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How does gene therapy work All life contains genetic information in the form of DNA DNA can be reproduced and passed on to subsequent generations A gene is a stretch of DNA that contains the blueprint for the sequence of amino acids making up a particular protein Proteins are vital because they are used in catalyzing various biochemical reactions they act as messengers they regulate cell growth development and reproduction transport oxygen in the blood and are a defense against diseases If a particular gene is mutated its protein product may not be made at all or it may work poorly or even too aggressively This disrupts cell and tissue functions that depend on the normal gene product and causes abnormal cell behaviour leading to symptons of disease Correction of mutated genes can be carried out by introduction of a normal gene into the cell or by direct repair Gene therapy consists of three steps 1 Administration introduction of the gene or a vector containing the correct version of the mutated gene into the body 2 Delivery transfer of the gene from the site of administration to the nucleus of the target cell 3 Expression production of normal protein product in the cell restoring normal cellular function Somatic vs germ-line gene therapy Gene therapy is the deliberate transfer of DNA for therapeutic purposes There are essentially two forms of gene therapy somatic and germ-line gene therapy At present gene therapy is being considered seriously only on somatic cells Somatic gene therapy is the manipulation of genes in a patients cells Any change will not be inherited in subsequent generations because target cells do not form a part of the germline On the other hand germ-line therapy would involve genetic engineering that transcends generations Gene therapy is targeted to germ cells Any genetic modification of germ cells will be passed on to the next generation This permanency frightens ethicists and it is largely due to ethical
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