Our genes have a significant impact on our health. There exists a "normal" DNA sequence for each and every one of our unique genes. However, variations in this DNA sequence do occur at times, and such changes are referred to as gene variations. Sometimes these are handed down from generation to generation. On the other hand, environmental factors, such as prolonged sun exposure, are sometimes to blame.
Gene therapy comes as a viable option in this regard. By using gene therapy, we can now alter our genotype with the aim of restoring any aberrant DNA to its original state. This may involve exchanging the defective gene for a functional one. As an alternative, the defective gene can be rendered dormant. It may also imply the introduction of a novel gene into the organism as a therapeutic strategy.
In 1990, scientists performed the earliest trial of gene therapy. Even now, after more than three decades, researchers are only beginning to scratch the surface of its promise in treating diseases like cancer and genetic disorders. A genetic disorder is an abnormality of the human body's genetic material.
In this article, we will talk about the benefits of gene therapy that patients can enjoy.
Various genetic abnormalities and other medical issues can be treated with gene therapy. Biologic therapies include gene therapy. The term "biologics" describes these substances since they are obtained from living organisms rather than chemical processes.
The purpose of gene therapy is to modify the function of pre-existing genes. This can happen in three ways:
Switching off the genes that promote or encourage diseases.
Overriding the harmful effects of a mutated gene by inserting a healthy copy of the gene into a cell that already has the mutated gene.
Transmits "instructions" (a new DNA sequence) to an unhealthy cell, which ultimately results in the cell's death.
Let's discuss some real-world applications of gene therapy to better understand the method.
One example of gene therapy is Luxturna (voretigene neparvovec), which is used to treat a kind of inherited blindness. The RPE65 gene in Luxturna is unaltered, which is significant because mutations in this gene can cause blindness. When injected into the eye, Luxturna's healthy RPE65 gene copy takes the place of the faulty one, restoring eyesight. This is an illustration of enhanced genetics.
Acute lymphoblastic leukemia and some forms of non-Hodgkin's lymphoma are treated with the gene therapy Kymriah (tisagenlecleucel). To produce the desired protein, doctors genetically modify a patient's own T cells, a type of white blood cell. The chimeric antigen receptor (CAR) is a protein that can help eliminate cancer by binding to and killing cancer cells. This is one instance of eliminating cancerous cells.
You've realized by now that gene therapy is both challenging and fascinating. People with cancer, uncommon genetic diseases, and other illnesses may greatly benefit from it. Gene therapy has both positive and negative outcomes, just like any other medical treatment. The potential outcomes of every medical intervention should be carefully considered. Let’s have a look at the benefits of gene therapy -
One in every 33 babies born in the United States each year, or around 3%, suffers from some form of birth defect, such as those affecting the brain or spine, the eyes, the heart, the mouth or face, the stomach or intestine, the muscles or bones, or the chromosomes. Trisomy 13 (Patau syndrome), trisomy 18 (Edwards syndrome), and trisomy 21 (Down syndrome) impact a total of 513, 1,187, and 5,568 babies, respectively.
There are around 4,000 annual infant deaths due to these birth abnormalities. Families may exhaust every available healthcare option in an attempt to alleviate these issues, but their efforts will ultimately be in vain because there is currently no treatment for them. However, gene therapy has the potential to treat some of these conditions at birth, allowing children to reach adulthood without risk.
Neuromuscular disorders, cancer, and even blindness can all be effectively treated with gene therapy. Some treatments have successfully eased the illness, though this is by no means guaranteed. Some infants born with severe vision loss due to retinal illnesses that formerly led to absolute blindness can now have their vision restored by gene therapy. Cancer patients may benefit from gene therapy by having their immune cells programmed in a manner similar to chimeric antigen receptor (CAR) T cell therapy.
Thanks to developments in gene editing as a form of gene therapy, there is great hope for treating sickle cell disease and beta-thalassemia, which are both single-gene disorders. Spinal muscular atrophy (SMA) is a neurological disease that causes weakness and ultimately paralysis or death in the muscles that govern and connect the spinal cord to the organs and muscles.
Replacement of faulty genes with functional ones in disorders like cystic fibrosis reduces the likelihood of remission. In most cases, a single session of this treatment is all it takes to permanently alleviate the patient's symptoms. Additionally, gene therapy benefits more than just the individual who receives treatment. In addition, it encompasses an entire generation. If a breast cancer risk gene is surgically deleted, the carrier will pass on healthy genes to her offspring instead of the mutated ones.
Major technological advances have made gene therapies possible, and as technology improves, more therapies will likely get FDA approval. Gene therapies may be expensive now, but as technology improves and new treatment alternatives become available, those costs are expected to decrease.
Gene therapy based on interleukin-12 (IL-12) is a promising new method for treating cancer in animals. Therapeutic gene delivery intramuscularly or intratumorally targeting nodules of disseminated disease or easily accessible tumors has been shown to be beneficial in large animal studies. Multiple canine, equine, and feline tumors have responded favorably to IL-12-based gene therapy in preclinical studies, which has been upgraded and supported by the findings of those studies.
An IFN-y's encoded induction, protein's systematic release, and local transgene response can be achieved by an IL-12-based gene therapy approach that is both effective and safe. This makes it a promising therapy for large animals with naturally occurring tumors.
When conventional treatments have failed, gene therapy may be the last hope for a patient.
The current crop of gene treatments is dosed in a single sitting. When compared to therapies that necessitate a number of doses, this is preferable.
It is possible that gene therapy could permanently eliminate a patient's symptoms. Many people's lives can be improved through gene therapy.
If you take out a bad gene from one parent, it won't be passed on to their offspring.
Huge technological advancements over the past few decades have allowed for the development of gene therapy. Since science and engineering are constantly developing, we may anticipate that gene therapy will also progress. Further FDA approval of gene treatments is also anticipated.
It's a difficult thing to say at this point. Gene therapy research and development take a long time and require close supervision. Teams of researchers evaluate the efficacy and safety of gene therapy at every stage of its development, from basic conception to research studies (clinical trials). All gene therapy items sold in the United States must comply with FDA guidelines. Gene therapy development and human testing need the approval of the appropriate authorities. The NIH also provides researchers with standards to follow when conducting gene therapy studies.
All of this oversight of gene therapy increases the likelihood that any dangers will be discovered and eliminated before the treatment is made available to the general public. However, it's not a completely safe method. Toxic consequences and unwanted side effects are always a possibility when taking medication. Due to its familiarity, gene therapy may have unanticipated hazards. And the time it takes for negative consequences to show up is unknown. People participating in clinical trials need to be closely monitored for a very long time because of the possibility of delayed side effects.
Researchers have talked at length about sharing data from gene therapy studies. When information about gene treatments' successes and failures is shared, everyone benefits. It's a step in the right direction toward creating additional gene therapy items that could potentially save the lives of countless individuals.
It's natural to wonder if you should try gene therapy if your doctor has approved it for your particular sickness or disease. Gene therapy is typically not considered initially. Most people who turn to gene therapies have already exhausted conventional medical options without success. If this describes you, consult your doctor about the potential risks and benefits of gene therapy.
Gene therapy is a medical procedure that allows for the alteration of genetic material. Rare genetic diseases, cancer, and other illnesses may greatly benefit from this complex but promising treatment. However, there are both benefits and drawbacks to employing gene therapy. If you're considering gene therapy to treat your medical problem, it's crucial that you understand the potential side effects you could experience. Gene therapy may be a possibility for you, but it's important to discuss the potential advantages and disadvantages with your doctor first.