Table 2 Clinical trials of precise genome-editing therapy

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