Gene Therapy – Kimaya

I chose this issue because i thought it was an interesting subject and that it could change the world. My dad’s company has a section that works on it, and he told me about it. I found it fascinating that genetic diseases (and other ones as well) could be treated by genes. Another thing that hooked me was that it can cure cancer which was previously extremely difficult to cure. This issue is relevant to me since we are teenagers right now, and by the time we grow into adults, this could be a vital part in our lives. Another way it could affect me (and other teenagers) is because other around us or us could need gene therapy.

While researching i learnt many things about gene therapy. One of things i learnt about it was what it does. Gene therapy either replaces a mutated gene (one that could cause diseases) with a healthy copy of the gene, “knocks out” (inactivates) a mutated gene (that again, isn’t working as it should be), or introduces a new gene to the body that helps fight the disease. Another thing i learnt is how its done. Usually, it’s done by using a harmless virus to carry genetic cargo into cells. There are two ways that gene therapy is performed; inside or outside the body. When it’s done inside the body, the doctors would inject the virus carrying the gene directly into the part of the body that has the mutated cells. When it’s done out of the body, blood or bone marrow is taken out of the patients’ body and immature (harmless) cells are separated out. Then, a gene is added into those cells and they are injected back into the patient. The last thing i learnt is that. The out of body method has been tested on children with immunodeficiancy (also known as SCID); a syndrome where patients are unable to fight infection, and often die in childhood. More than 30 patients with SCID have been treated using gene therapy, and more than 90% of them have been cured of this syndrome. A 90% success rate is extremely high. However, the 10% of children who were not cured instead developed cancer and died as they were unable to battle it. This is why it isn’t in use yet. My thinking has changed a lot over the course of this because before this i literally had no idea anything like this could ever exist. I believed that cancer was extremely hard to cure, and some genetic diseases were impossible to cure. Now i know that this is not the case, and instead it’s almost as if the genetic disorders are being stopped from their roots. However, we should be very sure that this is 100% successful before using it because it could make or break so someones life.


This relates to my topic because it shows a mutated gene being inactivated or knocked out OR a mutated gene being replaced by a healthy copy.

Leave a Reply

Your email address will not be published. Required fields are marked *