Gene Therapy: A Step Into The Future

Updated: May 17

Genes contain the biological instructions that dictate every part of our biological composition from the color of your eyes, how tall you are, and even your behavior (Britannica, 2020); they are units of DNA that code for proteins that essentially tell the cells of your body what to do and when to do its essential functions. However, improper gene expression can lead to a host of harmful diseases known as genetic disorders. Genetic disorders can be caused by a mutation in one or multiple genes, by a combination of gene mutations and external factors, or by damage to chromosomes, the structures which carry genes (NIH, n.d.). Genetic disorders such as cystic fibrosis, down syndrome, and sickle cell anemia affect all populations and body systems with approximately 60% of people suffering from a genetic disorder (WA, n.d.). However, a new treatment known as gene therapy is seeking to specifically target and lower the prevalence of such diseases.

Gene therapy is unique in that instead of targeting a problem’s symptoms it attempts to treat a disease from its underlying genetic cause. Gene therapy functions in 3 primary ways: replacing mutated genes, inactivating harmful genes, or introducing a healthy gene into the body. Research in gene therapy has made bold strides in recent years and has shown promise in a variety of clinical trials. In particular, gene therapy has been hypothesized to be a promising treatment for heart attack and Leukemia patients.

Heart disease is currently the leading cause of death in the United States and continues to be a growing health concern worldwide (Virani, 2021). Gene therapy offers an alternative issue for this often fatal illness by genetically turning off certain pathways that directly lead to heart failure. Such clinical trials have shown incredible promise in improved heart function and the recovery of heart pumping following a heart attack (Shasteen, 2022). Another promising treatment named SDF-1 seeks to remodel the heart by recruiting stem cells, or cells that do not have specified purpose yet, to encourage new cell growth at a site of injury caused by a heart attack (ESC, 2015).

Leukemia is a cancer of blood tissues, namely the bone marrow. Leukemia is a pressing health concern in the United States with over 397,501 people currently living with the disease in the United States (LLS, n.d.) . Leukemia has traditionally been treated with a series of intensive interventions such as Radiation, Chemotherapy, and Bone marrow transplants. However, clinical trials for gene therapy have been found to be a highly promising solution to this disease. One such FDA approved treatment is for leukemia in children and young adults, that allows modification of one’s own immune system cells to include a new gene that specifically targets and kills harmful Leukemia cells. This form of therapy was found to be effective in leukemia patients for 10 years and counting (Penn Medicine, n.d).

Genes are the fundamental unit of life and play a pervasive role in each and every organism’s life. New forms of gene therapy are theorized to treat a variety of illnesses including inherited disorders, viral infections, and even cancer (New England Journal of Medicine, 2022). In the future, gene therapy can even anticipate and fully prevent the existence of certain diseases in newborns. However, the ethics of gene therapy is often argued as its side effects on fetal development have not yet been completely studied. In addition, the boundaries of this technology are still unclear: how does one draw the line between disease prevention and uncontrolled gene modifications by the privileged? Though many questions still exist, ss the frontier of science expands, so does the promise of gene therapy in treating many of the world’s most harmful diseases.


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