State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Haihe Laboratory of Cell Ecosystem, Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College, Tianjin 300020, China.
Genomics Proteomics Bioinformatics. 2023 Dec;21(6):1221-1236. doi: 10.1016/j.gpb.2022.06.002. Epub 2022 Jun 23.
To achieve the enormous potential of gene-editing technology in clinical therapies, one needs to evaluate both the on-target efficiency and unintended editing consequences comprehensively. However, there is a lack of a pipelined, large-scale, and economical workflow for detecting genome editing outcomes, in particular insertion or deletion of a large fragment. Here, we describe an approach for efficient and accurate detection of multiple genetic changes after CRISPR/Cas9 editing by pooled nanopore sequencing of barcoded long-range PCR products. Recognizing the high error rates of Oxford nanopore sequencing, we developed a novel pipeline to capture the barcoded sequences by grepping reads of nanopore amplicon sequencing (GREPore-seq). GREPore-seq can assess nonhomologous end-joining (NHEJ)-mediated double-stranded oligodeoxynucleotide (dsODN) insertions with comparable accuracy to Illumina next-generation sequencing (NGS). GREPore-seq also reveals a full spectrum of homology-directed repair (HDR)-mediated large gene knock-in, correlating well with the fluorescence-activated cell sorting (FACS) analysis results. Of note, we discovered low-level fragmented and full-length plasmid backbone insertion at the CRISPR cutting site. Therefore, we have established a practical workflow to evaluate various genetic changes, including quantifying insertions of short dsODNs, knock-ins of long pieces, plasmid insertions, and large fragment deletions after CRISPR/Cas9-mediated editing. GREPore-seq is freely available at GitHub (https://github.com/lisiang/GREPore-seq) and the National Genomics Data Center (NGDC) BioCode (https://ngdc.cncb.ac.cn/biocode/tools/BT007293).
为了实现基因编辑技术在临床治疗中的巨大潜力,需要全面评估靶向效率和非预期编辑后果。然而,目前缺乏一种流水线、大规模、经济的方法来检测基因组编辑结果,特别是大片段的插入或缺失。在这里,我们描述了一种通过 CRISPR/Cas9 编辑后的条形码长距离 PCR 产物的 pooled nanopore 测序来高效、准确地检测多种遗传变化的方法。鉴于 Oxford nanopore 测序的高错误率,我们开发了一种新的方法,通过 nanopore 扩增子测序的 grepping reads(GREPore-seq)来捕获条形码序列。GREPore-seq 可以评估非同源末端连接(NHEJ)介导的双链寡脱氧核苷酸(dsODN)插入,其准确性可与 Illumina 下一代测序(NGS)相媲美。GREPore-seq 还揭示了完整的同源定向修复(HDR)介导的大片段基因敲入谱,与荧光激活细胞分选(FACS)分析结果高度相关。值得注意的是,我们在 CRISPR 切割位点发现了低水平的碎片化和全长质粒骨架插入。因此,我们建立了一种实用的工作流程来评估各种遗传变化,包括定量短 dsODN 的插入、长片段的敲入、质粒插入和 CRISPR/Cas9 介导编辑后的大片段缺失。GREPore-seq 可在 GitHub(https://github.com/lisiang/GREPore-seq)和国家基因组学数据中心(NGDC)BioCode(https://ngdc.cncb.ac.cn/biocode/tools/BT007293)上免费获得。