Miura Hiromi, Quadros Rolen M, Gurumurthy Channabasavaiah B, Ohtsuka Masato
Department of Molecular Life Science, Division of Basic Medical Science and Molecular Medicine, School of Medicine, Tokai University, Kanagawa, Japan.
Center for Matrix Biology and Medicine, Graduate School of Medicine, Tokai University, Kanagawa, Japan.
Nat Protoc. 2018 Jan;13(1):195-215. doi: 10.1038/nprot.2017.153. Epub 2017 Dec 21.
CRISPR/Cas9-based genome editing can easily generate knockout mouse models by disrupting the gene sequence, but its efficiency for creating models that require either insertion of exogenous DNA (knock-in) or replacement of genomic segments is very poor. The majority of mouse models used in research involve knock-in (reporters or recombinases) or gene replacement (e.g., conditional knockout alleles containing exons flanked by LoxP sites). A few methods for creating such models have been reported that use double-stranded DNA as donors, but their efficiency is typically 1-10% and therefore not suitable for routine use. We recently demonstrated that long single-stranded DNAs (ssDNAs) serve as very efficient donors, both for insertion and for gene replacement. We call this method efficient additions with ssDNA inserts-CRISPR (Easi-CRISPR) because it is a highly efficient technology (efficiency is typically 30-60% and reaches as high as 100% in some cases). The protocol takes ∼2 months to generate the founder mice.
基于CRISPR/Cas9的基因组编辑可以通过破坏基因序列轻松生成基因敲除小鼠模型,但其用于创建需要插入外源DNA(基因敲入)或替换基因组片段的模型的效率非常低。研究中使用的大多数小鼠模型涉及基因敲入(报告基因或重组酶)或基因替换(例如,含有由LoxP位点侧翼的外显子的条件性敲除等位基因)。已经报道了一些使用双链DNA作为供体来创建此类模型的方法,但其效率通常为1-10%,因此不适合常规使用。我们最近证明,长单链DNA(ssDNA)作为插入和基因替换的供体非常有效。我们将此方法称为具有ssDNA插入的高效添加-CRISPR(Easi-CRISPR),因为它是一种高效技术(效率通常为30-60%,在某些情况下高达100%)。该方案需要约2个月来生成奠基小鼠。