The Gene Therapy Development and Purpose Essay

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Gene therapy is a promising vector in modern medicine that has been actively researched for a few decades. This field of scientific knowledge has the potential to cure many acquired and inherited diseases. Although studying gene therapy has faced many challenges over the years, scientists worldwide put much effort into uncovering its full potential, which is why this medical treatment is rapidly developing.

The interest in gene therapy can be explained by its intended purpose: achieving a significant clinical benefit with a single treatment. The first steps in studying this field were taken almost fifty years ago when an American researcher Theodore Friedmann evaluated the potential and challenges related to the usage of gene therapy (as cited in Dunbar et al., 2018, p. 2). It can be highly effective in curing neurodegenerative and immune disorders, hemophilia, different forms of cancer, and other illnesses. However, this form of medicine also has many disadvantages, which is why it is not approved in many countries. The first trials of gene therapy “exposed serious therapy-related toxicities, including inflammatory responses to the vectors and malignancies caused by vector-mediated insertional activation of protooncogenes” (Dunbar et al., 2018, p. 1). In order to deal with the associated issues, additional research is conducted in cell biology, immunology, and virology (Dunbar et al., 2018). Scientists continue to explore gene therapy and seek new ways to make it as efficient and safe as possible.

However, the therapy under discussion is associated with a significant problem related to informed consent. The disadvantages of gene therapy may affect the patient and their entire bloodline due to possible germline mutations (Dunbar et al., 2018). Genotoxicity is a serious issue, and many patients may fear it and reject therapy even though it can liquidate their most severe diseases. Therefore, informed consent will remain a crucial problem until the methods to stabilize the germline and prevent negative consequences of gene therapy appear.

There is also a problem of implanted genes being passed on to the next generations. Nowadays, heritable germline editing is a realistic possibility due to the technological advancements in this area, yet many people do not find it an advantage (Dunbar et al., 2018). An international group of scientists and other professionals published a report in 2017 revealing a possible pathway to correct germline mutations (as cited in Dunbar et al., 2018). It would mean that the negative consequences of implanting genes could be reversed. However, the federal governments of different countries have put many restrictions on gene therapy as there is currently no guaranteed method to prevent implanted genes from being passed on to the next generations.

I think gene therapy is ethical since it aims to cure severe illnesses. Having cancer or a neurodegenerative disorder is worse than having an edited genome. Furthermore, heritable germline editing can decrease future generations’ chances of inheriting a chronic disease from people who have undergone gene therapy. Still, I do not think that people will “design” their babies in the future, as it is not the initial purpose of gene therapy. It is a medical instrument intended to cure diseases and help people who suffer from them. While that is true, I do not see an ethical issue in gene therapy, but using it for “designing” babies seems unethical at all points.

Reference

Dunbar, C. E., High, K. A., Joung, J. K., Kohn, D. B., Ozawa, K., & Sadelain, M. (2018). Gene therapy comes of age. Science, 359(6372), 1-11. Web.

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