The effect of 2′-O-(N-methylcarbamoyl)ethyl (MCE) modification on splice-switching oligonucleotides (SSO) was systematically evaluated. The incorporation of five MCE nucleotides at the 5′-termini of SSOs effectively improved the splice switching effect. In addition, the incorporation of 2′-O-(N-methylcarbamoylethyl)-5-methyl-2-thiouridine (s²T_MCE), a duplex-stabilizing nucleotide with an MCE modification, into SSOs further improved splice switching. These SSOs may be useful for the treatment of genetic diseases associated with splicing errors.

中文翻译：

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2'- O-（N-甲基氨基甲酰基乙基）5-甲基-2-硫尿苷的合成及其在剪接寡核苷酸中的应用

系统地评估了2'- O-（N-甲基氨基甲酰基）乙基（MCE）修饰对剪接转换寡核苷酸（SSO）的影响。在SSO的5'-末端掺入5个MCE核苷酸有效地改善了剪接转换效果。另外，将具有MCE修饰的双链稳定核苷酸2'- O-（N-甲基氨基甲酰基乙基）-5-甲基-2-硫尿苷（s ² T _MCE）并入SSO进一步改善了剪接转换。这些SSO可用于治疗与剪接错误有关的遗传疾病。