Table 1 Application of Cas9 nuclease in clinical therapy*

Target gene	Clinical ID	Therapy	Disease	Stage
TRAC, B2M	NCT03166878, NCT03229876, NCT03166878	CAR-T	B cell leukemia	Phase 1/2
TRAC, TRBC, B2M	NCT04244656, NCT03166878	T cell therapy, CAR-T	Myeloma, B cell lymphoma	–
TRAC, TRBC, PDCD1	NCT03399448, NCT03545815	TCR-T	B cell leukemia and solid tumor	Phase 1
TRAC	NCT03398967	CD19, CD20 or CD22 CAR-T therapy	Relapsed or refractory hematological malignancies	Phase 1/2
PDCD1	NCT02793856	T cell therapy	Advanced Non-small Cell Lung Cancer	Phase 1
NCT02863913	T cell therapy	Stage IV bladder cancer	Phase 1
NCT02867332	T cell therapy	Metastatic renal cell carcinoma	Phase 1
NCT03081715	T cell therapy	Esophageal cancer	Phase 1
NCT02867345	T cell therapy	Hormone refractory prostate cancer	Phase 1
CD7	NCT03690011	CAR-T	T cell leukemia	Phase 1
CD70	NCT04438083	T cell therapy	Hematologic malignancies and renal cell carcinoma	Phase 1
CCR5	NCT03164135	HSPC therapy	HIV and leukemia	–
BCL11A	NCT03432364, NCT03655678, NCT03745287, CRISPR_SCD001	HSC therapy	Transfusion-dependent β-thalassemia (TDT) and sickle cell disease (SCD)	Phase 1/2
HBB	NCT03728322	HSC therapy	TDT	Phase 1
CEP290	NCT03872479	AAV therapy	Leber congenital amaurosis type 10 (LCA10)	Phase 2
CISH	NCT03538613, NCT04089891	T cell therapy	Metastatic gastrointestinal epithelial cancer	Phase 1/2

*Because of space limitation, only typical clinical trials are cited here.