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Figure 7
Validation of ASO-induced dual exon skipping for USH2A exons 30–31 and USH2A exons 39–40 in WERI-Rb-1 cells
Co-transfection of WERI-Rb-1 cells with ASOs targeting either (A) exons 30 and 31 or (B) exons 39 and 40, resulted in the combined skipping of the exons of interest. Co-transfection of WERI-Rb-1 cells with ASOs targeting exons 30 and 31 also resulted in additional skipping of exon 32. In WERI-Rb-1 cells treated with ASOs targeting exons 39 and 40, no detectable amounts of alternatively spliced transcripts different from untransfected samples were present. GAPDH amplification is shown as a loading control. ASO, antisense oligonucleotide; mmASO, mismatch ASO; PCR(−), negative PCR control.