Gene Therapy Implications And Significance Essay


Discuss about the Gene Therapy Implications And Their Significance.


Gene therapy refers to a scientific technique whereby defective genes are replaced by normal genes in an attempt to treat or prevent a disease. The defective genes are replaced by normal ones that enable the body to function properly preventing negative conditions that are related to genes for example baldness and sickle cell disease. It is mainly used for diseases that hardly have any cure. According to Zhang et al (2016), it can be used in some types of cancers to reverse malignancies and certain viral infections. Although it is a considerable option in such conditions, gene therapy has been faced with a lot of criticism in our world today since it involves a lot of risk taking on the health of a person as it is an experimental technique. There are several social, ethical and legal implications that revolve around gene therapy. The aim of these discussion is to explain these implications and their significance.

Gene therapy can not only be used to correct disease causing genes but also genes that are responsible for traits such as tall and short statue (Georgiadis, et al (2016)). However there is no clear cut distinction regarding which gene therapy is meant for disease genes and that which is meant for the other traits unrelated to diseases. There has been both positive and negative views in the society concerning gene therapy. The positivity comes about when a gene therapy conducted brings about the preferred outcome of an individual. For example gene therapy has been used lately to provide cure to gene related diseases and this has improved health provision (Jackson, et al (2015)). An example of a gene related disease that has proved to be reversed by this form of therapy is the sickle cell disease. People suffering from this disease make abnormal versions of hemoglobin which is responsible for oxygen transport within the body. This condition can be caused by mutation of the beta globin gene that is responsible for forming subunits of hemoglobin. Scientific correction of the mutated gene through gene therapy can reverse the condition leading to normal production of hemoglobin.

There has been several social and ethical issues revolving around gene therapy and genetic engineering as a whole. Critics have argued that interfering with the genetic make-up of someone else is like playing God and though its science, it should not be acceptable in the society. As stated by Naldini, (2015), it’s also considered as an expensive way to cure some diseases which others might argue if it is beneficial it should be accessible to all. The thought of generating medicine and vaccines from modification of microbial has been welcomed since it has proven to be effective in disease prevention. Genetic engineering has also been widely used to increase plant and animal food production and this has been received well. However, although there are many advantages of gene therapy, there are quite a number of ethical issues raised by the same.

Since it is an experimental field, consent from the subject must be sought before introducing the gene therapy. The subject who is seeking cure or improvement of traits should be willing without being cohered to be done the therapy onto. As argued by Alton et al (2015), the introduction of the normal functional genes into a person may end up replacing important genes instead of the mutated ones since there are no clues as to where they are being placed. This could worsen a condition instead of curing it or lead to a new undesired negative condition. Therefore before such therapy is conducted the benefits should be considered to outweigh the risks associated to avoid harm onto the patient or subject. The correction of defective genes could also create some side effects which are negative. It could result into some allergic responses since new genes are being introduced or modified within the body.

In conclusion it is correct to say that some people have created a negative opinion on the subject based on religious views. Others have been against gene therapy due to lack of knowledge about its benefits with their focus being primarily on the negativity. The legality of gene therapy varies from country to country (Thwaite et al (2015)). However it universally revolves around the Belmont report that contains the principles of bioethics. The principles include respect for persons whereby any individual or subject to gene therapy should be treated as a person who has rights and the fact that their life comes first therefore anything done to them should be towards their well-being and safety. The second principle revolves around beneficence whereby the focus on the subject should be minimizing the harm or risk and maximizing on the benefit of the patient. The last principle focusses on distributive justice whereby in any research, burdens and benefits should be shared equally.


Alton, E. W., Armstrong, D. K., Ashby, D., Bayfield, K. J., Bilton, D., Bloomfield, E. V., ... & Carvelli, P. (2015). Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial. The Lancet Respiratory Medicine, 3(9), 684-691.

Georgiadis, A., Duran, Y., Ribeiro, J., Abelleira-Hervas, L., Robbie, S. J., S?nkel-Laing, B., ... & Bainbridge, J. W. B. (2016). Development of an optimized AAV2/5 gene therapy vector for Leber congenital amaurosis owing to defects in RPE65. Gene therapy, 23(12), 857.

Jackson, K. L., Dayton, R. D., Orchard, E. A., Ju, S., Ringe, D., Petsko, G. A., ... & Klein, R. L. (2015). Preservation of forelimb function by UPF1 gene therapy in a rat model of TDP-43-induced motor paralysis. Gene therapy, 22(1), 20.

Naldini, L. (2015). Gene therapy returns to centre stage. Nature, 526(7573), 351.

Thwaite, R., Pages, G., Chillon, M., & Bosch, A. (2015). AAVrh. 10 immunogenicity in mice and humans. Relevance of antibody cross-reactivity in human gene therapy. Gene therapy, 22(2), 196.

Zhang, L., Zheng, W., Tang, R., Wang, N., Zhang, W., & Jiang, X. (2016). Gene regulation with carbon-based siRNA conjugates for cancer therapy. Biomaterials, 104, 269-278.

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