Department of Mathematical and Life Sciences, Graduate School of Science, Hiroshima University, Hiroshima, Japan.
Nat Protoc. 2016 Jan;11(1):118-33. doi: 10.1038/nprot.2015.140. Epub 2015 Dec 17.
Programmable nucleases enable engineering of the genome by utilizing endogenous DNA double-strand break (DSB) repair pathways. Although homologous recombination (HR)-mediated gene knock-in is well established, it cannot necessarily be applied in every cell type and organism because of variable HR frequencies. We recently reported an alternative method of gene knock-in, named the PITCh (Precise Integration into Target Chromosome) system, assisted by microhomology-mediated end-joining (MMEJ). MMEJ harnesses independent machinery from HR, and it requires an extremely short homologous sequence (5-25 bp) for DSB repair, resulting in precise gene knock-in with a more easily constructed donor vector. Here we describe a streamlined protocol for PITCh knock-in, including the design and construction of the PITCh vectors, and their delivery to either human cell lines by transfection or to frog embryos by microinjection. The construction of the PITCh vectors requires only a few days, and the entire process takes ∼ 1.5 months to establish knocked-in cells or ∼ 1 week from injection to early genotyping in frog embryos.
可编程核酸酶通过利用内源性 DNA 双链断裂 (DSB) 修复途径来实现基因组工程。尽管同源重组 (HR) 介导的基因敲入已经得到很好的建立,但由于 HR 频率的不同,它不一定适用于每种细胞类型和生物体。我们最近报道了一种称为 PITCh(靶向染色体精确整合)系统的基因敲入替代方法,该方法由微同源介导的末端连接 (MMEJ) 辅助。MMEJ 利用 HR 的独立机制,并且它需要用于 DSB 修复的极短同源序列(5-25bp),从而实现精确的基因敲入,并且供体载体更容易构建。在这里,我们描述了一种简化的 PITCh 敲入方案,包括 PITCh 载体的设计和构建,以及通过转染将其递送至人细胞系或通过显微注射递送至青蛙胚胎。构建 PITCh 载体只需要几天的时间,整个过程需要大约 1.5 个月的时间才能建立敲入细胞,或者从注射到青蛙胚胎的早期基因分型需要大约 1 周的时间。
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