Extended Data Fig. 6: CLN3Δex7/8 mRNA is a substrate for nonsense-mediated decay.
From: Therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
a, RT-PCR analysis of CLN3Δex7/8 mRNA isolated from a CLN3 Batten disease patient fibroblast cell line transfected with the indicated ASOs and treated with puromycin to inhibit NMD ( + ) or left untreated (-). An SRSF2 mRNA isoform (ENST00000452355.7) that is a known substrate for NMD was analyzed to confirm NMD inhibition with puromycin treatment. β-actin was included as a loading control. These results were reproduced in one additional independent experiment. b, Quantitation of total CLN3 and SRSF2 mRNA in a. Size markers (basepairs) are shown at right of gel images.
