Biological Basis of Gene Therapy
Ginn et al. (2013) asserted that gene therapy aims at inserting genes into the host cells and tissues of an individual to treat a disease by replacing the defective mutant allele with a functional one. The principle of the technology is to salvage the mutant phenotypes by introducing normally functioning cells to avoid the progression of the infection. Some of the diseases that are currently being studied for possible gene therapy include hemophilia, severely combined immune-deficiency, Parkinson's disease, different types of cancers, and even HIV/AIDS (Ginn et al. 2013). The gene is primarily delivered into the host cell using a carrier called a vector. In most circumstances, viruses are employed as the preferred vectors. Also, important to note is that the viruses used as vectors must undergo attenuation to enhance their safety in the body tissues. The process of gene therapy is still in its infancy, but currently, three major approaches to the gene therapy are being investigated. First involves a process where a mutated gene is replaced with a healthy copy of the gene. The second significant process under investigation involves ‘'knocking out'' or inactivating a mutated gene that does not function properly. The third approach consists of the introduction of a new gene into the body which will work to fight the disease.
Van Dongen, El Hejazi, and Claassen (2017) illustrated that the vectors could be divided into two including the viral vectors and the non-viral vectors. As earlier intimated, viruses are the most preferred vectors but have to undergo a process of inactivation to avoid causing infection in the host cells. The viruses have a mechanism of delivering their genes into the cells of the human being pathological. As such, scientists have utilized this ability to their advantage in actualizing the gene therapy. The viral genome is manipulated with the desirable therapeutic genes while the disease-causing genes are removed. On introduction, the target cells, say the liver or the lung cells get infected with the vector which then proceeds to unload the genetic material containing the therapeutic genes into the target cells. Through the process of transcription and translation, a functional protein is released from the gene that acts to restore the target cell back to its normal state. All cells in the body have genes making them potential sites for gene therapy. The body cells are either somatic, which accounts for the majority, or can also be germline cells including the sperms and eggs. Gene therapy that targets the germline cells can result in permanent changes passed to the generations to come. The same case is not true for gene therapy on the somatic cells.
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Ginn et al. (2013) further noted that gene therapy in somatic cells could take two fashions including in vitro and in vivo. Vitro means a process where the cells are modified out of the body and subsequently transplanted in again. Some clinical trials have involved bone marrow or blood have been removed and grown in the laboratory. The cells are then exposed to the virus which carries the desired genes. The virus finds its way into the cell and releases the therapeutic genes into the cell’s DNA. The cells continue with their growth in the laboratory before they are returned to the patient via injection. The gene therapy is called ex vivo or in vitro since the cells are remedied outside the human body. In vivo gene therapy involves a situation where the genes are altered while in the body as earlier described.
Social and Ethical Implication
The biological tenets that interplay in gene therapy are extremely complex and therefore thorough research needs to continue for the process to be deemed safe. Many ethical concerns are raised given the fact that gene therapy involves changes to the genetic make-up. Scientific and ethical concerns regarding the process continue to cause varied reactions from various quarters. The first approved gene therapy clinical trial occurred in 1990 (Powell, & Buchanan, 2011). The clinical trial was considered a success because it improved the health of several people who participated in it. However, these patients continued to receive the traditional drug therapy hence making it difficult to understand the value of the gene therapy as a treatment strategy. One of the most significant ethical debates that have cropped up in the wake of the gene therapy regards the issue of germline gene therapy. Since introducing changes in the germline cells has a potential of changing the traits of an individual throughout his generations, the primary challenge that could emerge is people using the process to enhance fundamental human traits such intelligence, height, and athletic ability among others. Although it appears as a factor that should be welcomed, many ethicists, especially from the religious side, see this as a deviation from human nature as planned by God.
Another social issue that is likely to emerge with the gene therapy issue regards its cost. It is obvious that gene therapy would be an expensive process because of the scientific endeavors required to make it a success. As such, Powell and Buchanan (2011) noted that people fear that it will only be a medical remedy available for the rich. Thus, the implication is that more disparity will be created due to inaccessibility of medical intervention due to the cost. It would also be critical to note that gene therapy is a process involving the changing of the body's critical instructional processes. Therefore, there is a question of whether the increasing use of gene therapy would put the society in a position where it least accepts people who are different. Furthermore, there is a massive debate going on regarding the vectors used in the process of gene therapy. Because most of them are viruses which are potentially virulent, there are fears that the process could enhance the spread of diseases because the viruses can regain their potency while inside the human cells. Sociologists and ethicists continue to question how good and harmful use of the gene therapy can be distinguished because of the possibility of abuse.
Personal Viewpoint
Before illustrating my viewpoint, it would be first critical to appreciate that there are two sides in the controversial debate about gene therapy. The process can be advantageous because it potentially seeks to remedy some of the genetic diseases that have been difficult to treat via conventional medication. However, significant social and ethical concerns center on the veracity of the process, its safety, and the prospect of abuse. Despite the fact that there is minimal evidence supporting the feasibility of the process in solving medical problems, what remains apparent is that it is scientifically viable. Viruses can effectively be attenuated as seen in the cases of vaccines and inserted into the human body without any fear of resurgence. Therefore, my view is that gene therapy should be given a chance in the field of medicine given that most pharmacological drugs have been unable to treat most genetic infections and other life-threatening diseases like HIV/AIDS. The sociological and ethical issues can be addressed by the relevant medical organizations which will enhance continuous research to ensure that no adversities emerge from its incorporation in medical use. The medical association will also ensure that also ensure that the gene therapy is strictly used for medical reasons in addressing one of the major concerns of the ethicists and sociologists who feel that it would be bound to abuse.
References
Ginn, S. L., Alexander, I. E., Edelstein, M. L., Abedi, M. R., & Wixon, J. (2013). Gene therapy clinical trials worldwide to 2012–an update. The journal of gene medicine , 15 (2), 65-77.
Van Dongen, P., El Hejazi, Z. N., & Claassen, E. (2017). Analyzing Patent Terms and Citations to Determine the Value of Gene Therapies. Journal of Commercial Biotechnology , 23 (2), 61-73. Doi: 10.5912/jcb777
Powell, R., & Buchanan, A. (2011, February). Breaking evolution's chains: the prospect of deliberate genetic modification in humans. In The Journal of Medicine and Philosophy: A Forum for Bioethics and Philosophy of Medicine (Vol. 36, No. 1, pp. 6-27). Oxford University Press.
