A large number of gene-related diseases have been treated and even cured due to the advances in gene therapy. Within a short period of time, gene therapy has transferred from the conceptual stage of development to clinical trials that actually test patients of deadly diseases. Severe Combined Immune Deficiency is one deadly disease that scientist have began treating with gene therapy. Severe Combined Immune Deficiency, also known as ACA-SCID, is where affected individuals are born without an effective immune system. Investigators in Italy first found a cure or a long-term correction of the deadly genetic disorder. According to American Society of Gene & Cell Therapy, “The therapeutic gene called ADA was introduced into the bone marrow cells of such patients in the laboratory, followed …show more content…
After this transplant, the immune system of the individuals was reconstituted without any noticeable side effects. The same treatment was done to two patients with Chronic Granulomatous Disorder (CGD). Chronic Granulomatous Disorder is a condition where the immune system is unable to fight off fungal and bacterial infections. Both patients’ immune systems were able to fight off infections for at least two years with the gene therapy treatment (“Gene Therapy for Disease”). The use of gene therapy has been a life-changing treatment for patients with deadly gene condition. Although some patients may not have been fully cured, a chance at a normal and healthy life was possible for a substantial amount of time.
The Human Genome Project lead to revolutionary advances in the treatment of gene-related disease. The Human Genome Project began in 1990 and was run by the National Institutes of Health and the U.S Department of Energy. The goals of the Human Genome Project was to identify genes in the human DNA and determine the