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Gene Therapy

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Assignment 2: Gene Therapy
Science 115 – Introduction to Biology
March 1, 2014

Gene therapy is the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders (Genetics home reference, n.d). This is a promising treatment for diseases such as inherited disorders, some types of cancer, and certain viral infections. The way this treatment works is by introducing genetic material into cells to compensate for genes that are abnormal or are not making beneficial proteins. Mutated genes can cause a protein to be faulty or missing, so a new copy of the gene is introduced to proper function of the protein. This is still a very risky technique and is under study to make sure it will be safe and effective. Currently, it is only being used to treat diseases such as severe combined immunodeficiency—also referred to as SCID or boy-in-the-bubble syndrome—where patients are unable to fight infection and die in childhood (Scientific American, n.d). A further break down of the processes of gene therapy will help to understand exactly how to treatment works. A gene cannot be inserted directly into the cell or it will not function. A carrier called a vector has been genetically engineered to deliver the gene to the cell. This vector can be used in two ways: in-the-body gene therapy and out-of-the-body gene therapy. In-the-body therapy can be injected or given intravenously (through an IV) directly into a specific tissue of the body. In out-of-the-body gene therapy, researchers take blood or bone marrow from a patient and separate out immature cells (Scientific American, n.d). A gene is added to those cells and the cells that contain the vector are then returned to the patient. If the treatment worked, then the new gene will be delivered by the vector will make properly functioning protein and replacing all…...

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