Table 5.
Applications of the CRISPR/Cas9 system used in clinical trials.
| Disease | Target Gene | Type of Edit | Phase |
|---|---|---|---|
| Beta-thalassemia | Hemoglobin Subunit Beta (HBB) | Gene correction | I & II |
| Beta-thalassemia | BAF Chromatin Remodeling Complex Subunit 11A (BCL11A) | Gene disruption | II & III |
| Transfusion-Dependent β-Thalassemia | BAF Chromatin Remodeling Complex Subunit 11A (BCL11A) | Gene disruption | III |
| Acute Lymphoblastic Leukemia | CD19/CD52 molecule (CD19/CD52), T-Cell Receptor alpha & beta locus (TCRαβ) | Gene KO | I |
| Acute Lymphoblastic Leukemia | T-Cell Receptor alpha locus (TCRα) | Gene KO | I & II |
| Acute Lymphoblastic Leukemia | T-Cell Receptor (TRC), anti-CD19 CAR | Gene KO & KI | I & II |
| Acute Myeloid Leukemia | CD38/CD33 molecule (CD38/CD33) | Gene KO | I & II |
| Hepatocellular Carcinoma | Programmed Cell Death 1 (PD-1) | Gene KO | I |
| B-Cell Malignancies | T-Cell Receptor (TCR) & Beta-2-Microglobulin (B2M) | Gene disruption | I & II |
| B-Cell Non-Hodgkin Lymphoma | CD19 molecule (CD19), Programmed Cell Death 1 (PD-1), T-Cell Receptor (TCR) | Gene KO/KI | I |
| Billiary Tract Cancer | TGF-β receptor (TGFβR) | Gene KO | I |
| Prostate Cancer | Programmed Cell Death 1 (PD-1) | Gene KO | I & II |
| Esophageal Cancer | Programmed Cell Death 1 (PD-1) | Gene KO | I |
| Gastro-Intestinal Cancer | Cytokine inducible SH2 containing protein (CISH) | Gene KO | I & II |
| Human papillomavirus related cervical cancer | Human papillomavirus types 16 & 18 (HPV16 & HPV18 E6/E7) | Gene KO | I |
| Metastatic Non-small Cell Lung Cancer | Programmed Cell Death 1 (PD-1) | Gene disruption | I |
| Multiple Myeloma | T-Cell Receptor alpha & beta locus (TCRαβ) & Programmed Cell Death 1 (PD-1) | Gene KO | I |
| Nasopharyngeal Carcinoma | Programmed Cell Death 1 (PD-1) | Gene KO | I & II |
| Non-small Cell Lung Cancer | Programmed Cell Death 1 (PD-1) | Gene KO | I & II |
| Relapsed or Refractory B-cell malignancies | T-Cell Receptor alpha locus (TCRα), CD19/CD22/ CD52 molecule (CD19/CD22/CD52) | Gene KO | I |
| Renal Cell Carcinoma | Major Histocompatibility Complex Class I (MHC-I) & T-Cell Receptor (TCR) | Gene insertion/KO | I |
| Solid Tumors | KO of TCRαβ, & insertion of genes encoding chains of a neoantigen-specific TCR (neoTCR) | Gene insertion/KO | I |
| Solid tumors | CD38 molecule (CD38) | Gene KO | I |
| Herpes Simplex Virus Refractory Keratitis | Helicase-primase subunit/ Single-stranded DNA-binding protein (UL8/UL29) | Gene disruption | I & II |
| Leber Congenital Amaurosis | Centrosomal Protein 290 (CEP290) | Gene correction | I & II |
| Hereditary Angioedema | Kallikrein B1 (KLKB1) | Gene KO | I & II |