In silico and in vitro evaluation of exonic and intronic off-target effects form a critical element of therapeutic ASO gapmer optimization
利用计算机模拟和体外实验评估外显子和内含子脱靶效应是治疗性反义寡核苷酸（ASO）间隙体优化过程中的关键要素。
期刊:Nucleic Acids Research
影响因子:13.1
doi:10.1093/nar/gkv857
Kamola, Piotr J; Kitson, Jeremy D A; Turner, Gemma; Maratou, Klio; Eriksson, Sofie; Panjwani, Aliza; Warnock, Linda C; Douillard Guilloux, Gaelle A; Moores, Kitty; Koppe, Emma L; Wixted, William E; Wilson, Paul A; Gooderham, Nigel J; Gant, Timothy W; Clark, Kenneth L; Hughes, Stephen A; Edbrooke, Mark R; Parry, Joel D
