The Pros And Cons Of Gene Therapy

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In the United States today, Down Syndrome is the most frequent occurring chromosomal disorder; one in every 691 babies are born with this condition. A person with Down Syndrome has a full or partial extra copy of chromosome-21 (NDSS), resulting in mutations with phenotypic characteristics like slanted eyes, flattened head, and broad hands. There are many treatments and therapies that are able to help the needs of each patient because each individual has a different set of needs and problems. Although these treatments are supposed to make the patients’ lives easier, most develop other physical complications throughout their lives. One treatment in particular, gene therapy, has had success with patients who have other conditions. Gene therapy …show more content…

Since gene therapy is such a new and evolving study, there are many risks associated with the treatment. When the body is introduced to a new virus put in place by gene therapy, the body’s immune system may view them as intruders and attack them. If the immune system attacks these cells it could cause inflammation, and in severe cases, it could potentially cause organ failure. This is a very real possibility when patients with Down Syndrome are injected with viral vectors unfortunately, but with careful examination of the patients’ cells, organ failure could be prevented. Another risk that could potentially come along with these therapies is the possibility of causing a tumor if the new genes get placed in the wrong spot of the DNA inside the nucleus of the cells. With gene therapy regarding Down Syndrome, the possibility of a tumor forming could very well happen, although it would only happen if the new genes were put into the wrong place which would not happen at all if they were placed in the right place every time. One last major risk that could occur with gene therapy is the targeting of the wrong cells. If that were to happen, healthy cells could end up being altered when they should not be changed at all. These risks could potentially happen in gene therapy patients with Down Syndrome; however, scientists are working as hard as they can in order to fix any of these risks and stop them from happening. The possibility of curing the disorder and letting these people live longer and healthier lives is something that makes gene therapy worth the