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Table 2 Characteristics of genome editing methodologies used in 81 trials registered in WHO ICTRP

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

Genome editing characteristics No. (%) of trials
Platform:
 ZFN 17 (21.0)
 TALENa 1 (1.2)
 CRISPR/Cas9 29 (35.8)
 TALEN and CRISPR/Cas9b 1 (1.2)
 Not stated 33 (40.7)
Testing method:
 In vivoc 9 (11.1)
 Ex vivo 72 (88.9)
Disease applications:
 HIV infection and AIDS 12 (14.8)
 Neoplasms 57 (70.4)
 Hematological disordersd 8 (9.9)
 Metabolic diseasese 2 (2.5)
 Eye diseasesf 2 (2.5)
Edited cells:
 T cellsg 24 (29.6)
 CAR T cells 37 (45.7)
 Tumor infiltrating lymphocytes 2 (2.5)
 Stem or progenitor cellsh 8 (9.9)
 Hepatocytes 3 (3.7)
 Epithelial cells 3 (3.7)
 Human embryosi 1 (1.2)
 Not stated 3 (3.7)
Delivery:
 Adenovirus 1 (1.2)
 AAV 4 (4.9)
 Lentivirus 2 (2.5)
 Lentiviral and electroporation 3 (3.7)
 Plasmid 1 (1.2)
 mRNA 4 (4.9)
 Intratumoral injection 1 (1.2)
 Not stated 65 (80.2)
  1. Abbreviations: AAV Adeno-associated virus, CAR Chimeric antigen receptor, CRISPR Clustered regularly interspaced short palindromic repeats, TALEN Transcription activator-like effector nuclease, WHO ICTRP World Health Organization International Clinical Trial Registry Platform, ZFN Zinc finger nuclease
  2. aTrial conducted in China, using TALEN in vivo (suppository) in the treatment of HPV-related cervical intraepithelial neoplasia (CIN) (NCT03226470)
  3. bTrial conducted in China, using CRISPR/Cas9 and TALEN in vivo (plasmids in gel) in the treatment of HPV-associated CIN (NCT03057912)
  4. cHPV-related malignant neoplasm was an investigated condition in 3 trials testing only ZFN, TALEN, or TALEN and CRISPR/Cas9 (NCT02800369, NCT03226470, NCT03057912); hemophilia B or mucopolysaccharidosis in 4 trials using ZFN platform (NCT02695160, EUCTR2017-004805-42-GB, NCT03041324, NCT02702115); and different diseases of the visual system in the remaining 2 trials (and) – one used CRISPR/Cas9 (NCT04560790) and another did not specify the nuclear platform used (NCT03872479)
  5. dIncluding beta-thalassemia, sickle cell disease, and hemophilia B
  6. eReferring to mucopolysaccharidosis I and II
  7. f “Blindness, Leber congenital amaurosis 10, vision disorders, hereditary eye diseases, congenital eye disorders, retinal disease/degeneration” noted in NCT03872479; “viral keratitis, blindness, Herpes simplex virus infection” recorded in NCT04560790
  8. gRecorded terms as CD4 + T cells, Epstein Barr Virus (EBV)-specific cytotoxic T lymphocytes (CTLs), or only T cells
  9. hIncluding hematopoietic stem/progenitor cells (HSPCs), hematopoietic stem cells (HSCs), and induced hematopoietic stem cells (iHSCs)
  10. iCRISPR embryo editing by Chinese scientist He Jiankui, which later resulted in birth (ChiCTR1800019378)