Genes can play an essential role in health — a defective gene or genes can make you sick or be fatal. Acknowledging this, scientists have been working for decades on adjusting or replacing faulty genes with healthy ones to treat, cure or prevent a disease or medical disorder. Nowadays, this type of research is finally paying off. In August 2017, the U.S. Food and Drug Administration (FDA) approved three gene therapy. Two of them re-program a patient’s cells to attack cancer and a recently approved product that targets a disease caused by mutations in a specific gene. Furthermore, gene therapy can be performed inside and outside the body.
Occasionally part or the whole gene may be defective or missing from birth or change or mutate throughout life. These deviations can disrupt how proteins are made, thus contributing to health issues or diseases.
Gene therapy allows scientists to:
- Replace a gene that causes a medical issue with one that doesn’t
- Add genes to assist the body in fighting or treating disease.
- Turn off genes that are causing medical issues.
- Insert new genes directly into cells through vehicles called “vectors” that are genetically engineered to deliver the gene into the body.
Viruses can naturally deliver genetic material into cells and can be used as vectors. Before a virus can carry therapeutic genes into human cells, it is modified to prevent its ability to induce infectious diseases.
Gene therapy can be used to alter cells inside or outside the body. When it’s done inside the body, a doctor will inject the vector carrying the gene straight into the region of the body with defective cells. In contrast, when therapy is outside the body, the cells are taken from the patient and separated modify in the lab. Then, the vector containing the desired gene is presented into these cells. The cells are left to multiply in the laboratory and injected back into the patient, where they continue to multiply and ultimately produce the desired outcome.
Cells and Genes and how do they interact
Cells are the basic building blocks of all living things; the human body is composed of trillions of cells. Within those cells, thousands of genes supply the information for producing specific proteins and enzymes that create muscles, bones, and blood, which maintain our body’s functions, including digestion, creating energy, and growth.
How does gene therapy work?
Gene therapy presents the capability to permanently repair a disease at the most rudimentary level, the genome. Gene therapy offer treatments for numerous conditions thought to be incurable. Gene therapy is a flexible treatment option that has the potential to treat a combination of conditions by:
- Substituting missing or faulty genes that can induce inherited or acquired disorders such as HIV, heart failure, and Alzheimer’s
- Introducing genes that promote required proteins to the body.
- Introducing genes that enhance the body’s resistance to harmful conditions and function at higher levels.
- Introducing genes that promote cell growth and heal damaged tissue.
Gene therapy offers patients alternatives method for the body’s regular daily upkeep. Many patients suffering from chronic conditions are often a slave to swallowing pills and daily or weekly injections to treat and monitor their condition. Gene therapy can relieve the burden of bearing the disease by offering 1-2 treatment injections in a patient’s lifetime without never-ending follow-ups.
With recent progress in genetics, we believe gene therapy will play a prominent and transformative part in medicine. Gene therapy can potentially treat and cure monogenic (single-gene) diseases. Monogenic diseases are single genes in which a protein is defective or not expressed. Furthermore, there is a broad opportunity to apply gene therapy for acquired diseases as diverse as HIV, heart failure, Alzheimer’s, cancer, and more. As such, gene therapy can potentially address these congenital disorders.
If you enjoyed this article, please click here to donate and support our work.