Determining off-target effects of splice-switching antisense oligonucleotides using short read RNAseq in neuronally differentiated human induced pluripotent stem cells
Elsa C. Kuijper, Linda van der Graaf, Barry A. Pepers, Mariana Guimarães Ramos, Sylvia Korhorn, Lodewijk J.A. Toonen, Davy Cats, Ronald A.M. Buijsen, Eleni Mina, Hailiang Mei, Willeke van Roon‐Mom
This study uses RNAseq to assess off-target effects of three splice-switching AONs in human cells, identifying splicing changes and showing variability in AON-induced effects.
Human Molecular Genetics · Sep 23, 2025Peer-reviewedGenetics