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This is an experimental technique and is done by inserting genetic material into the patient’s cells. The genes thus introduced exert their benefits by either compensating for the abnormal genes or…
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What is gene therapy? Using genes to prevent or treat a disease is known as gene therapy (NIH, 2008). This is an experimental technique and is done by inserting genetic material into the patient’s cells. The genes thus introduced exert their benefits by either compensating for the abnormal genes or by producing beneficial protein or by supplementing the abnormal gene (Hecht, 2004). The first gene therapy clinical trial started in 1990 (Hecht, 2004). Currently, research is going on for gene therapy by various approaches in different parts of the world for various diseases like combined immuno-deficiencies, hemophilia, Parkinsons disease, cancer and even HIV (Hecht, 2004).
The uses of gene therapy are to replace absent or defective genes, deliver genetic material into cells that speed the destruction of cancer cells, deliver genetic material into cells that cause cancer cells to revert back to normal cells, deliver genes of bacteria or virus as a form of vaccination, deliver genetic material that either impedes or promotes growth of a new tissue and deliver genetic material that stimulates healing of a damaged tissue (Hecht, 2004).
There are 2 types of gene therapy:
1. Germ line gene therapy: In this type of therapy, the genetic material is introduced into the germ cells and gets integrated into their genomes. Thus the change is heritable.
2. Somatic cell gene therapy: Only the somatic cells which are involved in the disease process are dealt with. Currently, all trials and research in gene therapy involves somatic cells (Hecht, 2004).
There are 2 methods of gene therapy:
1. Ex vivo gene therapy: Here, gene delivery is done in cells after being removed from the body (Hecht, 2004). These cells are grown in the laboratory. The cells are than modified outside the body and then transplanted back into the body. In some research trials, cells from blood or born marrow are taken out and cultured in a laboratory. Thereafter, the cells are exposed to the virus with the desired gene. The virus infects the cells and transfers the therapeutic genetic material into the nucleus of the cells. After this, the cells are injected into the patient’s body by vein.
2. In vivo gene therapy: Here, gene delivery is done in the cells that are still in the body.
Gene therapy has been used to restore vision in dogs. It has also been attempted to treat Lebers congenital amaurosis, a type of inherited childhood blindness caused by a single abnormal gene. Studies have shown that tumor suppressing genes delivered in lipid-based nanoparticles reduces the number and size of human lung cancer tumors in mice. Reengineered lymphocytes have been used to attack cancer cells in metastatic melanoma and myeloid disorders. Other studies have suggested a possible cure for diseases like Hingtinton’s chorea, severe combined immunodeficiency and sickle cell diseas, thalassemia, cystic fibrosis and some cancers in the future (Genomics.energy.gov). Researchers are also experimenting with introducing a 47th (artificial human) chromosome into target cells. It is expected that this chromosome would autonomously stand along side the 46th chromosome without affecting its functions (Genomics.energy.gov).
References
Genomics.energy.gov. Gene Therapy. Retrieved November 22nd, 2009 from http://www.ornl.gov/sci/techresources/Human_Genome/medicine/genetherapy.shtml
Hecht, F. (2004). Gene Therapy - The Future Is Here! MedicineNet.com. Retrieved November 22nd, 2009 fromfromhttp://www.medicinenet.com/script/main/art.asp?articlekey=12662
NIH. (2008). What is gene therapy? Genetics Home Reference. Retrieved November 22nd, 2009 from http://ghr.nlm.nih.gov/handbook/therapy/genetherapy Read More
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