Table 1.

Summary of therapeutically implicated loci

Gene/virusTargetCoordinates (hg19)DiseaseRepairRefs.
Gene
 ALCAMAll exonschr3:105085753–105295744HIV-1 infectionNHEJ63
 BCL11AEnhancerchr2:60722309–60722472β-HemoglobinopathiesNHEJ64
 B2MAll exonschr15:45003686–45010357Hypoimmunogenic cells for transplantationNHEJ65
 CCR5Exon 1chr3:46414395–46415452HIV infectionNHEJ65
 CEP290Intron 26chr12:88494861–88495060Leber’s congenital amaurosis type 10NHEJ2, 66
 CXCR4Exon 2chr2:136872440–136873482HIV-1 infectionNHEJ67
 HLA-AExon 3chr6:29911046–29911320Hypoimmunogenic cells for transplantationNHEJ68
 PCSK9Exons 1–2chr1:55505512–55509707Cardiovascular diseaseNHEJ69
 PDCD1Exon 1chr2:242800916–242800990Tumor immunotherapyNHEJ/HDR70, 71
 PSIP1Exons 2, 12, 14chr9:15468629–15510186HIV-1 infectionNHEJ72
 TPST2All exonschr22:26921712–26992681HIV-1 infectionNHEJ63
 TRAC,TRBC1, TRBC2Exon 1chr14:23016448–23016719; chr7:142498738–142499111; chr7:142498726–142499111T cell immunotherapyNHEJ7375
 SLC35B2All exonschr6:44221838–44225308HIV-1 infectionNHEJ63
 ADAIntron 6/exon 7chr20:43251649–43251819Adenosine deaminase severe combined immunodeficiency (ADA-SCID)HDR76
 ALBIntron 1chr4:74270125–74270832Lysosomal storage disease, hemophilia A, BHDR77
 CFTRExon 10chr7:117199519–117199709Cystic fibrosisHDR78
 COL7A1Exons 2, 3, 14, 15, 54, 117chr3:48602217–48631981Epidermolysis bullosaHDR79
 CYBBExon 7chrX:37658207–37658337X-linked chronic granulomatous diseaseHDR80
 DMDExons/intron 45–55chrX:31533884–32250573Duchenne’s muscular dystrophyHDR81, 82
 FANCCIntron 4chr9:97934216–97934415Fanconi anemiaHDR83
 F9Intron 1chrX:138613012–138619169Hemophilia BHDR84
 FAHExon 8/intron 8chr15:80464492–80464690Hereditary tyrosinemia type IHDR85
 HBBExon 1chr11:5248162–5248251Sickle cell diseaseHDR86, 87
 IL2RGExon 5chrX:70329079–70329240X-linked severe combined immunodeficiency (X-SCID)HDR88
 SERPINA1Intron 4/exon 5chr14:94844848–94845047α-1-Antitrypsin deficiencyHDR89
Virus
 CytomegalovirusViral genomeCongenital defects, disease in immuno-compromised individualsNHEJ49
 Epstein bar virusViral genomeInfectious mononucleosis, malignanciesNHEJ49
 Hepatitis B virusViral genomeHepatitis BNHEJ51, 9092
 Herpes simplex virus type 1Viral genomeCold sores, keratitisNHEJ49
 HIV-1Viral genome (LTR)HIV-1 infectionNHEJ50
 Human papilloma virusE6–E7 oncogenesCervical carcinomaNHEJ93
 JC virusT antigenProgressive multifocal leukoencephalopathyNHEJ52