Imagine having control over all of your uncontrollable qualities; qualities that are "in your genes'' such as your height, your body type, or even a disorder. Gene therapy is simply interfering with the genome by inserting, deleting, or swapping out selected genes or gene regions, also known as loci. Alterations to the genetic makeup, genotype, of an organism via gene therapy will be reflected in its observable characteristics, its phenotype, as the cells multiply and die, and as genes are expressed. The prospects of gene therapy can range from being the next “big thing” in the cosmetics industry to saving millions of lives all over the world. Directing the research, public attention, and funding to the medical use of gene therapy is critical since it is one of the most promising technologies in the modern age, commercial or cosmetic use of it can become an aesthetic luxury like designer babies which would maim society, and the medical use of gene therapy can treat fatal diseases such as cancer.
As gene therapy is an emerging and rapidly developing technology, it has immense prospects in the modern age. As gene therapy can be performed on somatic or germline cells, the changes made can end with them or be inherited by the following generations. Although gene editing on germline cells is currently a banned practice around the world due to ethical and legislative controversy, all three forms of gene therapy (in vivo, ex vivo or in situ) on somatic cells have gone as far as giving vision to the blind or curing a disorder of the blood cells known as beta-thalassemia (“Gene Therapy Successes''). An emerging method in gene therapy is the CRISPR Cas-9 technology, which imitates an immune mechanism from a certain type of bacteria. In an article called “The CRISPR Craze” by Science Magazine George Church Ph.D., a pioneer of this technology at Harvard University School of Medicine, claims it to be much more efficient and easy to use in comparison to similar emerging methods of gene therapy. Gene therapy is already an industry worth billions of dollars, and it will only continue to grow. As technology progresses and builds on itself, what people now know as “impossible” will become regular practices.
When most people hear about gene therapy, the first thing they think of is “designer babies”, babies genetically engineered in their embryonic state to have certain cosmetic traits such as a certain eye color, height, hair type, or even superhuman qualities. Although this method seems plausible in theory, it raises countless questions on human rights, ethics and religion, or finance. “This is the first step in a well mapped-out process leading to GM [genetically modified] babies, and a future of consumer eugenics,” says David King, director of Human Genetics Alert (“British Researchers Get Green Light to Genetically Modify Human Embryos”). The genetics industry can very easily turn into a new branch of the tree which is the cosmetics industry. However, bioethicist Dr. Christopher Gynell from Murdoch Children’s Research Institute notes “[e]dited embryos won't lead to designer babies or eugenics – unless we want it to” (“GM Human Embryos Won't Create Designer Babies”). The testing alone would take years since engineered genes will likely turn out to be toxic to the cells and may even end up killing the organism. Moreover, in order for the testing to begin and for the technology to be implemented in the real world, countless ethical and political disputes need to be settled first. This may range from defining limits for the traits that can be engineered or even drafting a separate “Universal Declaration on the Human Genome and Human Rights.” The CRISPR Cas-9 technology, for example, bears great potential, and as stated, its capabilities are not limited to manipulating a few genes to make someone or their baby look more aesthetically appealing. So although the idea of customizing future generations may sound interesting, it is hazardous and definitely not a practice to be seen in the near future.
The actual future of gene therapy lies in medicine and public health. The main intention behind the development of gene therapy has always been to cure hereditary diseases and improve the quality of life. In addition to being the more virtuous outcome of the advancing technology, this goal is also supported by legislation as gene therapy for medical treatment is performed on somatic cells anyways. A great focus of the gene therapy industry has been cancer diagnosis and treatment. For instance, “The present in situ gene therapy clinical trial for human prostate cancer demonstrated safety, clinical efficacy, and biological effects of antitumor activity” (Satoh 2). Professionals are targeting certain types of cancers such as prostate and breast cancer, and developing gene therapy models apt for the loci of the selected regions. Similarly, a research paper titled “Precision cancer mouse models through genome editing with CRISPR-Cas9” notes: “In the future, the CRISPR-Cas9 system will be more finely tuned, accelerating in vitro and in vivo genome editing to establish novel cancer models and better understand cooperative effects among complex tumor suppressor gene and oncogene networks”(Mou 15). Progress and demand for the use of gene therapy in medicine are accelerating rapidly. The advancement of current technologies such as CRISPR, and the discovery of new methods over time will make way for revolutionary innovations in medicine such as more effective treatments for cancer.
It is important to remember that gene therapy is the same technology whether it is being used to make designer babies or cure cancer. However, if people manage to direct the funding and support to the correct and ethical implementation of gene therapy methods, it can be used to revolutionize medicine and public health. The technology to make designer babies exist yet scientists, bioethicists and professionals with expertise in the subject are purposefully avoiding this development due its deleterious consequences on society. On the other hand, the implementation of gene editing technologies in curing fatal diseases like cancer and beta-thalassemia, which used to be “impossible” to do, are producing great results will likely be seen as a common practice in the approaching decades.
Bibliography
“British Researchers Get Green Light to Genetically Modify Human Embryos.” The Guardian, Guardian News and Media, 1 Feb. 2016, https://www.theguardian.com/science/2016/feb/01/human-embryo-genetic-modify-regulator-green-light-research.
Chamary, JV. “GM Human Embryos Won't Create Designer Babies.” Forbes, Forbes Magazine, 30 May 2016, https://www.forbes.com/sites/jvchamary/2016/02/16/embryo-gene-editing/?sh=7aa1f54829f9.
“Gene Therapy Successes.” Learn.Genetics, 2014, https://learn.genetics.utah.edu/content/genetherapy/success/.
Mou, Haiwei, et al. “Precision Cancer Mouse Models through Genome Editing with CRISPR-Cas9 - Genome Medicine.” BioMed Central, BioMed Central, 9 June 2015, https://genomemedicine.biomedcentral.com/articles/10.1186/s13073-015-0178-7.
Pennisi, Elizabeth. “The CRISPR Craze.” Science Magazine, 23 Aug. 2013, https://www.science.org/doi/10.1126/science.341.6148.833. Accessed Oct. 2022.
Satoh, Takefumi, et al. “In Situ Gene Therapy for Prostate Cancer.” Current Gene Therapy, U.S. National Library of Medicine, 2005, https://pubmed.ncbi.nlm.nih.gov/15638715/.
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