Table 1.
List of the known IEIs with CRISPR-Cas technology has been applied in their studies.
| IEIs | Genes | Editing Strategy | Target Cells | Delivery | Refs |
|---|---|---|---|---|---|
| XHIGM | CD40LG | CRISPR/Cas9 mediated integration of a normal copy of the CD40L cDNA | HSCs, patient-derived T cells | AAV6 | (234) |
| XHIGM | CD40LG | CRISPR/Cas9 mediated insertion of a 5’‐truncated CD40LG cDNA within the first intron of the endogenous gene | HSCs, patient-derived T cells | AAV6 | (235) |
| XLA | BTK | CRISPR/Cas9 mediated integration of a codon optimized BTK cDNA into its endogenous locus | HSPCs, Jurkat, K562, Ramos cells, | Electroporation, AAV6 | (236) |
| XLP | SH2D1A | CRISPR/Cas9 or CRISPR/Cas12a drive insertion of SAP cDNA at the first exon of the SH2D1A locus | PrimaryT cells | Nucleofection, AAV6 | (237) |
| SCID | JAK3 | CRISPR/Cas9 nuclease or CRISPR/Cas9 D10A nickase mediated HDR | iPSCs | Nucleofection | (214) |
| SCID | RAG2 | CRISPR/Cas9 mediated multiplex HDR | HSPCs | AAV6 | (215) |
| X-SCID | IL2RG | CRISPR/Cas9 mediated integration of a cDNA into the endogenous locus | LT-HSCs | AAV6 | (216) |
| X-SCID | IL2RG | CRISPR/Cas9 mediated knock-in of a corrective IL2RG cDNA into the affected locus | HSPC | AAV6 | (217) |
| X-SCID | IL2RG | CRISPR/Cas9 mediated disruption of IL2Rg | Rabbit embryos | Microinjection of gRNA and Cas9 mRNA | (218) |
| SCID | IL2RG, RAG2 | CRISPR/Cas9 mediated gene knockout | Rat zygotes | Electroporation of gRNA and Cas9 mRNA | (219) |
| SCID | IL2RG, RAG2 | CRISPR/Cas9 mediated double knockout | Mice zygotes | Microinjection of gRNA and Cas9 mRNA | (220) |
| SCID | IL2RG | CRISPR/Cas9 mediated gene knockout | pig embryos | Microinjection of gRNA and Cas9 mRNA, nucleofection | (221–223) |
| SCID | IL2RG | CRISPR/Cas9 mediated gene knockout | Hamster embryos | pronuclear injection of gRNA and Cas9 mRNA | (224) |
| IPEX | FOXP3 | CRISPR/Cas9 mediated integration of FOXP3 cDNA into the endogenous locus | Patient derived HSPCs | AAV6 | (225) |
| XMEN | MAGT1 | CRISPR/Cas9 mediated HDR to insert a correct MAGT1 cDNA into the endogenous locus | HSPCs | AAV6 | (226) |
| X-CGD | CYBB | CRISPR/Cas9 mediated HDR for gene repair using ssODN | Patient derived HSPCs | Electroporation | (227) |
| X-CGD | CYBB | CRISPR/Cas9 mediated insertion of CYBB cDNA at endogenous locus | Patient derived HSPCs | AAV6 | (228) |
| X-CGD | CYBB | CRISPR/Cas9 mediated gene replacement and insertion | Patient-specific iPSCs | nucleofection | (229) |
| X-CGD | CYBB | CRISPR/Cas9 mediated gene knockout | Mouse zygotes | microinjection of gRNA and Cas9 mRNA | (230) |
| WAS | WAS | CRISPR/Cas9 mediated HDR for integrating a full-length cDNA into endogenous locus | Patient derived HSPCs, LT-HSCs | AAV6, electroporation | (231) |
| WAS | WAS | CRISPR/Cas9 mediated gene knockout | Rabbit embryos | microinjection of Cas9 mRNA and a pair of sgRNAs | (232) |
| WAS | WAS | CRISPR/Cas9 mediated gene knockout | primary T cells | Vapor nanobubble (VNB) photoporation | (233) |
XHIGM, X-linked hyper-IgM syndrome; XLA, X-linked agammaglobulinemia; XLP, X-Linked Lymphoproliferative Disease; SCID, Severe combined immunodeficiency; X- SCID, X-linked severe combined immunodeficiency; IPEX, Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome; XMEN, X-linked immunodeficiency with magnesium defect, EBV infection, and neoplasia; X-CGD, X-linked chronic granulomatous disease; WAS: Wiskott-Aldrich syndrome; HSCs, Hematopoietic stem cell; HSPCs, hematopoietic stem and progenitor cells; LT-HSCs, long-term hematopoietic stem cells; iPSCs, induced pluripotent stem cells; MSCs, mesenchymal stem cells.