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Table 3 Registered targets in 81 clinical trials on genome editing in human participants from WHO ICTRP

From: Inadequate reporting quality of registered genome editing trials: an observational study

WHO ICTRP targets No. (%) of trials
Single target
 BCL11A gene 5 (6.2)
 CCR5 gene 10 (12.3)
 CEP290 gene 1 (1.2)
 CISH gene 2 (2.5)
 Factor IX gene 2 (2.5)
 HBB gene 1 (1.2)
 HPV oncogenes E6 or E7 3 (3.7)
 IDS gene 1 (1.2)
 IDUA gene 1 (1.2)
 PDCD1 gene 15 (18.5)
 CAR T cells therapy targets: 23 (28.4)
  BCMA 3 (3.7)
  CD7 2 (2.5)
  CD19 12 (14.8)
  CD22 1 (1.2)
  CD70 2 (2.5)
  CD123 2 (2.5)
  CS1 1 (1.2)
Multiple targets
 PDCD1, NY-ESO-1, TRAC 1 (1.2)
 PDCD1, mesothelin (CARTs) 2 (2.5)
 PDCD1, CD19 (CARTs) 2 (2.5)
 PDCD1, MUC1 (CARTs) 3 (3.7)
 CCR5, CD4 (CARTs) 1 (1.2)
 CD19 and CD20/CD22 (CARTs) 1 (1.2)
 CD19 and HPK1 (CARTs) 1 (1.2)
 CD19, CD52, TRAC (CARTs) 1 (1.2)
 CD19, B2M, TRAC (CARTs) 1 (1.2)
 IL13 zetakine/HyTk (CARTs) 1 (1.2)
 CD7 and CD28 (CARTs) 1 (1.2)
Not stated 2 (2.5)
  1. Abbreviations: BCL11A Mouse B cell lymphoma factor 11A, BCMA B cell maturation antigen, B2M Beta-2-microglobulin, CAR Chimeric antigen receptor, CARTs Chimeric antigen receptor T cells, CCR5 Chemokine receptor 5, CD Cluster of differentiation, CEP290 Centrosomal protein 290, CISH Cytokine-induced SH2 protein, HBB Hemoglobin subunit beta, HPK1 Hematopoietic progenitor kinase 1, HPV Human papillomavirus, IDS Iduronate 2-sulfatase, IDUA α-L-iduronidase, MUC1 Mucin 1, cell surface associated, NY-ESO-1 New York esophageal squamous cell carcinoma 1, PDCD1 Programmed cell death 1, TRAC T cell receptor alpha chain, WHO ICTRP World Health Organization International Clinical Trial Registry Platform