使用胚胎干细胞/囊胚和 CRISPR/受精卵注射方法创建的具有广泛基因人源化(25-kbp)的活鼠。
Viable Mice with Extensive Gene Humanization (25-kbp) Created Using Embryonic Stem Cell/Blastocyst and CRISPR/Zygote Injection Approaches.
机构信息
Genetic Resource Science, The Jackson Laboratory, Bar Harbor, ME, USA.
Genetically Engineered Models and Services, Charles River Laboratories, Wilmington, USA.
出版信息
Sci Rep. 2018 Oct 9;8(1):15028. doi: 10.1038/s41598-018-33408-9.
Here, we describe an expansion of the typical DNA size limitations associated with CRISPR knock-in technology, more specifically, the physical extent to which mouse genomic DNA can be replaced with donor (in this case, human) DNA at an orthologous locus by zygotic injection. Driving our efforts was the desire to create a whole animal model that would replace 17 kilobase pairs (kbp) of the mouse Bcl2l11 gene with the corresponding 25-kbp segment of human BCL2L11, including a conditionally removable segment (2.9-kbp) of intron 2, a cryptic human exon immediately 3' of this, and a native human exon some 20 kbp downstream. Using two methods, we first carried out the replacement by employing a combination of bacterial artificial chromosome recombineering, classic embryonic stem cell (ESC) targeting, dual selection, and recombinase-driven cassette removal (ESC/Blastocyst Approach). Using a unique second method, we employed the same vector (devoid of its selectable marker cassettes), microinjecting it along with redundant single guide RNAs (sgRNAs) and Cas9 mRNA into mouse zygotes (CRISPR/Zygote Approach). In both instances, we were able to achieve humanization of Bcl2l11 to the extent designed, remove all selection cassettes, and demonstrate the functionality of the conditionally removable, loxP-flanked, 2.9-kbp intronic segment.
在这里,我们描述了一种扩展的 CRISPR 基因敲入技术相关的典型 DNA 大小限制,更具体地说,是通过受精卵注射将供体(在这种情况下是人类)DNA 整合到同源基因座上的程度。我们的努力是为了创建一个完整的动物模型,用人类 BCL2L11 的相应 25-kbp 片段替换小鼠 Bcl2l11 基因的 17 千碱基对(kbp),包括一个条件可去除的内含子 2 (2.9-kbp)片段,这个片段紧接在其 3'端的一个隐秘人类外显子,以及一个大约 20 kbp 下游的天然人类外显子。我们使用两种方法,首先通过细菌人工染色体重组、经典胚胎干细胞(ESC)靶向、双重选择和重组酶驱动的盒式去除(ESC/囊胚方法)进行替换。使用一种独特的第二种方法,我们使用相同的载体(去除其可选择的标记盒),将其与冗余的单指导 RNA(sgRNA)和 Cas9 mRNA 一起微注射到小鼠受精卵中(CRISPR/受精卵方法)。在这两种情况下,我们都能够实现 Bcl2l11 的人类化程度设计,去除所有选择盒,并证明条件可去除、loxP 侧翼、2.9-kbp 内含子片段的功能。