Department of Endocrine Neoplasia and Hormonal Disorders, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Department of Genetics, University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Genes (Basel). 2020 Jun 8;11(6):628. doi: 10.3390/genes11060628.
Although the Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR)/ CRISPR associated protein 9 (Cas9) technique has dramatically lowered the cost and increased the speed of generating genetically engineered mice, success depends on using guide RNAs and donor DNAs which direct efficient knock-out (KO) or knock-in (KI). By Sanger sequencing DNA from blastocysts previously injected with the same CRISPR components intended to produce the engineered mice, one can test the effectiveness of different guide RNAs and donor DNAs. We describe in detail here a simple, rapid (three days), inexpensive protocol, for amplifying DNA from blastocysts to determine the results of CRISPR point mutation KIs. Using it, we show that (1) the rate of KI seen in blastocysts is similar to that seen in mice for a given guide RNA/donor DNA pair, (2) a donor complementary to the variable portion of a guide integrated in a more all-or-none fashion, (3) donor DNAs can be used simultaneously to integrate two different mutations into the same locus, and (4) by placing silent mutations about every 6 to 10 bp between the Cas9 cut site and the desired mutation(s), the desired mutation(s) can be incorporated into genomic DNA over 30 bp away from the cut at the same high efficiency as close to the cut.
尽管簇状规律间隔短回文重复序列 (CRISPR)/CRISPR 相关蛋白 9 (Cas9) 技术极大地降低了生成基因工程小鼠的成本并提高了速度,但成功取决于使用指导 RNA 和供体 DNA,这些指导 RNA 和供体 DNA 可直接进行有效的敲除 (KO) 或敲入 (KI)。通过对先前用相同的 CRISPR 成分注射的胚胎进行桑格测序 DNA,以产生工程小鼠,可以测试不同的指导 RNA 和供体 DNA 的有效性。我们在这里详细描述了一种简单、快速 (三天)、廉价的方案,用于扩增来自胚胎的 DNA,以确定 CRISPR 点突变 KI 的结果。使用该方案,我们表明:(1)对于给定的指导 RNA/供体 DNA 对,在胚胎中观察到的 KI 率与在小鼠中观察到的 KI 率相似;(2)供体与整合在更全有或全无方式中的指导 RNA 的可变部分互补;(3)可以同时使用供体将两个不同的突变整合到同一个基因座中;(4)通过在 Cas9 切割位点和所需突变之间每隔 6 到 10 个 bp 放置沉默突变,所需突变可以以与靠近切割相同的高效率整合到基因组 DNA 中,距离切割超过 30 bp。