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What is gene therapy?
Gene therapy holds major promise for medical science to correct genetic defects by replacing, augmenting or eliminating absent or defective genes, as well as by providing genes encoding therapeutic or immunogenic proteins.
Gene therapy could potentially be used to combat inherited disorders, such as hemophilia; disorders requiring enhanced production of a protein, such as critical limb ischemia; or acquired diseases such as cancer, cardiovascular disease and HIV infection.
One method of gene therapy works by replacing a patient's ineffective or absent gene with a therapeutic gene (i.e. a replacement gene). The therapeutic gene enables the patient's cells to produce a specific protein that prevents or fights a disease or disorder.
Because the new protein is produced in the patient's own cells, it is recognized as "self."
Gene therapy requires that the therapeutic gene be delivered into the cell. The vehicles for this delivery are called vectors. The most effective vectors come from simple viruses and DNA plasmids, which can penetrate the cell and deliver the therapeutic gene.
As genetic research moves ahead, gene therapy will provide researchers and patients with the ability to combat a wide range of diseases and disorders.
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