Gene therapy

When exploring the historical context of gene editing, one must trace the timeline of scientific advancements that created a foundation for groundbreaking technology like CRISPR-Cas9 to be invented. The earliest of these advancements was the discovery of the structure of DNA, known as the double helix. This discovery led scientists and researchers to learn more about DNA sequences and how they are passed from parent to offspring. This era of genetic discovery allowed for researchers to gain a better understanding of the basic workings of life and to come to the realization that what might seem like small changes in DNA sequence can be the deciding factor between a healthy life or diseased one. This is where the idea of genetic modification comes into play as the scientific community discovered the root, so to speak, of many diseases and began speculating as to how to fix genetic “mistakes” through technology.

By the 1970s and 1980s, modern technologies of the time allowed for the first successful genomic modifications which were created in yeast and mice, raising questions about when something like this could be used to change the human genome and whether it was ethical. Scientists were able to simulate alterations in DNA, and much like Darwin with the process of descent with modification, they got a deeper understanding of how genetic changes can have an impact on passed traits. Eventually, in 2012, a cutting-edge genetic modifier known as CRISPR-Cas9 was discovered and refined, advancing to the point that it became possible for researchers to precisely remove and insert certain sections of DNA wherever necessary. The issue of gene therapy is not found in research contexts, but concerns about the possible consequences rose in therapeutic or clinical settings as there are many challenges and ethical dilemmas that are associated with gene therapy. While, theoretically, gene therapy may be the solution to many diseases and conditions which previously seemed unstoppable, there is a vast amount of risks and complexities that are directly associated with gene therapy. The scientific community does not have much research into how this could affect the patients or their offspring, possibly resulting in alterations to human evolution. This causes difficulties in using this technology to its full capacity and it creates a debate surrounding the ethical implications of this practice.

Advocates of genetic therapy highlight its massive potential to effectively treat debilitating genetic diseases at the root, providing many patients with little to no treatment options a chance to do more in the fight against their condition. This side of the debate aims to correct genetic “mistakes” or “defects” through genetic therapy and allow for increased research in an attempt to get an idea of how the technology can be used to the best of its ability. Essentially, the argument being made is that the risks and possible consequences of the practice is outweighed by the possible positive effects that could result from implementing gene therapy into a society. Those in favor see this sort of technological advancement as something of the future, going against religious implications as they attempt to play god and fix these “mistakes”.

In contrast, those against gene therapy argue that the risks and possible consequences that are associated are not worth practicing in a clinical or therapeutic setting let alone integrated into a society. With how little we know about the possible negative effects of gene therapy as well as what we do know about how it could enormously affect the genome of patients as well as their offspring, potentially altering human evolution or worse, those on this side of the debate would rather be safe than sorry when it comes to those affected and how it would affect future generations and societies. With the rate that humanity is advancing, perhaps there will come a day where many have a change of heart due to safer technologies and a more complete understanding of the inner workings of gene therapy, though an issue lies in the ethical and moral implications with increased research, slowing down the process.

Similar to those engaging in the debate, a reductionist point of view on the issue of gene therapy as a practice in society varies based on individual values. Though, a reductionist may break the concept down simply in the sense that those who highly value technological and societal advancement would be in favor rather than those who value ethics and morals as well as preservation of natural human life as we know it. Additionally, a reductionist may consider alternative arguments that lie between those against or in favor, such as considering allowing for gene therapy research to be practiced in certain settings with certain methods in an attempt to mitigate the risks associated while also allowing effective research on gene therapy.

With the technologies that are capable of gene modification and how little is known about the adverse effects that may result, the only “technological fix” seems to come from advancements in technology that we have not made yet. Similar to the reductionist thought on the issue, the accepted answer lies within both sides of the debate which involves ethically progressing our understanding of genetic modification through extensive research without real testing. This mainly has to do with the consensus of the researchers as well as the public.

Every society struggles with the problem of minimizing those with debilitating conditions and disease, largely because it is a unique problem that can have little to no treatment in many cases. Humans have an innate responsibility to take care of the sick or those who need medical help, and developing technologies for this cause is always a priority. Advocacy for technologies with the ability to limit these genetic “mistakes” has persisted through time until it comes at the expense of others. For those who have heard the opinions of researchers, the decision against gene editing in a clinical and therapeutic setting is easy, even when shown the positive effect that it could have.

Clearly, those against gene therapy are winning the debate, which conveniently coincides with the stances of most who are involved in the decision making, though it limits the ability for researchers to learn more about its potential. It is no wonder why the people working toward making this technology possible are being so hesitant as the reward for expanding on research is so great but losing the support of the public can be a deciding factor. The current state of what is known about gene therapy is limited to a certain degree as a result of these societal factors.

The social pressures combined with the scientific potential surrounding this issue creates a dynamic which allows for a lot of speculation by society. In theory, gene therapy could revolutionize modern medicine in a way that society has never seen before, opening up a lot of possibilities regarding applying this to enhancing or removing one's desired characteristics and even being able to boost performance. With the idea of gene therapy, there is also the debate as to who will have access to it, bringing up themes of inequality among those who do not get access as well as how accessible it would be. Additionally, there is the argument that these so-called genetic “mistakes” found in a patient's DNA are not necessarily mistakes or meant to be changed at all, connecting to religion and the idea of playing god when manipulating key aspects of nature.

With all of the extraordinary possibilities that come with gene therapy, the extreme potential that this branch of science holds is undeniable and the question of whether or not it is truly possible will be answered with time. If integration is successful, meaning that society accepts genetic editing as commonplace, it could result in massive genetic change in the human race on a large scale. Being able to effectively and efficiently manipulate one of humanity's most fundamental building blocks of life would not only change the lives of many within society for the better but effectively have a positive impact on the lives of future generations to come.

References

“What Are the Ethical Concerns of Genome Editing?” Genome.Gov, www.genome.gov/about-genomics/policy-issues/Genome-Editing/ethical-concerns. Accessed 5 Dec. 2023. Ayanoğlu, Fatma Betül, et al. “Bioethical Issues in Genome Editing by CRISPR-Cas9 Technology.” Turkish Journal of Biology = Turk Biyoloji Dergisi, U.S. National Library of Medicine, 2 Apr. 2020, www.ncbi.nlm.nih.gov/pmc/articles/PMC7129066/. Davies, Benjamin. “The Technical Risks of Human Gene Editing.” Human Reproduction (Oxford, England), U.S. National Library of Medicine, 1 Nov. 2019, www.ncbi.nlm.nih.gov/pmc/articles/PMC6913216/. “Gene Editing.” Encyclopædia Britannica, Encyclopædia Britannica, inc., 5 Dec. 2023, www.britannica.com/science/gene-editing. Carroll, Dana. “Genome Editing: Past, Present, and Future.” The Yale Journal of Biology and Medicine, U.S. National Library of Medicine, 19 Dec. 2017, www.ncbi.nlm.nih.gov/pmc/articles/PMC5733845/. Public Engagement - Human Genome Editing - NCBI Bookshelf, www.ncbi.nlm.nih.gov/books/NBK447279/. Accessed 6 Dec. 2023. Howard, Heidi C, et al. “One Small Edit for Humans, One Giant Edit for Humankind? Points and Questions to Consider for a Responsible Way Forward for Gene Editing in Humans.” European Journal of Human Genetics : EJHG, U.S. National Library of Medicine, Jan. 2018, www.ncbi.nlm.nih.gov/pmc/articles/PMC5839051/.

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