| Gene therapy |
Condition |
Target cells |
| Adenosine
deaminase |
ADA
defeciency |
Lymphocytes,
bone marrow cells. |
| Tumor
necrosis factor |
Melanoma |
Tumor
infiltrating lymphocytes, autologous tumor cells. |
| Interleukin
- 2 |
Melanoma,
glioblastoma, renal cell cancer |
Autologous
tumor cells, tumor cells |
| Factor
IX |
Hemophilia
B |
Autologous
skin fibroblasts |
| LDL
receptor |
Hypercholesterolemia |
Autologous
liver cells |
| Histocompatibility
locus antigen class –I B7 plus β2-microglobulin |
Melanoma,
colorectal cancer, renal cell cancer |
Tumor
cells |
| HSV
thymidine kinase |
Glioblastoma,
AIDS, ovarian cancer |
Tumor
cells – T cells |
| Cystic
fibrosis transmembrane conductance regulator |
Cystic
fibrosis |
Nasal
and airway epithelium |
| Multi
drug resistance -I |
Breast
cancer |
Blood
CD 34+ cells |
| Granulocyte-macrophage
colony stimulating factor |
Melanoma |
Tumor
cells |
| Human
CNTF |
Amyotrophic
lateral sclerosis |
Encapsulated
transduced and exogenic cells. |
| p53 |
Head
and neck squamous carcinoma |
Tumor
cells |
| Fanconi
anemia |
Fanconi
anemia |
Bone
marrow cells. |
| Interleukin-
1 receptor antagonist |
Arthritis |
Autologous
fibroblasts |
