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GENE THERAPY By: Carmen Nahous & Calisa Lakhan What is a gene? ~A gene is the basic unit of heredity in a living organism that determines physical traits. ~They are found on particular loci (positions) of chromosomes. ~Your cells use the information from your genes to manufacture proteins that do the work in your body. ~Genes contain your DNA. . What is Gene Therapy? ~Gene therapy involves altering the genes inside your body’s cells to stop diseases, by replacing a faulty gene or adding a new gene in an attempt to cure a disease or make changes in the body so that it is better able to combat diseases. ~Gene therapy holds promise for treating a wide range of diseases including cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS. Why is Gene Therapy done? Replace missing or mutated genes This is currently the most common gene therapy approach. Some cells become diseased because some genes have been permanently switched off. Other cells may be missing certain genes. Researchers hope that replacing missing or defective genes can help treat certain diseases. Changing the regulation of a gene Mutated genes that cause diseases could be turned off or healthy genes that prevent diseases could be turned on so that they can inhibit the disease. Making diseased cells more evident to the immune system The immune system does not always attack the diseased cells because they are not recognized as intruders. Using gene therapy, doctors could potentially infuse mutated cells with genes that make them more recognizable to the immune system. Risks associated with Gene Therapy Gene therapy poses a number of risks. A gene can’t simply be inserted directly into the cell. It usually has to be delivered using a carrier, called a vector. Commonly used as vectors are viruses because they can recognise certain cells and carry genetic material into the cells genes. ~This technique presents the following risks: Immune response The body’s immune system may see the newly introduced viruses as intruders and attack them. Viral Spread Because viruses can affect more than one type of cell, it is possible that the viral vectors may infect cells in addition to the ones for which they were intended – just those containing mutated or missing genes. If this happens, healthy cells may be damaged, causing other illnesses or diseases, including cancer. Possibility of Inducing a Tumor If the new genes get inserted in the wrong spot in the genome, there is a chance that the insertion might lead to a tumor formation. Conclusion The possibilities of gene therapy hold much promise. To date however, that promise has not been realised. Some clinical trials have recorded small successes for a few participants. But several significant barriers stand in the way of gene therapy becoming a reliable form of treatment. The barriers include developing reliable vectors, consistently ensuring safety, targeting the correct cells and preventing genetic changes from being passed on from parent to child. Although at this time no clinical trial has been seen as a breakthrough in overcoming all these barriers, gene therapy is a very active area of research. Researchers hope that with continued study, advances in gene therapy will eventually make it a practical approach to treating diseases.