Division of Tumor Biology and Immunology, The Netherlands Cancer Institute, Plesmanlaan 121, 1066 CX Amsterdam, The Netherlands.
Department of Clinical Genetics, VU University Medical Center, Van der Boechorststraat 7, 1081 BT Amsterdam, The Netherlands.
Nucleic Acids Res. 2018 Apr 6;46(6):2945-2955. doi: 10.1093/nar/gky076.
Single-stranded oligodeoxyribonucleotide (ssODN)-mediated repair of CRISPR/Cas9-induced DNA double-strand breaks (DSB) can effectively be used to introduce small genomic alterations in a defined locus. Here, we reveal DNA mismatch repair (MMR) activity is crucial for efficient nucleotide substitution distal from the Cas9-induced DNA break when the substitution is instructed by the 3' half of the ssODN. Furthermore, protecting the ssODN 3' end with phosphorothioate linkages enhances MMR-dependent gene editing events. Our findings can be exploited to optimize efficiencies of nucleotide substitutions distal from the DSB and imply that oligonucleotide-mediated gene editing is effectuated by templated break repair.
单链寡脱氧核苷酸(ssODN)介导的 CRISPR/Cas9 诱导的 DNA 双链断裂(DSB)修复可有效用于在特定基因座引入小的基因组改变。在这里,我们揭示了当取代由 ssODN 的 3' 半部分指导时,DNA 错配修复(MMR)活性对于 Cas9 诱导的 DNA 断裂远处的有效核苷酸取代至关重要。此外,用硫代磷酸酯键保护 ssODN 的 3' 端可增强 MMR 依赖性基因编辑事件。我们的发现可用于优化 DSB 远处核苷酸取代的效率,并暗示寡核苷酸介导的基因编辑是通过模板断裂修复实现的。