Tiffany Chung, 11th Grade

Currently, over 250 million people worldwide live with a genetic disorder, with the majority lacking any FDA-approved treatments (1). Enter genetic engineering – a rapidly advancing field of biotechnology that allows manipulation of genes to address various health issues, including diseases and cancer.

The prospect of genetic engineering seemed far-fetched during its conception in the early 20th century. From the early stages of using recombinant DNA technology for gene editing, newer techniques have built upon previous techniques with the hopes to deliver novel therapies for patients (2). Upon completion of the Human Genome project and rapid advances in the field, gene engineering has since transcended beyond the confines of imagination towards a tangible reality.

Fortunately, scientists have been able to discover new ways to treat illnesses as they continue to threaten individuals’ lives and in this case, through gene therapy. Patients affected by beta-Thalassemia have an error in the beta-globin gene, which is responsible for an oxygen-carrying protein in red blood cells and the majority of them will require blood transfusions in order to survive (3). Therefore, they lack a sufficient amount of red blood cells resulting in inadequate oxygen transport to all of the body’s tissues (3). A patient with a severe case of this genetic defect underwent gene therapy in which blood stem cells taken from the bone marrow were treated with a retrovirus to reintroduce a functional copy of the beta-globin gene into the patient’s body where they successfully began producing healthy RBCs (3). Seven years subsequent to the procedure, the patient remained thriving without the need for any blood transfusions (3).
Genetic engineering also proves to be beneficial as a cancer treatment, T-VEC, uses a virus that has been manipulated to destroy only cancer cells, prevent cold sores, and also emit signals that draw the patient’s own immune cells, aiding them in being able to recognize and attack cancer cells throughout the body (3). Subsequent to the virus’s direct injection within the patient’s tumors, the cancer cells burst, further releasing viruses that can potentially infect other cancer cells (3).

With new methods of genome editing in development, opportunities for advances in medicine emerge as well, offering the potential to improve human health. Due to its efficiency, speed, and low cost compared to other gene editing methods, the CRISPR-Cas9 system has been gaining traction within the scientific community (4, 5). It is currently being used to modify DNA for medical purposes in which a small piece of RNA with a short “guide” sequence connects to a particular target sequence in the cell’s DNA as well as to the Cas9 enzyme (5). This enzyme then cuts the DNA at the intended position after being recognized by the guide RNA and researchers can edit the DNA by replacing a section with a customized DNA sequence or add or delete segments of genetic code using the cell’s own DNA repair mechanism (5).
Despite ongoing research on CRISPR/Cas9, its potential was demonstrated in an experiment conducted by He Jiankui, who worked with the world’s first gene-edited twins (6, 7). Using CRISPR technology, Jiankui was able to impair the CCR5 gene that facilitates HIV infection, allowing for the twins to be born HIV- free despite their father being infected (7). However, the experiment was not legally supported by Chinese regulations and faced public criticism (7). Furthermore, with several successful and conventional methods for preventing HIV infection along with the fact that sperm washing had been utilized to ensure that only the aseptic DNA was used, editing the twins’ genes was unnecessary (6, 7). Thus, altering early embryos presented no benefits for the babies, and in contrast, generated potentially considerable risks. One of the major limitations surrounding genetic engineering in developing embryos is mosaicism, where there is a significant chance that other embryonic cells may carry undesirable mutations which may result in unanticipated consequences (6). Furthermore, genome editing also raises the ethical question of whether parents have the right to make the decision for their embryo since technically, they would be considered the patient. Although some individuals believe that it is unfeasible to acquire informed consent for germline therapy, others argue that important findings can still be derived as a result of these studies that can potentially be applied towards other cases.

In addition to the controversy surrounding genetic engineering in embryos, the commercialization of therapies may exacerbate the existing disparities in healthcare (8). Some fear that if used to its maximum capabilities, germline editing and other techniques may establish the creation of distinct classes based on how well their engineered genome is (8). The healthcare gap is especially evident in rural communities, as a 2006 survey indicated that 25% of children living were not receiving access to proper medical care (9). One of the major factors determining whether or not a patient seeks medical treatment is insurance, and those from high-income backgrounds were found to be twice as likely to receive treatment for genetic diseases (9). The impact of genetic engineering is only as effective as its implementation, and in order to ensure equity in healthcare, there must be policies in place to eliminate the barriers of receiving proper treatment.
With its extensive applications, genetic engineering has the potential to revolutionize the future of medicine. Yet with current ethical concerns surrounding this topic, there are limits that researchers must consider. Despite its limitations, genetic engineering can be more widely viewed as a safe approach used to enhance the quality of human life and improve daily function while decreasing the prevalence of major disorders and diseases. Public hesitancy is reasonable given that it is a relatively new field. To diminish concerns, greater research transparency and promising findings may gradually increase acceptance towards this groundbreaking technology. Genetic engineering still has a long way to go, but by fostering transparency and cooperation, we can work towards a world where genetic engineering fulfills its potential as a tool to positively impact medicine and offer hope for a healthier future.