Gene Therapy: The Future of Medicine
What if scientists could solve any biological or genetic dysfunction at its source? How transformative would that be? No more ineffective medication, no more risky surgeries, no more physically and emotionally draining treatment. With one procedure, patients with dysfunctions, including those with life-threatening diseases, could be cured for life. This would be an incredible advancement that would completely revolutionize medicine and benefit people worldwide. An innovation of this sort is emerging in the scientific world through gene therapy.
What is Gene Therapy?
Gene therapy is a technique that attacks a malfunction in the body at its source by altering a gene’s biological properties, thereby treating or curing the malfunction. Genes and their protein production change as a result of age or environmental exposure. Often, cells can recognize this change and repair themselves. However, if that fails, a disease or disorder can emerge. This would require treatment, but with a method like gene therapy, the condition can be treated at its source by fixing the malfunctioning gene. Gene therapy enables the correction of errors in a body’s blueprint, rather than treating the symptoms that result (Cleveland Clinic, 2023).
The genetic material delivered influences how a cell produces proteins. Gene therapy seeks to reduce disease-causing proteins, boost the production of non-disease-causing proteins, or create new modified proteins depending on the situation (Cleveland Clinic, 2023).
The Cell Process
To put this all in context, a cell’s nucleus stores chromosomes, which are made up of deoxyribonucleic acid (DNA) and carry genetic information in the form of genes, which determine traits. Genes are what produce instructions for making proteins, which are one of the main controls for body functions. A small change in DNA alters how proteins operate, which gene therapy aims to do to correct any issues within the body (Cleveland Clinic, 2023).
Performing Gene Therapy
There are two main ways to administer gene therapy: in vivo and ex vivo. An in vivo procedure involves injecting genetic material directly into the body, and an ex vivo procedure involves removing target cells from the body, applying gene therapy to these cells, and then returning the modified cells to the body (Food and Drug Administration, 2018).
The genetic material is packaged into a delivery vehicle known as a vector to reach its target in the body. There are several different gene therapy products to transport genetic material, including:
Plasmid DNA: Circular DNA molecules that can be genetically modified to carry therapeutic genes to human cells.
Viral vectors: Viruses, which have the natural ability to deliver genes throughout the body, are removed of their infectious disease to be used as gene therapy vehicles.
Bacterial vectors: Once modified to remove the ability to cause disease, bacteria can be used as a vector (Food and Drug Administration, 2018).
Gene therapy works in three main ways: it can replace a disease-causing gene with a healthy copy or simply add a healthy copy (gene addition); deactivate a malfunctioning gene (gene silencing); or introduce a modified gene that can treat the disease or directly modify the target gene (gene editing) (Food and Drug Administration, 2018).
The various approaches to gene therapy require different procedures and vectors. In the case of gene addition, inserting a new copy of a gene that contains instructions to produce more of a necessary protein, Adeno-Associated Viruses (AAVs) are often used (Cleveland Clinic, 2023). AAVs are viral vectors with ideal properties for gene therapy, such as their small size and inability to cause human diseases (Barbieri, 2024).
Another approach to gene therapy is gene silencing. This is when genetic material is used to prevent the activity of a gene that is already in a cell, targeting messenger ribonucleic acid (mRNA) to decrease protein production in the cell (Cleveland Clinic, 2023). mRNA is involved in protein synthesis, in which its role is to transfer information stored in DNA in the nucleus to the protein-making organelles in the cytoplasm (Sen, 2025).
The final method of gene therapy is gene editing. Gene editing is the process of modifying DNA by altering the genes it contains to correct the proteins it produces. DNA is often modified using a technology called CRISPR (Cleveland Clinic, 2023), which stands for “clustered regularly interspaced short palindromic repeats” (Smith, 2025). An enzyme called Cas9, for CRISPR-associated protein (IGI, 2025), scans the DNA, looking for a specific sequence that the gene editing is targeting (Smith, 2025). Cas9 acts like a molecular pair of scissors that can cut into DNA to allow for the alteration of the genetic sequence (IGI, 2025).
Pros and Cons
Gene therapy poses several exciting benefits. Primarily, it is a new and hopeful treatment option for diseases that do not have other options and could potentially lead to death. There is promising research on this topic that suggests gene therapy can slow or halt the progression of diseases. However, there are still risks associated with gene therapy. First of all, despite promising results thus far, gene therapy is still in the midst of its research. This means that there are still chances that it will not actually be effective, or that people who underwent testing could experience side effects in the future. There are also questions surrounding how the majority of people will respond to gene therapy, since gene therapy includes inserting foreign material into the body, which could trigger an immune response. As it is still undergoing phases of testing, there is a lot that is still unknown, including the long-term effects of gene therapy and whether they are dangerous or beneficial (Cleveland Clinic, 2023).
Despite the uncertainties, the implications of gene therapy are revolutionary. Gene therapy can treat many genetic or infectious diseases at the source, many of which don’t currently have treatments. Some examples include cancer, macular degeneration, genetic conditions, and human immunodeficiency virus/acquired immunodeficiency syndrome. Many variations of gene therapy are still in their clinical trial phases; however, there are a few Food and Drug Administration (FDA) approved gene therapy products, including Luxturna and Zolgensma. Luxturna was approved in December 2017 and is a one-time surgical injection to improve the vision of people with vision loss due to inherited eye diseases. Zolgensma, approved in May 2019, is an infusion that treats spinal muscular atrophy in children under two years old (Cleveland Clinic, 2023).
Conclusion
Gene therapy represents a groundbreaking advancement in modern medicine. It offers the potential to treat and cure diseases at their genetic roots. By modifying malfunctioning genes, this technique provides hope for individuals suffering from conditions that currently have limited or no treatment options. While gene therapy is still undergoing testing, scientists are confident in its success. Gene therapy could revolutionize health care by providing life-saving treatments to patients with otherwise incurable diseases. The future of medicine is being reshaped, bringing us closer to a world where previously incurable conditions can be effectively managed or eradicated.
References
Barbieri, E. (Ed.). (2024, November 1). What is AAV for gene therapy?. Target ALS. https://www.targetals.org/2022/11/08/what-is-aav-for-gene-therapy/#:~:text=AAV%20gene%20therapy%20refers%20to,in%20humans%20on%20their%20own
Center for Biologics Evaluation and Research. (2018, July 25). What is gene therapy?. U.S. Food and Drug Administration. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/what-gene-therapy
File:Viral mediated delivery of genes to neurons 1.jpg - Wikimedia Commons. (2022). Wikimedia.org. https://commons.wikimedia.org/wiki/File:Viral_mediated_delivery_of_genes_to_neurons_1.jpg
Professional, C. C. medical. (2023, December 4). What is gene therapy?. Cleveland Clinic. https://my.clevelandclinic.org/health/treatments/17984-gene-therapy
Sen, S. K. (2025, March 6). Messenger RNA (mRNA). Genome.gov. https://www.genome.gov/genetics-glossary/Messenger-RNA-mRNA
Smith, M. (2025, March 6). CRISPR. Genome.gov. https://www.genome.gov/genetics-glossary/CRISPR
What is CRISPR?. Innovative Genomics Institute (IGI). (2025, February 14). https://innovativegenomics.org/what-is-crispr/