Chapter 18 - Genetic modification of mice using CRISPR-Cas9: Best practices and practical concepts explained

Document Type

Article

Publication Date

2024

JAX Source

Rigor and Reproducibility in Genetics and Genomics: Academic Press; 2024. p. 425-52.

DOI

https://doi.org/10.1016/B978-0-12-817218-6.00018-8

Grant

We acknowledge support from the National Institutes of Health under Award Number R01 CA265978 (VH), and the Director’s Innovation Fund at JAX (JAX-DIF-FY17).

Abstract

The development of precision targetable nucleases has led to a massive acceleration in creating ge- netically modified mice and other species [1–6]. This began more than a decade ago with Zinc Finger Nucleases (ZFNs) [7], followed by TALENs (Transcription Activator-Like Effector Nucleases) [8–10], and then CRISPR-Cas9 (Clustered Regularly Interspaced Short Palindromic Repeats with CRISPR- associated effector protein 9) [11,12]. At present, RNA-guided CRISPR-Cas9 is the most affordable and straightforward to design, construct, and implement. This accessibility, combined with its gener- ally high degree of targeting efficiency, has pushed CRISPR-Cas9 to the forefront of gene-editing methods. Regardless of its relative simplicity, the complexity of the resulting nuclease-derived genetic modifications, including the modified organism’s phenotype, should not be underestimated [13–15].

Herein, we outline our experience using CRISPR-Cas9 to precisely and directly engineer mouse zygotes, focusing on the general methodology and screening used to characterize the resulting alleles. These screening strategies are simple, straightforward, and reproducible. While the focus of this chap- ter is on CRISPR-modified alleles generated in mice, these screening regimes can be applied to other organisms and to the characterization of genetic modifications resulting from ZFNs, TALENs, or any other gene-editing technology.

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