Table 1.
Summary of therapeutically implicated loci
| Gene/virus | Target | Coordinates (hg19) | Disease | Repair | Refs. |
| Gene | |||||
| ALCAM | All exons | chr3:105085753–105295744 | HIV-1 infection | NHEJ | 63 |
| BCL11A | Enhancer | chr2:60722309–60722472 | β-Hemoglobinopathies | NHEJ | 64 |
| B2M | All exons | chr15:45003686–45010357 | Hypoimmunogenic cells for transplantation | NHEJ | 65 |
| CCR5 | Exon 1 | chr3:46414395–46415452 | HIV infection | NHEJ | 65 |
| CEP290 | Intron 26 | chr12:88494861–88495060 | Leber’s congenital amaurosis type 10 | NHEJ | 2, 66 |
| CXCR4 | Exon 2 | chr2:136872440–136873482 | HIV-1 infection | NHEJ | 67 |
| HLA-A | Exon 3 | chr6:29911046–29911320 | Hypoimmunogenic cells for transplantation | NHEJ | 68 |
| PCSK9 | Exons 1–2 | chr1:55505512–55509707 | Cardiovascular disease | NHEJ | 69 |
| PDCD1 | Exon 1 | chr2:242800916–242800990 | Tumor immunotherapy | NHEJ/HDR | 70, 71 |
| PSIP1 | Exons 2, 12, 14 | chr9:15468629–15510186 | HIV-1 infection | NHEJ | 72 |
| TPST2 | All exons | chr22:26921712–26992681 | HIV-1 infection | NHEJ | 63 |
| TRAC,TRBC1, TRBC2 | Exon 1 | chr14:23016448–23016719; chr7:142498738–142499111; chr7:142498726–142499111 | T cell immunotherapy | NHEJ | 73–75 |
| SLC35B2 | All exons | chr6:44221838–44225308 | HIV-1 infection | NHEJ | 63 |
| ADA | Intron 6/exon 7 | chr20:43251649–43251819 | Adenosine deaminase severe combined immunodeficiency (ADA-SCID) | HDR | 76 |
| ALB | Intron 1 | chr4:74270125–74270832 | Lysosomal storage disease, hemophilia A, B | HDR | 77 |
| CFTR | Exon 10 | chr7:117199519–117199709 | Cystic fibrosis | HDR | 78 |
| COL7A1 | Exons 2, 3, 14, 15, 54, 117 | chr3:48602217–48631981 | Epidermolysis bullosa | HDR | 79 |
| CYBB | Exon 7 | chrX:37658207–37658337 | X-linked chronic granulomatous disease | HDR | 80 |
| DMD | Exons/intron 45–55 | chrX:31533884–32250573 | Duchenne’s muscular dystrophy | HDR | 81, 82 |
| FANCC | Intron 4 | chr9:97934216–97934415 | Fanconi anemia | HDR | 83 |
| F9 | Intron 1 | chrX:138613012–138619169 | Hemophilia B | HDR | 84 |
| FAH | Exon 8/intron 8 | chr15:80464492–80464690 | Hereditary tyrosinemia type I | HDR | 85 |
| HBB | Exon 1 | chr11:5248162–5248251 | Sickle cell disease | HDR | 86, 87 |
| IL2RG | Exon 5 | chrX:70329079–70329240 | X-linked severe combined immunodeficiency (X-SCID) | HDR | 88 |
| SERPINA1 | Intron 4/exon 5 | chr14:94844848–94845047 | α-1-Antitrypsin deficiency | HDR | 89 |
| Virus | |||||
| Cytomegalovirus | Viral genome | — | Congenital defects, disease in immuno-compromised individuals | NHEJ | 49 |
| Epstein bar virus | Viral genome | — | Infectious mononucleosis, malignancies | NHEJ | 49 |
| Hepatitis B virus | Viral genome | — | Hepatitis B | NHEJ | 51, 90–92 |
| Herpes simplex virus type 1 | Viral genome | — | Cold sores, keratitis | NHEJ | 49 |
| HIV-1 | Viral genome (LTR) | — | HIV-1 infection | NHEJ | 50 |
| Human papilloma virus | E6–E7 oncogenes | — | Cervical carcinoma | NHEJ | 93 |
| JC virus | T antigen | — | Progressive multifocal leukoencephalopathy | NHEJ | 52 |