Gene Therapy: A Promising Therapeutic Technology

 By: Omkar Bakshi, National Youth Tech Journal


Omkar Bakshi is a junior at Cupertino High School. He is a tech enthusiast, interested in pursuing study in the fields of business and biomedical engineering. Bakshi writes for the National Youth Tech Journal which is a Generation Z blog that writes about the latest and most popular technologies in the tech space. Like them on Facebook and follow their Instagram page to hear more of Generation Z’s voice.


Medicinal technology is a trending topic in today’s society and gene therapy is in the spotlight. Gene therapy is an emerging therapeutic technology in which new healthy genes are inserted into the patient’s mutated genes to replace, attack, or deactivate – and hopefully cure – the root problem of the genetic disorder.

Gene therapy could potentially replace drugs and surgery as the preferred form of treatment for patients with chronic autoimmune disorders and life-threatening diseases. Currently, most gene therapies are under extensive clinical study to determine their safety and effectiveness.

There are countless inherited disorders, most of which are incurable. Still, many of them are manageable with drugs. Many of these diseases are chronic, life-threatening, or both. Some diseases are very common, while others are very rare.

Wilson’s disease is one such example; it is a very rare, chronic, life-threatening genetic disease that originates from a missing copper-regulating gene (ATP7B) in the body. If the copper-regulating gene is not present, there is copper accumulation in the body. Excessive copper is toxic to the body, and it could cause critical damage internal vital organs such as the liver and brain. There is no cure for Wilson’s disease yet. Fortunately, medical researchers developed a few drugs to help treat patients with Wilson’s disease. Most of the time, drugs prove to be fairly effective in fighting a disease, but in some cases, drugs just fail to treat the patient. Some diseases don’t even have any form of drug treatment. And in those cases, transplant surgery is the unideal but final option.

Patients with critical conditions have no other choice but to go through invasive surgery such as transplantation. Having a transplant has several negative effects. Generally, post-transplant, patients will face a degree of autoimmune rejection because the newly transplanted organ is a stranger to the immune system. The rejection process itself is unpleasant. If the rejection is very severe, the patient will need another transplant done or face death. Moreover, transplants never guarantee results; the same genetic disease could recur in the body system. Clearly, there are more downsides than upsides to invasive surgery.

Unlike drugs or transplantation, gene therapy offers a fix to the root source of the problem in genetic diseases; as new genes directly attack or replace the mutated genes. Scientists are examining a few approaches to gene therapy: introducing a new gene into the body to combat the disease, deactivating a mutated gene, and replacing a mutated gene with a healthy one. It is a non-invasive procedure as well. People with very rare genetic diseases, such as Wilson’s disease, could be effectively cured with gene therapy.


“Gene Therapy.”

Now that there are substantial advancements in the fields of technology, science, and medicine, progress in gene therapy has been rapidly accelerating. Like any other drug, gene therapy must go through extensive approval testing to determine its safety and effectiveness on patients. Like transplantation, autoimmune rejection is quite possible in gene therapy too. However, gene therapy is a medicinal technology which can be altered as needed. Clinical labs in leading medical institutes around the world are in the final stages of experimentation with gene therapy. Some patients report regaining their sight and hearing after undergoing experimental gene therapy. Slowly, gene therapies are being approved by the Food and Drug Administration (FDA). Hopefully, in the coming years, gene therapy will be rolled out to doctors throughout the world.