You can think of our body as a big instruction book. We call the small instructions in this instruction book "genes" . These genes determine everything in our body, for example, the color of our eyes, the texture of our hair. So, sometimes a small mistake in one of the instructions in this instruction book, that is, if a letter is changed, it can cause serious diseases. Today we are talking about a very advanced medical method that corrects such incorrect instructions, or inserts a new correct instruction. This is called gene therapy.
What is gene therapy simply?
Simply put, gene therapy is a special method of treating genetic disorders. Our body's genes give instructions for making proteins. If a gene changes or becomes faulty, the protein it produces can be made incorrectly or stopped altogether. This is what causes genetic disorders.
Gene therapy either changes the faulty gene that causes the disease , or gives your cells a new working copy of a gene . This is often a one-time treatment. After this treatment, your body is able to make the proteins it needs correctly, which relieves symptoms.
How does this treatment work?
There are three main ways in which gene therapy works. Let's look at the table below to understand this clearly.
| Treatment method | Simply put... | What are you doing? |
|---|---|---|
| Gene Addition | Inserting a new good gene. | If a necessary protein is not produced in the body due to a defect in a particular gene, a copy of a healthy gene that can make that protein is inserted into the cells. |
| Gene Editing | Correcting the faulty gene. | Scientists use special tools like CRISPR/cas9 to "cut and paste" the faulty gene that causes the disease. Then, that gene starts producing the correct protein. |
| Gene Silencing | Turning off the wrong gene. | Some genes work incorrectly and make proteins that are harmful to the body. This method stops the function of a gene that gives such incorrect instructions. That is, it is silenced. |
How do you insert these modified genes into the body?
Okay, now you have a question: How do you get these modified genes into the body? Scientists package these genes in something like a parcel. This parcel is called a "vector." This vector is often a specially modified virus.
Don't worry! This is not a virus that makes us sick. It has completely removed its ability to make us sick, and instead has been inserted with our healthy genes. Like a delivery vehicle, this carrier's only job is to carry the genes and deliver them to the right cells.
In some treatments, some of your own blood stem cells are taken, modified in a lab, and then put back into your body. In other cases, a carrier containing these genes is given as an injection or as an infusion into a vein. Over time, the cells with these new genes divide and multiply, replacing the diseased cells. So the effects of this treatment should be long-lasting.
What diseases can be cured with gene therapy?
Gene therapy is most often successful for diseases caused by a single gene defect. Here are some of the diseases for which this treatment is currently approved:
- Genetic diseases that affect the brain, such as Cerebral Adrenoleukodystrophy .
- Inherited blood diseases: For example , sickle cell disease, thalassemia, and hemophilia .
- Inherited neuromuscular diseases: Conditions such as Duchenne Muscular Dystrophy and Spinal Muscular Atrophy.
- Inherited eye diseases: Diseases that affect eyesight, such as Leber's Congenital Amaurosis.
- Other genetic diseases such as Metachromatic Leukodystrophy.
In addition, scientists are still researching whether gene therapy can be used to treat diseases such as cancer and HIV .
What are the advantages and disadvantages of gene therapy?
Like any treatment, gene therapy has its benefits as well as some risks and disadvantages. It is important to be aware of these.
| Pros | Cons and Limitations |
|---|---|
| New Hope: Provides new treatment options for many diseases for which other treatment options are lacking. | Difficulty of access: Due to high costs and limitations of technology, not everyone can access this treatment. |
| Convenience: Most gene therapies are once-in-a-lifetime treatments. You don't need to take medication every day. | Side effects: As with all treatments, there is a risk of side effects. For example, the body's immune system may react to the gene carrier (virus). |
| Early treatment: If this treatment can be obtained before the disease causes significant damage to the body, that damage can be prevented. | Preparation process: Some gene therapies require other treatments, such as chemotherapy, before treatment, which can cause unpleasant side effects. |
| Treating the root cause of the disease: Instead of just treating the symptoms, we treat the underlying cause of the disease, which is the genetic defect itself. | Still a new field: Although this has been in the research stage for decades, its use in medicine is still relatively new. There is still much to learn. |
Gene therapy holds great promise for people with genetic diseases. It can treat the root cause of a disease, rather than just the symptoms. Although this is still an emerging field, it has the potential to provide life-changing results for patients who have had few treatment options in the past. If you or someone in your family has a genetic disease, it is best to talk to your doctor about this and learn more.
Take-Home Message
- Gene therapy is an advanced treatment method that corrects the underlying genetic defect that causes some genetic diseases.
- This either corrects a faulty gene or inserts a copy of a healthy gene into the body.
- Although this is often a one-time treatment, it is currently not common everywhere in the world and is expensive.
- Like any treatment, there are benefits, risks, and side effects.
- It is always essential to discuss this with your doctor before making any decisions about such treatments.
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