Abstract Targeted DNA editing has great potential to cure some genetic diseases; however, the use of artificial nucleases such as CRISPR-Cas9 and TALEN in gene therapy can potentially cause severe side effects due to off-target DNA cleavages. Single-stranded (ss) DNAs and 5'-tailed duplexes (TDs) can achieve target base substitutions when introduced without artificial nucleases into cultured cells and mouse liver. In this study, ss DNA and TD were separately co-introduced into human U2OS cells, together with a target plasmid DNA bearing an inactivated lacZα gene, and the gene correction efficiencies were compared. Unlike the genes examined in previous studies, ss DNA and TD showed similar efficiencies. Therefore, ss DNAs might be as useful as TD for gene correction, depending on the target sequence.

中文翻译：

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lacZα DNA 序列转换中的单链 DNA 与有尾双链体

摘要 靶向 DNA 编辑在治疗某些遗传疾病方面具有巨大潜力；然而，在基因治疗中使用 CRISPR-Cas9 和 TALEN 等人工核酸酶可能会由于脱靶 DNA 裂解而导致严重的副作用。单链 (ss) DNA 和 5' 尾双链体 (TD) 在不使用人工核酸酶的情况下引入培养细胞和小鼠肝脏时，可以实现目标碱基替换。本研究将ss DNA和TD分别与携带失活lacZα基因的靶质粒DNA共同导入人U2OS细胞，比较基因校正效率。与之前研究中检查的基因不同，ss DNA 和 TD 显示出相似的效率。因此，根据目标序列，ss DNA 可能与 TD 一样用于基因校正。