In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization.
Kamola PJ, Kitson JD, Turner G, Maratou K, Eriksson S, Panjwani A, Warnock LC, Douillard Guilloux GA, Moores K, Koppe EL, Wixted WE, Wilson PA, Gooderham NJ, Gant TW, Clark KL, Hughes SA, Edbrooke MR, Parry JD.
Kamola PJ, et al. Among authors: douillard guilloux ga.
Nucleic Acids Res. 2015 Oct 15;43(18):8638-50. doi: 10.1093/nar/gkv857. Epub 2015 Sep 3.
Nucleic Acids Res. 2015.
PMID: 26338776
Free PMC article.