Mays Joseph C, Mei Sally, Kogenaru Manjunatha, Quysbertf Helberth M, Bosco Nazario, Zhao Xin, Bianchi Joy J, Goldberg Aleah, Kidiyoor Gururaj Rao, Holt Liam J, Fenyö David, Davoli Teresa
Institute for Systems Genetics and Department of Biochemistry and Molecular Pharmacology, New York University Grossman School of Medicine, New York, NY 10016, USA.
Institute for Systems Genetics and Department of Biochemistry and Molecular Pharmacology, New York University Grossman School of Medicine, New York, NY 10016, USA. Current Address: Volastra Therapeutics, New York, NY 10027, USA.
bioRxiv. 2024 Sep 29:2023.09.08.555746. doi: 10.1101/2023.09.08.555746.
Investigating chromosomal instability and aneuploidy within tumors is essential for understanding tumorigenesis and developing diagnostic and therapeutic strategies. Single-cell DNA sequencing technologies have enabled such analyses, revealing aneuploidies specific to individual cells within the same tumor. However, it has been difficult to scale the throughput of these methods to detect rare aneuploidies while maintaining high sensitivity. To overcome this deficit, we developed KaryoTap, a method combining custom targeted DNA sequencing panels for the Tapestri platform with a computational framework to enable detection of chromosome- and chromosome arm-scale aneuploidy (gains or losses) and copy number neutral loss of heterozygosity in all human chromosomes across thousands of single cells simultaneously. KaryoTap allows detecting gains and losses with an average accuracy of 83% for arm events and 91% for chromosome events. Importantly, together with chromosomal copy number, our system allows us to detect barcodes and gRNAs integrated into the cells' genome, thus enabling pooled CRISPR- or ORF-based functional screens in single cells. As a proof of principle, we performed a small screen to expand the chromosomes that can be targeted by our recently described CRISPR-based KaryoCreate system for engineering aneuploidy in human cells. KaryoTap will prove a powerful and flexible approach for the study of aneuploidy and chromosomal instability in both tumors and normal tissues.
研究肿瘤内的染色体不稳定性和非整倍体对于理解肿瘤发生以及制定诊断和治疗策略至关重要。单细胞DNA测序技术使得此类分析成为可能,揭示了同一肿瘤内个别细胞特有的非整倍体情况。然而,要扩大这些方法的通量以检测罕见的非整倍体同时又保持高灵敏度一直很困难。为了克服这一不足,我们开发了KaryoTap,这是一种将用于Tapestri平台的定制靶向DNA测序面板与一个计算框架相结合的方法,能够同时在数千个单细胞中检测所有人类染色体上的染色体和染色体臂水平的非整倍体(增加或减少)以及杂合性的拷贝数中性缺失。KaryoTap检测增加和减少的平均准确率对于臂水平事件为83%,对于染色体水平事件为91%。重要的是,连同染色体拷贝数一起,我们的系统使我们能够检测整合到细胞基因组中的条形码和gRNA,从而能够在单细胞中进行基于CRISPR或ORF的功能筛选。作为原理验证,我们进行了一个小规模筛选,以扩展可被我们最近描述的基于CRISPR的KaryoCreate系统靶向的染色体,该系统用于在人类细胞中构建非整倍体。KaryoTap将被证明是一种用于研究肿瘤和正常组织中非整倍体和染色体不稳定性的强大且灵活的方法。