Various diseases, including cancer, are caused by genetic mutations. A 5′-tailed duplex (TD) DNA, consisting of a long single-stranded (ss) editor DNA and a short (∼35-base) ss assistant oligodeoxyribonucleotide, can introduce a base-substitution in living cells and thus correct mutated genes. Previously, several hundred-base DNAs were employed as the editor DNAs. In this study, 5′-TDs were prepared from various editor DNAs with different lengths and examined for their gene correction abilities, using plasmid DNA bearing a mutated copepod green fluorescent protein (copGFP) gene, in human cells. High-throughput analysis was performed by the reactivated fluorescence of the wild-type protein encoded by the corrected gene as the indicator. The analysis revealed that 5′-TDs with ∼100-base ss editor DNAs enabled gene editing at least as efficiently as those with longer editor DNAs. Moreover, the antisense strand was more effective as the editor than the sense strand, in contrast to the 5′-TDs with longer editor strands. These results indicated that the 5′-TD fragments with shorter editor strands than those used in previous studies are useful nucleic acids for gene correction.

包括癌症在内的各种疾病都是由基因突变引起的。由长单链 (ss) 编辑 DNA 和短（~35 个碱基）ss 辅助寡脱氧核糖核苷酸组成的 5' 尾双链体 (TD) DNA 可以在活细胞中引入碱基替换，从而纠正突变基因. 以前，使用数百个碱基的 DNA 作为编辑器 DNA。在这项研究中，使用带有突变桡足类绿色荧光蛋白（copGFP) 基因，在人类细胞中。以校正基因编码的野生型蛋白的再激活荧光为指标进行高通量分析。分析表明，具有约 100 个碱基的 ss 编辑器 DNA 的 5'-TD 使基因编辑至少与具有更长编辑器 DNA 的那些一样有效。此外，与具有更长编辑器链的 5'-TD 相比，反义链作为编辑器比有义链更有效。这些结果表明，编辑链比先前研究中使用的更短的 5'-TD 片段是用于基因校正的有用核酸。