Figure 5

PPMO- k H25A(+95+A+119) mediated excision of exon 25 from patient cells. H25A(+95+A+119) synthesized as the PPMO-k chemistry was transfected into MyoD adenovirus converted patient cells (c.3385 Insertion A). The cells were collected 7 days after transfection for analysis of dystrophin mRNA (A) and protein (B) levels. Samples from normal myoblasts (Normal) and MyoD converted patient myogenic cells (Patient) are included for comparison. Sequencing of the predominant PCR products generated from the PPMO-k treated patient cells and control myoblasts (A) confirmed the normal exon 24/25 boundary in control myoblasts (C) and the exclusion of exon 25 in the PPMO-k treated patient transcripts (D).