Gene Therapy Research
The National Institute of Health and the Food and Drug Administration are the government bodies responsible for the regulation of gene therapy experimentation. The NIH oversees gene therapy clinical trials through the NIH Guidelines for Research Involving Recombinant DNA Molecules and the Recombinant DNA Advisory Committee (RAC). The Food and Drug Administration (FDA) also oversees the trials through such regulatory measures as scientific review, testing, and inspection.10
Following the death of Jesse Gelsinger, the Advisory Committee to the Working Group on NIH Oversight of Clinical Gene Therapy Research was established to examine the purpose and role of the NIH in gene therapy regulation. It made several recommendations, many of which were adopted by the NIH as amendments to its research guidelines.12
In March of 2000, two initiatives by the FDA and the NIH were announced by the Department of Health and Human Services to increase protection for individuals involved in gene therapy experiments. The first initiative, the Gene Therapy Clinical Trial Monitoring Plan, requires that the sponsors of gene therapy studies regularly submit their monitoring plans to the FDA for review. The FDA will also inspect clinical trials to insure that the plans are being followed, and any problems are managed. In addition, scientists experienced in gene therapy will be gathered to discuss monitoring practices at conferences of the inspectors. The second initiative, the Gene Transfer Safety Symposia, includes a series of symposia that take place four times a year to discuss gene therapy data, inviting some of the most experienced authorities in their respective fields. The NIH and FDA are also providing support for other organizations interested in holding safety conferences on the topic of gene therapy.12
Proposed Review Process3
The guidelines of the NIH for gene therapy research have also changed since 2000. In one amendment to the guidelines governing gene transfer research, the NIH altered the way research subjects are enrolled for gene therapy experiments. Under the amendment, clinical trial protocols must be submitted to the NIH Office of Biotechnological Activities (OBA) for RAC review, and the RAC must finish its review before the local Institutional Biosafety Committee (IBC) gives its approval to the trial. Subjects for research can only be accepted once the IBC and the International Review Board, IRB, approve the trial.
Harris, Carol. "Women Under Fire in World War Two". BBC News. BBC, 17 Feb. 2011. Web. 19 Apr. 2014
IRBs review all aspects of the researchers' project: the study design, the recruitment process, the participant population, the informed consent document and process, the risk/benefit ratio, privacy and confidentiality, data storage and protection, and safeguards for vulnerable participants (University of St. Francis, n.d.). In this way, participants' rights are protected because the effort is made even before the research begins. The review process ensures that participants are chosen fairly and adequately and the information collected during research is safeguarded through collection, use, and storage. Research using human participants is such an important part of medicine that it is imperative it is performed in a way that its intrigue is not compromised. The Institutional Review Board Process Applying for exempt research study approval from the IRB at the University of St. Francis involves submitting a copy of the work, an application, and a statement explaining why the researcher believes the study would be exempt from expedited or full-level IRB review approval.
Over 20 years after the proclamation of these specific ethical guidelines, we are introduced to the University of Pennsylvania’s Institute for Human Gene Therapy’s study on a delivery mechanism for gene therapy that resulted in the death of an 18 year old research subject Jesse Gelsinger. Gelsinger suffered from partial OTC (ornithine transcarbamylase) deficiency caused by a defective single gene (Obasogie, 2009).
Many factors affected the changes in women’s employment. The change that occurred went through three major phases: the prewar period in the early 1940s, the war years from 1942-1944, and the post war years from around 1945-1949. The labor shortage that occurred as men entered the military propelled a large increase in women’s entrance into employment during the war. Men's return to the civilian workforce at the end of the war caused the sudden drop to prewar levels. The cause of the sudden decline during post war years of women in the paid workforce is unclear. Many questions are left unanswered: What brought women into the war industry, ...
The beginning of Gene Therapy began in the late 1980’s, which was completely unsuccessful. In the fall of 1999 the death of University of Pennsylvania trial participant, Jesse Gelsinger was followed by much public outcry and legal problems that put an immediate halt on all gene therapy research. The reason Jesse was being treated with Gene therapy was to attempt to cure the teenager’s rare liver disease.
... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today. As scientist discovers more genes and their functions, the potential of this treatment is limitless. Though gene therapy is an auspicious treatment choice for numerous diseases (including inherited disorders, some types of cancer, and certain viral infections), the procedure remains precarious and is still under study to make sure that it will be safe and effective. Thus government regulators and scientist must take a lead role in adopting a practical approach to address these issues and determining the correct procedures for dealing with them.
Before the World War II, many women only held jobs in the house providing for their children, husband, and the needs that came with taking care of the household, but during the war, this completely changed. Many women were given new opportunities consisting of new jobs, new skills, new challenges, and greater chances to do things that were once only of imagination to them. Women made the war especially possible with taking over the jobs that men would usually do, but could not do because of the war. One of the first things that encouraged women to take on jobs of the men who went off to war was the propaganda. Propaganda consisted of films, radio, and print. These advertisements used showed women fighting in the army and many working in factories. Often, the advertisements directed women in the direction to take on roles of the males.
Teutch, S., & Tuckson, R. Department of Health & Human Services, (2008). U.S. system of oversight of genetic testing: A response to the charge of the secretary of health and human services. Retrieved from website: http://osp.od.nih.gov/sites/default/files/SACGHS_oversight_report.pdf
Over 40 years ago, two men by the names of James Watson and Francis Crick discovered deoxyribonucleic acid, or DNA. DNA is hereditary material in humans and almost all other organisms (What is DNA?). From this finding, gene therapy evolved. Today, researchers are able to isolate certain specific genes, repair them, and use them to help cure diseases such as cystic fibrosis and hemophilia. However, as great as this sounds, there are numerous ethical and scientific issues that will arise because of religion and safety.
Despite being magical of gene therapy, it is high-risk. Few people got benefits from it, and it has a low rate of success. Prior to the human trial, Batshaw and Wilson had done experiment on animals to ensure the safety. Over 20 experiments have been done on mice but only 12 of them survived at last (Sophia, M. and Kolehmainen, J.D., 2000). More seriously, complicating diseases, which can be more dangerous than genetic diseases, might set in during the treatment period. In December 200...
The prohibitionist national policy towards drugs in U.S has been extremely contentious in the present times. After decades of the stance that costs billions how many each year, the paltry achievements and the countless negative externalities have led to a clamor for alternative policies instead of a “War on Drugs”.
High profile adverse events resulting in disproportionate media attention have prevented a greater difficulty for the field, with the death of Jesse Gelsinger in a trial of gene therapy for ornithine transcarbamylase deficiency undermining public trust of clinical research in the US. There is a danger that the gene therapy field may have become too risk-averse in response to these adverse events, and that this could manifest as fewer trials that take longer to commence. In the context of a research environment that is increasingly turning to the developing world for the expedient conduct of clinical trials, it is imper...
Bergeson, E. (1997) The Ethics of Gene Therapy [Online] Available at: http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm [Accessed 14 July 2011]
Due to the fact that the field of biotechnology is very serious and potentially dangerous, rules must be set down in order to keep the research in check. The high risk research of genetic therapy needs guidelines that have to be followed in order to keep the study just. The articles that are discussed in this essay focus on ethical issues and ideas that should be followed in the field in order to keep research safe and valid.
One of the biggest concerns involved in gene therapy in humans is the lack of knowledge and the possibility for consequences later on or i...