|
Figure 6
ASOs induce a dose-dependent skipping of USH2A target exons
HEK293T cells are co-transfected with the exons 30–31 minigene splice vector and different concentrations of ASOs targeting (A) exon 30 and (B) exon 31 or with the exons 39–40 minigene splice vector and different concentrations of ASOs targeting (C) exon 39, (D) exon 40, or (E) exons 39 and 40. Each transfection resulted in a concentration-dependent increase in transcripts without the targeted exon(s). Amplicons with and without the targeted exon(s) are indicated adjacent to the gel image. For simultaneous transfection of multiple ASOs, the total ASO concentration is shown. GAPDH amplification is shown as a loading control. ASO, antisense oligonucleotide; mmASO, mismatch ASO; PCR(−), negative PCR control.