Gene which is Human Immunodeficiency Virus (HIV),

Gene therapy is the experimental treatment of normal genes into cells in place of absent or deficient ones in order to rectify genetic disorders. The NIH reported that it has taken two decades for this clinical therapy to finally mount success. Several clinical trials have raised hope that gene therapy can be used as a standard treatment option in curing brain disease. However, today, a recent study has found that for the first time ever, doctors are able to effectively use this treatment to save their patients from an irreversible deterioration brain disease (Kolata, 2017). NYTIMES’s article “In a First, Gene Therapy Halts a Fatal Brain Disease,” explores the key factor that was contributed in making gene therapy efficient, which is Human Immunodeficiency Virus (HIV), an adversary in medicine (Kolata, 2017). In the latest study, researchers aimed to study patients who were children that inherited a mutated disease, Adrenoleukodystrophy (ALD).  ALD is a rare disorder that results from the lack of myelin surrounding the nerve cells of the brain. Nerve cells dying in the brain result in children losing the ability to walk, talk, eat, see, hear or think, and ramifies in them dying within a short span (Kolata, 2017). ALD targets one in every 20,000 boys; entailing that symptoms originate as early as the age of seven. Formerly, the only treatment of ALD was either a bone-marrow transplant if a well-suited donor could be traced or an umbilical cord blood transplant if it was noticed at birth (Kolata, 2017). Researchers collected 17 boys of the ages from 4 to 13 who received gene therapy. The results from the sample indicated after two years, 15 patients were functioning normally without obvious symptoms, and the remaining two died, one from the disease rapidly growing and the other withdrew from the study to get a transplant. Researchers believe that this study could now open new avenues for using gene therapy to treat brain diseases. The news article employs the use of statistics by presenting the findings from the study conducted. The percentage of patients killed from transplants, 20%, is an identifiable statistic, as it represents the data collected from the experiment. With the help of these statistics, researchers were able to draw the logical conclusion that the best decision for the treatment of ALD, was inserting a disabled form of HIV into human cells (gene therapy), which was considered more safe and helped stop brain degeneration. Since the percentage of patients who were cured from gene therapy was greater than the percentage of patients who died from transplants, the researchers were able to reject the null hypothesis and conclude the gene therapy treatment significance. The article bases it premise off the statistics collected from conducting experimental research, a method of manipulating variables to obtain data. In this experiment, the independent variable, the variable being manipulated, was the gene therapy treatment for each participant and the dependent variable was the usage of a disabled form of HIV to deliver a normal form of the ALD gene. The researchers used random assignment to assign the participants to a condition, either a disabled form of HIV is inserted into genes in human cells or undergo a bone-marrow transplant (Katala, 2017). Using random assignment eliminates any potential biases for both the researchers and participants. The data presented is ratio data, and represents a continuous variable. The article used both descriptive and inferential statistics, providing the reader with the percentage of patients who are killed from bone-marrow transplants, and inferring the effectiveness of gene therapy treatment to the ALD population. After introducing the idea of the new treatment, the article explains the recent experiment conducted, which we can assume used NHST testing. The researchers were able to reject the null hypothesis that the bone-marrow transplant had no greater effect on ALD than gene therapy, and could conclude that the gene therapy treatment did in fact cure patients with ALD.