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Gene Therapy
Gene Therapy In the middle of the 19th cent, Gregor Mendel performed his landmark experiments indicating that certain traits can be inherited and he proposed a discrete unit of inheritance that we now call a gene. Since then scientists have come to appreciate the gene and have focused on the link between genes and diseases. Gene Therapy is a young and experimental procedure with goals to ultimately prevent diseases by changing the expression of a persons genes. However, there are factors that prevent the absolute success of this procedure, but scientists are continuing to work to find cures for genetic disorders and with technology improving will eventual have absolute success. Gene Therapy is a novel approach to treat, cure, or ultimately prevent diseases caused by a persons genes. Gene Therapy can either be targeted to somatic (body) or germ (egg and sperm) cells. In somatic gene therapy the recipients genome is changed, but the change is not passed down to the next generation. In germline gene therapy, the parents egg and sperm cells are changed with the goal of passing on the changes to their offspring. Currently, germline gene therapy is not being tested on large animals or humans. The understanding of doing the procedures may be clear, but three factors have prevented researches from developing successful gene therapy techniques according to James Wilson from the Institute for Human Gene Therapy at the University of Pennsylvania. The first hurdle is the gene therapy tool. How is a gene inserted into the body? It is done by vehicles called vectors or gene carriers, which deliver therapeutic genes to the patients cells. The most common vectors are viruses, but once inserted into the body, they may introduce other problems. Researchers are experimenting with a 47th artificial chromosome to introduce to the body that can reduce or even get rid of that problem. The second hurdle is understanding the gene function. Of the estimated 100,000 genes, scientists know the function of very few. Once the human genome sequence is complete, scientists will focus on researching the functions of genes. The third hurdle are the multigene disorders. Most genetic disorders involve more than one gene. Most diseases also involve the interaction of the environment. Many people who develop cancer not only inherit the gene for the disorder, but also may smoke to contribute to the disease. With technology advancing, it is not out of the question to understand and fully develop successful gene therapy techniques. Currently, scientists are working on a cure for Cystic Fibrosis. It may sound ridiculous, but scientists in Philadelphia have combined pieces from two of the worlds deadliest microbes, the Ebola virus and the virus that causes AIDS, to make a hybrid virus they hope will someday cure Cystic Fibrosis. The goal is to make a virus with Ebola’s unusual talent for attaching to lung cells and the AIDS virus’s exceptional ability to persist in the body, then use the new virus to deliver curative genes to patients with lung disease. The approach is safe because scientists only use pieces of the two virus’s and eliminate the genes that would kill someone. As technology improves it is safe to say that scientists will figure out all the complications that interfere in successful gene therapy treatments. In the future their may be a cure for all known diseases using the application of somatic gene therapy. Bibliography:
Word Count: 563
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