By: Devika Rao
What is Gene Therapy?
Used as an alternative to drugs and surgery, gene therapy is a form of medical treatment that fixes and prevents diseases by correcting the underlying problems in the genes themselves. The earliest form of this approach, known as the “gene transfer”, was created in order to introduce a new gene into cells to help fight a disease, and also introduce a non-faulty/clear copy of the gene to replace the disfigured copy that was causing the disease.
When was Gene Therapy discovered?
The journey of gene therapy and its usage has been a long and controversial topic for many years. Even though it was originally discovered in 1953, patients were not successfully treated until 1990. Though everything seemed to be going well during the beginning of the 1990s, a patient in 1999 ended up passing away from a severe immune response during a clinical trial. In the years to come, more problems began to rise as patients began to develop cancer from the viruses (that were used as a vehicle to kickstart the gene therapy) in their bodies.
Over the years there have been many setbacks due to the costs of gene therapy and also delays with FDA approval which has significantly affected gene therapy’s outreach to people in need of it.
How safety with Gene Therapy has improved:
In this day and age, there are over 600 potential therapies that are in the works, which are being made for various conditions. Gene therapy is projected to treat many health issues: different types of cancers, bleeding disorders, and also many different types of rare diseases. In the future, there may be gene therapies created for both common as well as more uncommon and rare conditions.
How ethical is Gene Therapy?
Morality and ethicality have been some of the most important issues that have been brought up, because of gene therapy. Gene therapy makes significant changes to the human body, as it alters the building blocks of the human body. The work that is currently being done revolves around somatic cells (body cells such as the blood cells and bone marrow). This alteration does not affect the germ cells (egg and sperm cells), hence it cannot be passed on to the person’s offspring. However, there is a new approach that could affect the germ cells, known as germline gene therapy, as it can alter the germ cells. This approach is controversial, as the unborn fetuses can’t choose whether or not they want to go through the treatment. Another reason why this is controversial is attributed to how germline gene therapy hasn’t ever been done before and how there are no known long-term side effects from the therapy.
All in all, the field of gene therapy, though it could be controversial at times, has brought many new developments to medicine and has created change to many people around the world.
Sources:
U.S. National Library of Medicine. (n.d.). What is gene therapy?: Medlineplus Genetics. MedlinePlus. Retrieved May 2, 2022, from https://medlineplus.gov/genetics/understanding/therapy/genetherapy/
U.S. National Library of Medicine. (n.d.). What are the ethical issues surrounding gene therapy?: Medlineplus Genetics. MedlinePlus. Retrieved May 2, 2022, from https://medlineplus.gov/genetics/understanding/therapy/ethics/
Dorholt, M., & Director, M. (2021, July 8). We're on the verge of a breakthrough for Gene Therapies. Evernorth. Retrieved May 2, 2022, from https://www.evernorth.com/articles/the-history-and-future-of-gene-therapy
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