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Gene therapy includes modifying the genes within your body's cells in order to treat or prevent disease.
Your DNA is included in genes, which is the code that regulates much of your body's appearance and function, from growing you taller to regulating your body systems. Disease can be caused by genes that do not function properly.
Gene therapy attempts to cure sickness or increase your body's ability to fight disease by replacing a broken gene or adding a new gene. Gene therapy has the potential to treat a broad variety of ailments, including cancer, cystic fibrosis, heart disease, diabetes, hemophilia, and AIDS.
Gene therapy is still being researched as to how and when it should be used. Gene therapy is currently only offered in the United States as part of a clinical trial.
WHY IS IT DONE?
Gene therapy is used to fix faulty genes in order to cure an illness or help your body fight disease more effectively.
Researchers are looking into a variety of approaches, including:
- Replacing mutated genes: Some cells get ill because particular genes function wrongly or no longer function at all. Replacing the faulty genes may aid in the treatment of some disorders. A gene called p53, for example, generally blocks tumor growth. Several forms of cancer have been linked to p53 gene abnormalities. If scientists were able to replace the faulty p53 gene, cancer cells might perish.
- Fixing mutated genes: Mutated genes that cause disease may be switched off so that they no longer promote disease, or good genes that help prevent disease could be activated to limit disease.
- Increasing the awareness of diseased cells to the immune system: Your immune system may not target sick cells in some situations because it may not recognize them as intruders. Doctors could utilize gene therapy to educate your immune system to spot dangerous cells.
WHAT ARE THE RISKS OF GENE THERAPY?
There are certain hazards associated with gene therapy. A gene cannot be easily put into your cells. Rather, it must usually be supplied via a carrier known as a vector.
Viruses are the most commonly used gene therapy vectors because they can recognize specific cells and deliver genetic material into the cells' genes. Researchers delete the virus's original illness-causing genes and replace them with the ones required to end disease.
The following hazards are associated with this technique:
- An unwanted immune system reaction: The newly introduced viruses may be recognized as intruders by your body's immune system and attacked. This can result in inflammation and, in extreme circumstances, organ failure.
- Targeting the wrong cells: Because viruses can infect more than one type of cell, it's feasible that the modified viruses will infect other cells as well as the targeted cells with mutant genes. If this occurs, healthy cells may be destroyed, perhaps leading to additional illnesses or diseases such as cancer.
- Infection caused by the virus: It's likely that once viruses are introduced into the body, they'll regain their potential to cause disease.
- Possibility of producing a tumor: If the new genes are placed in the wrong place in your DNA, they have the potential to cause tumor growth.
The Food and Drug Administration and the National Institutes of Health are actively monitoring gene therapy clinical trials in the United States to ensure that patient safety is a key focus during research.
WHAT TO EXPECT
At the moment, the only method to obtain gene therapy is to take part in a clinical trial. Clinical trials are research studies that assist doctors in determining whether a gene therapy strategy is safe for patients. They also assist clinicians in comprehending the impacts of gene therapy on the body.
Your precise procedure will be determined by the type of gene therapy employed and the ailment you have.
In one type of gene therapy, for example:
- You may be given blood or have bone marrow extracted from your hipbone using a big needle.
- The cells from the blood or bone marrow are then exposed in a lab to a virus or another form of vector with the desired genetic material.
- Once the vector has entered the cells in the lab, the cells are injected back into your body by a vein or tissue, where the vector and the changed genes are taken up by your cells.
Viruses are not the only vectors that may be used to deliver changed genes into the cells of your body. Clinical trials are also being conducted on the following vectors:
Stem cells: Stem cells are the cells that give rise to all other cells in your body. Stem cells can be trained in a lab to become disease-fighting cells for gene therapy.
Liposomes: These fatty particles have the potential to transport new, therapeutic genes to target cells and integrate the genes into the DNA of your cells.
WHAT ARE THE RESULTS OF GENE THERAPY?
The possibilities of gene therapy are very promising. Human clinical trials of gene therapy have shown some success in curing some disorders, including:
- Serious combined immune deficiency
- Hemophilia
- Retinitis pigmentosa, which causes blindness.
- Leukemia
However, several substantial challenges prevent gene therapy from becoming a reliable type of treatment, including:
- Finding a dependable method of introducing genetic material into cells
- Targeting the Right Cells
- lowering the risk of side effects
Gene therapy remains a vital and active area of research aiming at creating novel, effective therapies for a wide range of ailments.
WHEN SHOULD I DISCUSS GENE THERAPY WITH MY DOCTOR?
Researchers are looking at gene therapy as a treatment for cancer, eye problems, hereditary conditions, and HIV/AIDS. Speak with your doctor if you want to take part in a clinical trial using gene therapy. Your doctor can advise you on whether gene therapy is a viable treatment option for your illness.