Gene Therpy Essay Research Paper Human Gene

Gene Therpy Essay, Research Paper

Human Gene Therapy “To much of the general public, genetic engineering is a vague notion of high tech manipulation of plants and animals. Its results too difficult to understand” (Cowen 18). It was not until this century that humans were granted a glimpse into the possible origins of biological inheritance. Scientists have only been aware of the existence of the hereditary factors, known as genes, for a little more than one hundred years, and the molecular nature of the gene for about twenty-five years. For the rest of our history, we have lived in absolute ignorance of genetics. Genetic engineering is a growing area of science capable of delivering amazing treatments to many hereditary ailments. Doctors estimate that at least ten per cent of diseases are genetic in origin. There is at present no cure for genetic defects. Gene therapy is a tremendous breakthrough in modern technology, benefical only if it is used to aid the medically inflicted. Although human gene therapy is a relatively new branch of science, there are unlimited potential benefits; therefore, the genetically imperfect individual should have the option to assume these risks and undergo experimental procedures to improve his or her overall health. As a result of recent advances in medical science, researchers believe that a gene can be transplanted into humans suffering from severe diseases. Such gene transplants can alleviate or perhaps even cure diseases for which no adequate treatment presently exists. The treatment is called human gene therapy, which is one of a series of emerging genetic techniques commonly called genetic engineering. These sciences are based on our emerging knowledge about how genes work. Genes, consisting of a chemical called DNA, direct the various traits of each individual’s body (Avigen). According to David Suzuki, Professor of Biology at the University of British Columbia, “The system may be thought of as a tinkertoy set, in which sticks, wheels, and blades can be snapped together into windmills, castles, cars, and a diversity of other forms limited only by the child’s imagination and patience” (22). Together, the total genetic information contained in genes comprises a genome, a unique genetic blueprint for each organism (Friedman 244).When the DNA information of a particular gene contains mistakes, the gene may not function properly. Sometimes the malfunction will not be serious, but other times it will result in a severe genetic disease. Examples of some genetic diseases are cystic fibrosis, sickle cell anemia, and hemophilia (Reuters Foundation). Hemophilia, for instance, is caused by the malfunctioning of the gene responsible for blood clotting (Chadwick 147). As more is learned about human genetics, scientists are discovering that diseases such as diabetes, cancer, heart disease, and some manic depressive illnesses result in part from faulty DNA information, and therefore also hold the potential of being remedied by human gene therapy. Human gene therapy is a possible alternative to the painful, expensive approach to the treatment of some genetic diseases. Gene therapy technologies are now making it possible to provide treatments that twenty years ago were hardly dreamed possible. These new technologies will bring about a whole new era in medicine. The basic idea behind gene therapy is to insert normal genes with correct information into the DNA of the cells that contain malfunctioning genes. Adding genes in this way is called gene insertion. The added genetic information allows these cells to function properly and can reduce or eliminate the signs or symptoms of the disease. For example, instead of repeatedly treating a hemophiliac with a blood coagulant, the insertion of the correct genetic material into his cells enables those cells to make their own clotting factor (Chadwick 147). Every year thousands of ailing children are born with cystic fibrosis, which leads to a clogging of the lungs, and to problems with digestion and other functions. Because a recessive gene possessed by one in every twenty Caucasians is responsible for the abnormality, one in every four hundred couples risks giving birth to afflicted progeny (Chadwick 135). Although improvements in care and treatment mean that people with cystic fibrosis can now live for up to twenty-five years, there is no cure. But there is gene therapy. Human gene therapy is capable of treating this disease which is stealing the lives of many young people. A much rarer but nevertheless well known genetic disease, is severe combined immunodeficiency (SCID). Children with this condition have a defect in both copies of a gene which is required for the proper functioning of the immune system. The children can be kept alive but they have to live in sterile plastic bubbles to isolate them from bacteria and viruses. These children live horrible lives then die of infections which only keep the healthy population in bed for a couple of days. Again, while there are treatments, there is no cure. However, the cure is obvious. If the children are ill because they have two bad copies of a gene, they could be given a good copy of the gene that would be healthy like their parents’. The new genetic technology also benefits these individuals because they are now less prone to pass the disease on to their offspring.Another disease treated through gene therapy is sickle cell anemia. This disease is caused by a single DNA ba