Negative Research Paper: Human Gene Therapy

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Deborah McNeal Dr. Lipka BIO 4 10-15-14 Human Gene Therapy In today’s time, gene therapy is one of the most controversial topics in medicine. By definition, it is the delivery of genetic material into a cell for the purpose of either correcting a genetic problem or giving a cell a new biological function. The purpose of gene therapy is to correct an undesirable trait or disease by introducing a modified copy of a gene into a target cell. In most cases, the purpose is not to replace a defective gene in the host cell but rather to provide a new copy so that the correct protein can be expressed and the detrimental effects of the defective gene neutralized (). This “therapy’ is a fairly new practice of only about twenty or so years and much more…show more content…
However, the treatment did not stop the retina from continuing to degenerate. In another trial, 6 out of 9 patients with the degenerative disease choroideremia had improved vision after a virus was used to deliver a functional REP1 gene. People with hemophilia are missing proteins that help their blood form clots. Those with the most-severe forms of the disease can lose large amounts of blood through internal bleeding or even a minor cut. In a small trial, researchers successfully used an adeno-associated viral vector to deliver a gene for Factor IX, the missing clotting protein, to liver cells. After treatment, most of the patients made at least some Factor IX, and they had fewer bleeding incidents. Patients with beta-Thalassemia have a defect in the beta-globin gene, which codes for an oxygen-carrying protein in red blood cells. Because of the defective gene, patients don't have enough red blood cells to carry oxygen to all the body's tissues. Many who have this disorder depend on blood transfusions for…show more content…
These genes gave cells that don't normally make dopamine the ability to do so. After treatment, all of the patients in the trial had improved muscle control. In the fall of 2013, a new technique was introduced that allows scientists to edit genetic information with greater precision. With regular gene therapy, detailed alterations can be made to human DNA, potentially allowing scientists to treat genetic disorders. Scientists believe the technique is so accurate it might be used in place of the normal procedure for gene therapy - replacing faulty genes with healthy ones . Controversially, it might also be used to correct gene defects in human IVF embryos, allowing disorders to be ‘ironed out’ before a baby is born. Previously gene therapy relied on using viruses to insert DNA at random into the human genome - an inaccurate and risky process. The new technique, is called ‘Crispr’, uses ‘cutting enzymes’ to target specific parts of the 23 pairs of human chromosomes, without introducing unintended mutations or

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