Genetic Disorders: The Pros And Cons Of Gene Therapy

The human body is inherently flawed. Every person, no matter their background, is born with a couple of genetic disorders, whether they are visible or not. However, scientists have come to a point where they might be able to alter and correct these genetic disorders through the application of gene therapy, where a disorder or disease is corrected by the insertion of a gene into a cell rather than using antibiotics (“What is Gene Therapy”). Despite the extraordinary benefits gene therapy could have for the world’s population, developing the practice has been plagued by difficulties and questions of ethics. The concept of gene therapy was originally proposed as early as 1970, but it was not until the 1980’s that experiments on the efficacy of

The scientists then theorized these cells could be injected into a person with the disease. As the 1980’s continued, gene therapy attained a foothold in medical scientists’ minds as a feasible procedure to treat certain diseases. After gaining an increased ability to identify genetic abnormalities that caused disorders and inherited diseases, scientists realized all diseases had a genetic component, meaning gene therapy could be a method to diminish the effects of, or even cure, all diseases. Finally, on September 14th, 1990, a girl suffering from a disorder preventing her from producing a critical enzyme became the very first person to undergo the process of gene therapy. This girl was

For example, scientists have repeatedly tried to apply gene therapy to cystic fibrosis. Usually, a virus containing the DNA needed to fix a disease, called a vector, is injected into the infected cells to fix the anomaly in the genetic code. However, natural barriers in the airways that prevent viruses from entering the lungs have prevented the use of gene therapy as plausible method of curing the disease. Since the vector cannot be accurately injected into the outer membrane of the lungs, the area affected by the cystic fibrosis gene, the disease cannot be cured currently (DeVine). Another large problem in developing gene therapy is the inherent complexity of the concept. Despite genetic defects being quite common in society, single-gene defects, which are the defects that are most susceptible to treatment by gene therapy, are very uncommon. Diseases such as muscular dystrophy and Huntington’s disease are single cell defects, but are rare among the general population. The rarity of these diseases not only adds to complexity, as the source of a disorder can vary dramatically (Judson 3). For example, a disease like sickle-cell anemia is present in red blood cells, while a disease like Huntington’s disease is a neurological disorder present in brain cells (Smith 1). Just from these two diseases alone, it is obvious both would need different forms of injection into the body. These two major problems