Table 2 Clinical trials of gene editing in the treatment of human diseases.

From: Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

Platform

Disease applications

Target

Edited cells

Delivery

Sample size

Phase

Trial number

ZFN

HIV-1 infection

CCR5

CD4+ T cells

Adenovirus

12

I

NCT00842634

 

HIV-1 infection

CCR5

CD4+ T cells

Adenovirus

19

I

NCT01044654

 

HIV-1 infection

CCR5

CD4+ T cells

Adenovirus

21

I/II

NCT01252641

 

HIV-1 infection

CCR5

CD4/CD8 T cells

Adenovirus

26

I

NCT01543152

 

HIV-1 infection

CCR5

CD4/CD8 T cells

mRNA

12

I/II

NCT02225665

 

HIV-1 infection

CCR5

CD4+ T cells

mRNA

14

I

NCT02388594

 

HIV-1 infection

CCR5

CD4+ T cells

mRNA

30

I/II

NCT03666871

 

HIV-1 infection

CCR5

CD4+ T cells

mRNA

12

I

NCT03617198

 

HIV-1 infection

CCR5

CD34 + HSPCs

mRNA

18

I

NCT02500849

 

HPV-induced cervical precancerous lesions

HPV16/18 E7

Epithelial cells

DNA

20

I

NCT02800369

 

Mucopolysaccharidosis I

IDS gene

Hepatocytes

AAV

9

I/II

NCT03041324

 

Mucopolysaccharidosis II

IDUA gene

Hepatocytes

AAV

3

I/II

NCT02702115

 

Hemophilia B

Factor IX gene

Hepatocytes

AAV

12

I

NCT02695160

 

β-Thalassemia

BCL11A gene

CD34 + HSPCs

mRNA

6

I/II

NCT03432364

 

Recurrent/refractory malignant glioma

IL13Ralpha2

CD8 + T cell

Injection

6

I

NCT01082926

TALEN

Relapsed/refractory B-ALL

CD52, TRAC

CAR T cells

Lentivirus

18

I

NCT02808442

 

HPV-related cervical intraepithelial neoplasia

HPV16/18 E6/E7

Epithelial cells

Plasmid

40

I

NCT03226470

TALENs and CRISPR/Cas9

HPV-related cervical intraepithelial neoplasia

HPV16/18 E6/E7

Epithelial cells

Plasmid

60

I

NCT03057912

CRISPR/Cas9

AML

CD123, TRAC

CAR T cells

mRNA

162

I

NCT03190278

 

Metastatic non-small cell lung cancer

PDCD1

T cells

DNA

12

I

NCT02793856

 

Castration-resistant prostate cancer

PDCD1

T cells

DNA

Withdrawn

I

NCT02867345

 

Muscle-invasive bladder cancer

PDCD1

T cells

DNA

Withdrawn

I

NCT02863913

 

Advanced esophageal cancer

PDCD1

T cells

DNA

16

I

NCT03081715

 

Metastatic renal cell carcinoma

PDCD1

T cells

DNA

Withdrawn

I

NCT02867332

HIV-1 infection with ALL

CCR5

CD34+ HSPCs

Liposome and electroporation

5

I

NCT03164135

 

EBV-positive cancers

PDCD1

T cells

DNA

20

I

NCT03044743

 

Relapsed refractory multiple myeloma, melanoma, synovial sarcoma, and myxoid/round cell liposarcoma

NY-ESO-1, TRAC PDCD1

T cells

Lentiviral and electroporation

18

I

NCT03399448

 

Relapsed or refractory CD19+ leukemia and lymphoma

TRAC, B2M

CAR T cells

Lentiviral and electroporation

80

I/II

NCT03166878

 

Relapsed or refractory CD19- leukemia and lymphoma

CD19 and CD20 or CD22, TRAC

CAR T cells

Lentiviral and electroporation

80

I/II

NCT03398967

 

Mesothelin-positive multiple solid tumors

PDCD1 and TRAC

CAR T cells

Lentiviral DNA

10

I

NCT03545815

Mesothelin-positive multiple solid tumors

PDCD1 and TRAC

CAR T cells

Lentiviral DNA

10

I

NCT03747965

 

Metastatic gastrointestinal epithelial cancer

CISH

TIL

Electroporation

Withdrawn

I/II

NCT03538613

 

T cell leukemia or lymphoma

CD7, CD28

CAR T cells

–

21

I

NCT03690011

 

Neurofibromatosis type 1

NF1

iPSCs

DNA

20

I

NCT03332030

 

β-Thalassemia

HBB gene

iHSCs

–

12

I

NCT03728322

 

β-Thalassemia

BCL11A gene

CD34+ HSPCs

–

45

I/II

NCT03655678

 

Sickle cell disease

BCL11A gene

CD34 + HSPCs

–

45

I/II

NCT03745287

 

LCA10

CEP290 gene

Photoreceptor cells

AAV

18

I/II

NCT03872479

  1. ZFN zinc-finger nuclease, CCR5 chemokine receptor 5, HSPCs hematopoietic stem/progenitor cells, IDS iduronate 2-sulfatase, IDUA α-L-iduronidase, BCL11A mouse B cell lymphoma factor 11A, B-ALL B acute lymphoblastic leukemia, TRAC T cell receptor alpha chain, TALEN transcription activator-like effector nuclease, CRISPR clustered regularly interspaced short palindromic repeat, AML acute myeloid leukemia, PDCD1 programmed cell death 1, NF1 neurofibromatosis type 1, TIL tumor-infiltrating lymphocytes, iPSCs induced progenitor stem cells, iHSCs induced hematopoietic stem cells, LCA10 Leber congenital amaurosis type 10, CEP290 centrosomal protein 290, AAV adeno-associated virus
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