Single-stranded DNA versus tailed duplex in sequence conversion of lacZα DNA.
Hidehiko KawaiKento SatoWataru ShirahamaTetsuya SuzukiHiroyuki KamiyaPublished in: Nucleosides, nucleotides & nucleic acids (2020)
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.
Keyphrases
- circulating tumor
- crispr cas
- cell free
- single molecule
- genome editing
- nucleic acid
- genome wide
- gene therapy
- induced apoptosis
- endothelial cells
- escherichia coli
- oxidative stress
- circulating tumor cells
- gene expression
- risk assessment
- cell cycle arrest
- genome wide identification
- signaling pathway
- cell death
- cancer therapy