There are some diseases that come from your mother and father, right? Or there are some diseases that are very difficult to cure completely even with medication or surgery. Imagine, what if there was a way to change the genes in our own body and cure a disease at its root? Gene therapy is one of the great hopes for the future. Although this is still very new, it is considered to be something that can make a big difference in medical science.
What is gene therapy simply?
Simply put, gene therapy is a medical technique that uses our own genetic material to prevent and treat diseases. Traditionally, we would use things like drugs, pills, syrups , injections, or surgery. But with this technique, doctors can affect your own genetic makeup and correct the underlying cause of a disease.
Think of our body as a big building. The blueprint used to build that building is our genes . Sometimes there can be a small mistake or flaw in this blueprint. That mistake is what causes some diseases. So gene therapy is about correcting that wrong blueprint. That means:
- Reducing disease-causing proteins: When certain genes malfunction, they produce proteins that are harmful to the body. This method can reduce their production.
- Increasing the production of good proteins: Sometimes the body doesn't produce enough of the proteins it needs. In that case, this method can instruct genes to make those proteins properly.
- Producing new or modified proteins: This method can also create a new type of protein within the body that can fight a disease.
How does this work in our body?
To understand this, let's first learn a little about genes. Every cell in our body has a nucleus. Inside that nucleus are things called chromosomes . These chromosomes are made up of DNA . This DNA contains the information that determines everything from your hair color, eye color, and height.
These specific parts of DNA are what we call genes . It's like a recipe in a big cookbook (DNA). These genes give instructions on how to make the proteins that are essential for our bodies to function. That recipe tells us how to make the cake.
Sometimes as we age, toxins in the environmentWhen exposed to chemicals, or inherited from parents, small changes (gene variants/mutations) occur in these genes. It's like a letter or word in that recipe is wrong. Then the protein (cake) that is made is also wrong. That's the root of many diseases.
Gene therapy does not just try to treat the symptoms of a disease. It tries to correct the genetic error that caused the disease in the first place.
What diseases is gene therapy currently used for?
In fact, many gene therapy treatments are still in the clinical trials stage. This means that they are being tested to see if they are safe and effective before being given to people. The main areas of research currently underway for this method are:
However, there are now several gene therapy methods approved by the US Food and Drug Administration (FDA), meaning this is no longer just a dream. For example:
- Luxturna®: This is a one-time treatment for people who lose their vision due to certain inherited eye diseases .
- Zolgensma®: This is approved to treat Spinal Muscular Atrophy, a severe disease that affects children under the age of 2.
Are there different types of this treatment?
Yes, there are three main ways in which gene therapy is performed. Let's see what they are.
| Treatment method | What do you simply do? |
|---|---|
| Gene Addition | A healthy copy of the same gene is inserted into a cell that has a malfunctioning or damaged gene. It's like bringing a brand new, better machine to replace a broken one. |
| Gene Silencing | What this does is stop the function of a faulty gene that causes a disease. It's like silencing someone who gives bad advice by shutting them up. |
| Gene Editing | This is the most advanced method. What is done here is to 'cut out' or 'correct' the incorrect part of the DNA and rearrange it. It's like erasing a wrong word in a book and writing the correct word in its place. Technologies like CRISPR/cas9 are used for this. |
How are genes introduced into the body?
These healthy genes don't go directly into our cells. They need a vehicle. That vehicle is called a 'vector' . Imagine you are sending a parcel. A courier (the vector) takes that parcel (the gene) exactly where it needs to go (the cell).
Most of the time, viruses are used as these 'vectors'. Now you might be thinking, "Wouldn't it make you sick if you put a virus in your body?" What scientists do here is remove all the disease-causing parts of a virus, leaving only the parts that can enter cells. Then, they insert the healthy gene into the virus and introduce it into the body.
There are two main ways to do this:
1. In vivo (within the body): Here, the 'vector' containing the genes is injected directly into the body as a vaccine.
2. Ex vivo (outside the body): This involves taking some cells from your body (e.g. blood cells), inserting genes into those cells in the lab, and then re-introducing those modified cells back into your body.
What are the advantages and disadvantages of gene therapy?
Like everything, this method has its pros and cons.
| Benefits | Disadvantages and Risks |
|---|---|
| A new hope:Diseases that were previously considered incurable are now receiving a new treatment option. | No complete guarantee: This is still new, so it is not possible to say that the treatment will be 100% successful. There may be unexpected side effects. |
| Treating the root cause of the disease: Instead of suppressing the symptoms, you can correct the cause of the disease. | A complex process: When genetic material is introduced from outside, the body's immune system can react against it. |
| Early treatment: If treatment is given before the disease causes damage to the body, that damage can be prevented. | Lack of knowledge of long-term effects: Because this is new, it is not yet known exactly what effects will occur after many years. |
Should I talk to my doctor about this?
If you or someone in your family has a hereditary disease, cancer, or a condition that is difficult to treat, such as those mentioned here, it is very important to talk to your doctor about it.
Gene therapy is still not a common treatment in Sri Lanka. But it is important to be aware of these developments happening around the world. Perhaps in the future, these may become common treatments.
The most important thing is, don't make your own treatment decisions based on what you read or hear online. Always seek the advice of your doctor. He or she can explain to you what treatments are best for your condition, the safest, and the best available.
This technology will take some time to develop, but it shows that medical science is constantly striving to find a solution to a disease, no matter how serious it is. That is a great hope for all of us.
Take-Home Message
- Gene therapy is a new medical method that treats diseases by changing our own genes.
- This aims to treat the root cause of the disease rather than the symptoms.
- This method holds great promise for treating serious conditions such as cancer and hereditary diseases.
- This is still largely in the research stage, and has both benefits and risks.
- If you are interested in such modern treatments, it is very important to discuss them with your doctor.
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