Gene Therapy For Human Genetic Diseases Essay

1113 Words Aug 16th, 2016 5 Pages
Gene therapy is the process of using genes to help prevent and cure genetic diseases for patients. The process of gene therapy can involve replacing a mutated gene with a healthier copy, inactivating a mutated gene that is no longer functioning properly or introducing a new gene into the patient’s body, which will then help fight off the disease (Genetics Home Reference, 2016). Gene therapy was first discovered around the 1960s and 1970s, however it was only the concept of the experiment. In 1970, Stanfield Roger proposed an idea that defective DNA could be replaced with “good DNA”. This was later cited by Theodore Friedmann and Richard Roblin in their paper called “Gene therapy for human genetic diseases” created in 1972 (Robertson, 2010). In 1990, the gene therapy process was finally used to help treat a patient. The very first person to have gene therapy performed on them was a four year old girl. The girl had a congenital disease known as adenosine deaminase (ADA) deficiency, which affected her immunity and ability to fight off diseases. The procedure involved taking out her white blood cells and then inserting the genes that helped create ADA into the white blood cells. These would then be reinjected into her, thus completing the procedure. The process is explained further in figure 1. A gene therapy procedure will usually involve the use of viral vectors. A viral vector is a former virus that has had all of the genes that can cause its disease removed from the virus,…

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