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. 2022 Sep 14;13:932715. doi: 10.3389/fimmu.2022.932715

Figure 3.

Figure 3

CRISPR-Cas9 gene editing to model IEIs/PIRDs. The CRISPR-Cas9 system consists of the Cas9 protein in complex with a targeting gRNA, forming a ribonucleoprotein (RNP). Specificity is conferred through various mechanisms including the sgRNA and presence of the PAM sequence. The Cas9 opens and cuts the targeted sequence on both strands to generate double-strand breaks (DSBs). This activates DNA repair pathways (NHEJ and HDR) in cells and results in modifications at a target locus. The NHEJ pathway can be used for scenarios in which genetic variants result in the loss of expression of an encoded protein, while HDR can be employed for introduction of missense mutations. IEIs, inborn errors of immunity; PIRDs, primary immune regulatory disorders; sgRNA, single-guide RNA; PAM, protospacer adjacent motif; NHEJ, non-homologous end joining; HDR, homology-directed repair. Adapted from “CRISPR/Cas9 Gene Editing”, by BioRender.com (2022). Retrieved from https://app.biorender.com/biorender-templates/t-5f873df466346900a43c6db1-crisprcas9-gene-editing.