Gene Therapy Treatment

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Human Gene therapy trials conducted

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

Related Links
Gene Therapy Introduction
Genetic Counseling
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