Table 2.

Clinical trials of precise genome-editing therapy

Disease type	Target	Strategies	Phase	Trial number
Mucopolysaccharidosis I	Albumin	ZFN-HDR	I/II	NCT02702115
Mucopolysaccharidosis II	Albumin	ZFN-HDR	I/II	NCT03041324
Relapsed Or Refractory CD19+ Leukemia and Lymphoma	TCR	Cas9-HDR	I	NCT05037669
Relapse/Refractory B-cell Lymphoma	AAVS1	Cas9-HDR	I	NCT04213469
Locally advanced or metastatic solid tumors	TRAC	Cas9-HDR	I	NCT03970382
Relapsed or Refractory B-Cell Malignancies	TRAC	Cas9-HDR	I	NCT04035434
β-thalassemia	HBB	Cas9-HDR	I	NCT03728322
Sickle cell disease	HBB	Cas9-HDR	I/II	NCT04774536
Sickle cell disease	HBB	Cas9-HDR	I/II	NCT04819841
β-thalassemia	BCL11A	BE	I	NCT06065189
β-thalassemia	BCL11A	BE	I	NCT06024876
Sickle cell disease, β-thalassemia	HGB1 and HGB2	BE	I/II	NCT05456880
T cell malignancies	CAR7	BE	I	NCT05397184
Acute myeloid leukemia (AML)	CAR33	BE	I	NCT05942599
Acute lymphoblastic leukemia/CD7+ acute myeloid leukemia	CD7, TRAC, PDCD1, and CD52	BE	I/II	NCT05885464
Familial hypercholesterolemia	PCSK9	BE	I	NCT05398029

ZFN zinc-finger nuclease, HDR homology-directed repair, BE base editor