The Jackson Laboratory for Genomic Medicine, Farmington, CT, 06032, USA.
Department of Genetics and Genome Sciences, University of Connecticut Health Center, Farmington, CT, 06030, USA.
Nat Commun. 2019 Oct 31;10(1):4968. doi: 10.1038/s41467-019-12891-2.
Selectable markers are widely used in transgenesis and genome editing for selecting engineered cells with a desired genotype but the variety of markers is limited. Here we present split selectable markers that each allow for selection of multiple "unlinked" transgenes in the context of lentivirus-mediated transgenesis as well as CRISPR-Cas-mediated knock-ins. Split marker gene segments fused to protein splicing elements called "inteins" can be separately co-segregated with different transgenic vectors, and rejoin via protein trans-splicing to reconstitute a full-length marker protein in host cells receiving all intended vectors. Using a lentiviral system, we create and validate 2-split Hygromycin, Puromycin, Neomycin and Blasticidin resistance genes as well as mScarlet fluorescent proteins. By combining split points, we create 3- and 6-split Hygromycin resistance genes, demonstrating that higher-degree split markers can be generated by a "chaining" design. We adapt the split marker system for selecting biallelically engineered cells after CRISPR gene editing. Future engineering of split markers may allow selection of a higher number of genetic modifications in target cells.
可选择标记物广泛应用于转基因和基因组编辑,用于选择具有所需基因型的工程细胞,但标记物的种类有限。在这里,我们提出了可拆分的选择标记物,它们允许在慢病毒介导的转基因和 CRISPR-Cas 介导的基因敲入中选择多个“非连锁”的转基因。与称为“内含子”的蛋白剪接元件融合的拆分标记基因片段可以分别与不同的转基因载体共同分离,并通过蛋白转剪接重新连接,在接收所有预期载体的宿主细胞中重新构成全长标记蛋白。我们使用慢病毒系统创建并验证了 2 个拆分的潮霉素、嘌呤霉素、新霉素和博来霉素抗性基因以及 mScarlet 荧光蛋白。通过组合拆分点,我们创建了 3 个和 6 个拆分的潮霉素抗性基因,证明可以通过“链状”设计生成更高程度的拆分标记物。我们将拆分标记系统适应于 CRISPR 基因编辑后筛选双等位基因工程细胞。未来的拆分标记物工程可能允许在靶细胞中选择更多的遗传修饰。