All humans look different with different skin textures, eye colors, noses, and ear sizes. The factor for these variations in each person is their genes, as all features we get are due to our genes. In a way, genes are the “instruction guide” for our body, and these contain a chemical known as DNA. Gene therapy impact factor is huge in taking care of our health. All unique genes have a DNA sequence that is normal” for that type of gene. It is known as a gene variant. Sometimes, they pass from parent to child.
Here is where gene therapy plays its part. With the benefits of gene therapy, we can modify our DNA. Here the objective is to turn any abnormal DNA back to its normal condition. It can mean changing the faulty gene with a healthy copy. Or this would also suggest making defective genes inactive. It would mean a new gene is entering the body for treating the health condition.
The study for the first gene therapy was conducted in 1990. It’s been three decades, and still, we are learning about the benefits of gene therapy in treating medical conditions like cancer and genetic disorders. By a genetic disorder, we mean a health condition due to abnormal DNA.
With time, gene therapy became famous, and more studies have been done to find its benefits. In this blog, we will find how it works and discuss its benefits and risks. To know more about the benefits of gene therapy, read through our blog.
How Does Gene Therapy Work?
There are many benefits of gene therapy treatment methods. It mainly treats genetic disorders and other medical conditions. They are also known as “biologics,” as their source is living organisms.
The main idea behind the gene therapy treatment process is to change the behavior of the present genes in the body. The benefits of gene therapy are possible through three procedures:
Removing cells that cause diseases: The unhealthy cells get “instructions” (a new DNA set). It kills the old, ill cells.
Gene augmentation: This means adding a healthy gene copy to a cell with a faulty gene. It ensures that the healthy gene copy is shadowing the negative effects of the unhealthy gene.
Inhibition of genes: This is shutting down those genes that are the main reasons behind the disease.
Real-Life Examples of Gene Therapy
Let’s consider Luxturna, the gene therapy type for treating genetic vision loss. The Luxturna has a healthy copy of the gene RPE65, resulting in blindness after mutation. After injecting Luxturna in the eye, the healthy copy starts acting instead of the RPE65 mutated gene and restores vision loss. It is one example of gene augmentation.
We will now check the Kymirah. The Kymirah consists of a person taking T cells of their own (a white blood cell type) and changing these in a lab to make them have a certain kind of protein. The protein chimeric antigen receptor (CAR) links to the cancer surface cells and puts an end to them to get rid of cancer in the body. It is an example of killing the cells causing the disease.
Is Gene Therapy A Safe Treatment Method?
Thirty years ago, medical experts tested the first gene therapy with gene therapy impact factors and outcomes. Studies stated that gene therapy poses severe risks to health like toxicity and cancer. Later on, medical experts and researchers studied its mechanisms and developed improved techniques that were less likely to have dangerous immune reactions with more benefits of gene therapy. As gene therapy techniques are new, there can be possible risks. However, various firms are working on it to ensure that the research on gene therapy proceeds forward.
The Food and Drug Administration currently controls all gene therapy products in the US. Federal laws protect people from involving in clinical trials or research studies. Researchers wanting to test an approach in a clinical trial should firstly get FDA permission.
The National Institutes of Health (NIH) also has a vital role in ensuring gene therapy safety. The NIH has laws for institutions after conducting gene therapy clinical trials.
After successful clinical trials, some gene therapies got approval, including medicines to treat genetic disorders such as Leber congenital amaurosis and spinal muscular atrophy.
With such laws, any major health risks of gene therapy are possible to find and prevent before the treatment is ready for the public. However, the process is not fully proven, as toxicity and side effects are possible with any treatment.
Researchers stated the importance of sharing data in studies of gene therapy. It is a new concept, so the risks are also there in addition to the benefits of gene therapy. Also, there is a chance of late side effects for which subjects must be checked for a long time. With data, we learn what works and what doesn’t.
Gene Therapy Pros and Cons
The benefits of gene therapy are life-changing for people with rare genetic diseases, like cancer and much more. However, there are some gene therapy pros and cons like all treatment methods. You should always determine the benefits and risks of gene therapy. Let’s discuss some risks and benefits of gene therapy.
- Has many options: Gene therapy has the power of curing diseases where other treatment forms fail to work.
- Only single treatment: The current gene therapies are like a one-time dose. Gene therapy is more promising than other treatments with multiple doses.
- Positive outcomes for the long term: With gene therapy, the patient can get rid of their symptoms for a lifetime. With gene therapy, many people can improve their quality of life.
- Positive effects in the future: Removing faulty parent genes ensures that they won’t pass it to their children.
- New tech is always around the corner: People started realizing the benefits of gene therapy as tech advanced. As tech is always improving, gene therapy is also getting better. We are also hoping gene therapy to get FDA approval soon.
- Costly: The cost of gene therapy is much higher than other treatment forms, making it out of reach for many people. For instance, gene therapy Zolgensma for treating spinal muscular atrophy is the most expensive treatment in the U.S. The overall treatment course costs around $2.1 million, which is not possible to afford for a normal citizen.
- Still in Testing Phase: The treatment form is new, and still, we are yet to know many benefits and negatives of gene therapy. As of now, no guarantees are there for the treatment. Medical experts are unsure about how they will treat the issue.
- Possibly dangerous: Some concern is still there that if gene therapy reaches cells in the body, it might have harmful effects besides the faulty cell. Also, it might replace the sex cells without detection, and the parents can pass it on to their kids.
- Might result in an infection: Some gene therapies require dead viruses for carrying healthy genes into faulty cells. Thus, possibly these viruses can also infect the person getting the treatment.
Is Gene Therapy Helpful For Me?
Gene therapies are not the first choices. If you’re suffering from any medical condition treatable through FDA-approved gene therapy, you might doubt using it. Most people use it before trying other medical therapies that don’t work. For such a case, you can consult your healthcare provider to learn about the benefits of gene therapy. With knowledge about the benefits of gene therapy and its risks, you can know whether it would be a safe treatment choice or not.
With medical treatment, gene therapy lets us modify our DNA. While there are many gene therapy pros and cons, the benefits of gene therapy are also good for people with rare genetic issues like cancer. With the pros and cons, you should find the possible risks and talk to your doctor to learn about them. University Cancer Centers (UCC) use extensive gene therapy and other treatment options like systemic chemotherapy, radiation therapy, surgery, and endocrine therapy for treating cancer. They provide custom cancer treatment options with the benefits of gene therapy for patients to ensure total recovery. To get an idea about our work, you can look at the outcome data of our patients and soon notice the benefits of gene therapy and other treatment types by booking an online appointment.