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Introduction
Gene therapy is a genetic technology in which genes and nucleic acids (RNA or DNA) are used for the cure, treatment, or prevention of a disease. This technology can be accomplished by replacing a defective or mutated gene with a healthy copy to reduce the problem; removing or inactivating a mutated or defective gene to reduce or eliminate improper functioning, and/or introducing a new kind of gene in the body to help in the fight against disease. Initially, the technology was used for orphan diseases such as primary immunodeficiencies, but with the passage of time, scientists found its applicability in other disorders such as heart failure and cancer. In the near future, doctors would be able to treat or cure a patient by inserting a helpful gene into the patient rather than using drugs or surgery (Kaufmann, Büning, Galy, Schambach, & Grez, 2013).
The process of gene therapy can be done with the help of advanced technological processes and tools such as viral and non-viral vectors (carriers) (Kaufmann et al., 2013). These processes are used as a directly inserted gene into a cell would not be able to work properly. Certain viruses can work as vectors as they can help in delivery of the new gene into the cell by infecting it. For example, retroviruses can integrate new gene (along with the other genetic material of the virus) into a human chromosome, and adenoviruses introduce the DNA into the nucleus. Those viruses are modified through genetic engineering, so that they would not cause any disease in people. These vectors are either injected intravenously into a particular tissue in the human body from where they go to the individual cells, or they are exposed to cells in a laboratory setting and then the cells are returned to the patient (Genetics Home Reference, 2016).
Gene therapy can be used for somatic cells, which are involved in developing organs such as liver, heart, skin, etc. and reproductive cells, such as sperm cells, eggs, and cells in the very early stages of embryo. Changes in somatic cells do not pass from one generation to the next generation, but changes in reproductive cells could pass to the next generation (University of Missouri, n.d.).
Social and Ethical Implications
Many researchers from different parts of the world are working on gene therapy but still the risks associated with the disease are unpredictable. Moreover, various studies have shown serious health risks associated with the technology, such as inflammation, toxicity, and cancer. Therefore, comprehensive federal policies, regulations, laws, and guidelines have been presented to protect people from bad impact of the research studies. The U.S. Food and Drug Administration (FDA) and the National Institutes of Health (NIH) ensure the safety of research on gene therapy. Strict protocols and/or plans have been developed to deal with the medical, ethical, social, and/or safety issues (Genetics Home Reference, 2016).
Gene therapy is related to making changes in the basic set of instructions in the body; therefore, it can lead to various ethical and social issues. Those issues are related to the decision about “good” or “bad” uses of genetic therapy; definition of normal and/or disturbed characteristics; high costs of gene therapy; acceptance of people, who have gone through the process of gene therapy in the society, and the use of gene therapy to improve the basic human abilities such as intelligence, height, or athletic ability (Genetics Home Reference, 2016).
Presently, gene therapy is related to the treatment of individuals by targeting their cells such as blood cells or bone marrow. Although this form of treatment could be of significant help, ethical issues arise when gene therapy is targeted to germ cells (eggs and sperm cells) that could help in passing the inserted gene to the next generation. This process is referred to as germline gene therapy. This therapy could help a family from getting rid of genetic disorders, but it is controversial as it could result in the development of a fetus in an unpredictable manner or may result in unwanted side effects that are still not known (Genetics Home Reference, 2016).
One of the most important social implications related to the use of gene therapy, especially germline gene therapy, is the responsibility that may come with the individual. For example, in the absence of germline gene therapy, a person has no responsibility of his or her genes as they are selected by nature. On the other hand, in case of germline gene therapy, huge responsibility comes on the shoulders of people involved in the therapy, and if they do not fulfill their responsibility, society may suffer from unknown and serious outcomes (United States. Congress. Office of Technology Assessment [OTA], 1984).
Another issue is that with gene therapy, humanity can affect its own evolutionary process that is helping in dealing with the natural problems (United States. Congress. Office of Technology Assessment [OTA], 1984).
Personal Viewpoint
Gene therapy is just like other advancements in technologies that is directly affecting the abilities and characters of people. Although it can help in the development of novel therapeutic strategies for people, it can also result in several new responsibilities. For example, it can affect the decisions made by doctors and medical researches that must be in favor of not only the individual but also the society. Socially, the gene therapy can also affect the basic reproductive freedom. Due to gene therapy, people may start finding mates with better ability of reproducing smarter and attractive kids. This would affect the reproductive freedom, thereby affecting the whole society. These implications of germline gene therapy could result in severe discriminations in a society, i.e. people with certain disabilities would not be able to fit themselves properly in the society.
It is important to note that with appropriate guidelines and policies, gene therapy can help in improving the lives of a huge number of people in the world. If we consider the social and ethical issues related to gene therapy, it can be found that several issues could also develop in other therapeutic interventions such as acceptance of surgery and the use of antibiotics. If those therapeutic interventions are commonly used in the present day, gene therapy can also be used with appropriate precautions. Although several side effects and deaths have been reported in the research of gene therapy, further studies are still required to confirm the root causes of side effects and deaths. In this regard, medical institutions, medical researchers, and regulatory authorities can work together to make the gene therapy as safe as possible. Researchers can work on the improvement of cell targeting by a vector. Moreover, they can work only on those disorders, which are still incurable or unresponsive to treatments.
References
Genetics Home Reference. (2016). Help Me Understand Genetics - Gene Therapy. Retrieved from https://ghr.nlm.nih.gov/primer/therapy.pdf
Kaufmann, K. B., Büning, H., Galy, A., Schambach, A., & Grez, M. (2013). Gene therapy on the move. EMBO molecular medicine, 5(11), 1642-1661.
United States. Congress. Office of Technology Assessment [OTA]. (1984). Human Gene Therapy: Background Paper: Washington, DC: U.S. Government Printing Office [GPO].
