GeneticsRNA BiologyPeer-reviewed
DMD antisense oligonucleotide mediated exon skipping efficiency is affected by flanking intron retention time and target position within the exon
Remko Goossens, Nisha E. Verwey, Yavuz Ariyürek…·Annemieke M Aartsma-Rus
This study evaluates how exon skipping efficiency by antisense oligonucleotides in DMD is influenced by intron retention time and AON position within the exon.
2 citations