DISEASE

GENES TARGETED FOR GENE THERAPY

SCID

Adenosine deaminase

Cystic Fibrosis

CFTR

Familial hypercholesterolemia

LDL Receptor

Hemophilia B

Factor IX

Thalassemia

α or β globin

Sickle cell anemia

β globin

Lesch nyhan syndrome

HGPRT

Gaucher's disease

Glucocerebrocidase

Peripheral artery disease

Vascular endothelial growth factor

Fanconi Anemia

Fanconi anemia C

Melanoma

Tumor necrosis factor

Renal cancer

IL- 2

Brain cancer ( Glioblastoma), AIDS, Ovarian Cancer

Thymidine Kinase ( Herpes Simplex Virus)

Head & neck cancer

p53

Breast Cancer

Multi Drug Resistance