Kaylynn A. Foulk, Cinema and Television Arts Major, California State University, Fullerton
Abstract
This research paper explores severe combined immunodeficiency disease based from the research provided by four different articles and a book. Two of the articles were provided by the professor, but the other two articles I have researched on my own. The research paper provides a brief description of severe combined immunodeficiency disease and how it affects the immune system of a child’s body. The differences and similarities between adult stem cells and embryonic stems is provided as well. In addition, the research paper will explain why modification of stem cells are necessary and what would happen if the cells were left unmodified. Lastly, the use of embryonic stem cells being considered as controversial will be explained.
Keywords: SCID, gene therapy, embryonic stem cells
Bubble Boy Disease Popped Severe combined immunodeficiency disease is the most serious primary immunodeficiency illness. The essential characteristic of SCID is the nonappearance of T cells and, consequently, lack of B cell function as well. Unless these defects are modified the child will die of opportunistic infections before their first or second birthday. Severe combined immunodeficiency disease can be triggered by more than a few different genetic defects, most of which are hereditary. SCID is to afflict anywhere from one in 100,000 to one in 50,000 live births as stated by Haberman’s article (Haberman 2015). In the past, children with this disorder were kept in harsh seclusion, sometimes in a plastic isolator or "bubble". Bubbles are no longer used as a typical of care, but the name remains a part of the past of severe combined immunodeficiency disease. The SCID Disease SCID, usually pronounced "skid”, is a disorder in which a baby is born with an immune response that is unable to correctly produce a kind of white blood cell. This leaves the infant defenseless to infections and generally leads to death within the first year of life. There are fourteen forms of severe combined immunodeficiency and one of those forms is triggered by an absence of an enzyme called adenosine deaminase, or ADA for short, which is essential for lymphocytes to grow. Therefore, this kind of form would be known as adenosine deaminase deficiency SCID, generally called ADA SCID. ADA SCID is fatal without treatment. One known treatment is gene therapy which is the transfer of normal genes into cells in place of misplaced or malfunctioning ones with the purpose of accurate genetic disorders. According to Park’s article Dr. Kohn treated Evangelina with gene therapy by co-opting a modified virus to carry the healthy ADA gene so it could infect the stem cells from Evangelina’s bone marrow (Park 2014). In bone marrow, they typically produce white blood cells, but in those with SCID, a faulty gene disturbs normal white blood cell production. Next, in a test tube, the bone marrow stem cells were infested with a transgenic virus carrying the working gene. In cases where the procedure worked, the virus injected the good gene into the DNA of the stem cells, which remained at that point injected back into the baby’s bone marrow. There, the cells could construct normal white blood cells, forever curing the disease. However, as stated by the article written by Amy Dockser Marcus, many wonder whether gene therapy was too risky and too complex to become a safe and effective …show more content…
This modification of the gene therapy procedure mimics one originally used with some accomplishment by a discrete group of scientists. The patients who benefitted the greatest were also the youngest, as well as a baby boy, diagnosed with ADA SCID at ten months old after a major illness. Gene therapy has had slight accomplishment treating illnesses. Success has been pushed back by quite a few complications, including: problems getting working genes into the specific cells where they are needed, getting working genes into enough cells and at the right rate to produce a physiological effect, the transfer organism entering unintended cells, and regulating gene expression (Phelan 2015). Furthermore, more than one faulty gene may cause a disease, or the gene causing a disease may be