPSCs (Δexon 48–55). (D,E) Expression of dystrophin in Con-iPSC-CMs, DMD-iPSC-CMs, and Ed-DMD-iPSC-CMs determined using western blotting and immunofluorescence staining (scale bar 50 µm).