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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,...

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