How Gene Therapy Can Treat Common Diseases
Genes are the blueprint for how your body functions, and if there is an error in your genome code, it can lead to a variety of issues. It can be as manageable as being slightly immuno-compromised to more serious issues such as Down Syndrome. In some cases, the issues in the genome code can be present from birth, while in other cases, your DNA can mutate as you mature into an adult. Issues in your genes can be a scary idea, as they play a critical role in human health and wellness. However, medical science has been advancing rapidly over the last decade. It is reaching the point where it can alter the genes inside or outside your cells to treat infections and diseases.
Is It Effective?
Instead of treating symptoms and diseases with traditional drugs and surgery, this treatment can eliminate the disease. The treatment can target faulty genes, but it can also battle cancer and viral infections. Scientists have gone so far as to slow down the symptoms of life-threatening illnesses using this method. Gene therapy, in simple terms, aims to cure a disease or improve your body’s ability to fight infection by replacing a damaged gene or inserting a new gene. Cancer, heart disease, hemophilia, diabetes, and AIDS are among the disorders this therapy has shown helpful.
How Does It Work?
Genes cannot be inserted directly into your cells. Instead, they need to be delivered via a carrier, known as a vector. Often, safe viruses are used as a vector to deliver the genetic material into your cells. This strategy has both advantages and disadvantages. Gene therapy has been touted as a cure for various uncommon diseases and a source of hope for patients with complex genetic problems. This information will help you determine whether gene therapy is the best option for you.
There are three primary methods through which gene therapy can treat diseases. However, it bears mentioning that this form of treatment is only available as a clinical trial in the United States so far. You would need to partake in these trials to receive genetic therapy treatment.
- Replacing Mutated Genes
If you have a gene that has mutated or is defective, it can cause your cells to work incorrectly or no longer work at all. For example, the p53 gene is noted to prevent tumor growth, which helps your body fight off cancer. However, a gene defect can leave you more susceptible to tumors. The p53 gene would be replaced with an effective version through gene therapy, triggering the cancer cells to die.
- Fixing Mutated Genes
The type of gene therapy you need depends on the disease you’re dealing with. For instance, you might have a mutated gene that makes you more likely to contract certain types of cancer. In this case, the best course of action would be to turn that gene off so that your body eliminates the weakness. On the other hand, you can also turn on defective genes to improve the body’s capabilities and overall health. According to scientists, you have about 20,000 to 25,000 genes, and they control many functions in your body. They are also responsible for things like the color of your eyes or the texture of your hair. It is safer to activate or deactivate a gene at times than replace it.
- Making Diseased Cells Stand Out to Your Immune System
Your immune system is like a home security system. It needs to recognize the intruder before it can combat it. However, the body makes mistakes in other situations and fails to recognize the virus or bacteria as potentially hazardous. In this situation, gene therapy can teach your body that those cells are harmful and stimulate the body’s natural immune response to combat the condition.
What Are the Risks?
As was mentioned earlier, this technique utilizes viruses to deliver the genetic code. Researchers remove the virus’s original disease-causing genes, replacing them with the genes needed to stop the disease. However, it does still carry some risks.
- Aggressive Immune Response
There is a non-zero chance that the body could see the vector as an invading organism and attack it. That can result in side effects like inflammation and fevers. In extreme cases, the immune response by the body could even lead to organ failure. Always be sure to consult your doctor about the risks associated with your treatment before committing to a clinical trial.
- Affecting Additional Cells
The virus used as a vector may affect more than one cell type. In this case, there is a possibility that the virus would not only alter the target cell with the mutated gene but could also change healthy cells. This method risks your functional cells becoming defective through the treatment, leading to more issues. If the resulting mutation in your cells is serious enough, it could result in you developing cancer.
- Causing An Infection
Scientists take every precaution to remove any disease-causing elements from the virus’ protein code. However, the virus may mutate or reactivate these elements after being introduced to a new environment. As such, you could contract a viral infection through gene therapy.
- Possibility of a Tumor
While this is the least likely outcome, you may develop a tumor if the genetic sequence is injected into the wrong target site due to human error. Depending on where in your DNA the gene was inserted, the tumor maybe
benign or aggressive.
Gene therapy is a viable treatment option for various disorders, and its fundamental method is simple. In contrast, some risks are associated with this treatment. It is closely monitored by the Food and Drug Administration and the National Institutes of Health to ensure that patient safety issues are a top priority during research and treatment. So far, clinical trials of gene therapy in people have shown some success in treating many diseases.
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How Gene Therapy Can Treat Common Diseases