The genes in your body’s cells play an important role in your health — indeed, a defective gene or genes can make you sick.Recognizing this, scientists have been working for decades on ways to modify genes or replace faulty genes with healthy ones to treat, cure or prevent a disease or medical condition. Now this research on gene therapy is finally paying off. Since August 2017, the U.S. Food and Drug Administration has approved three gene therapy products, the first of their kind. Two of them re-program a patient’s own cells to attack a deadly cancer, and the most recent approved product targets a disease caused by mutations in a specific gene. Gene therapy can be performed both inside and outside the body.
Sometimes the whole or part of a gene is defective or missing from birth, or a gene can change or mutate during adult life. Any of these variations can disrupt how proteins are made, which can contribute to health problems or diseases.
In gene therapy, scientists can do one of several things depending on the problem that is present:
In order to insert new genes directly into cells, scientists use a vehicle called a “vector” which is genetically engineered to deliver the gene.
Viruses, for example, have a natural ability to deliver genetic material into cells, and therefore, can be used as vectors. Before, a virus can be used to carry therapeutic genes into human cells. However, it is modified to remove its ability to cause an infectious disease.
Gene therapy can be used to modify cells inside or outside the body. When it’s done inside the body, a doctor will inject the vector carrying the gene directly into the part of the body that has defective cells.
In gene therapy that is used to modify cells outside of the body, blood, bone marrow, or another tissue can be taken from a patient, and specific types of cells can be separated out in the lab. The vector containing the desired gene is introduced into these cells. The cells are left to multiply in the laboratory, and are then injected back into the patient, where they continue to multiply and eventually produce the desired effect.
Cells are the basic building blocks of all living things; the human body is composed of trillions of them. Within our cells there are thousands of genes that provide the information for the production of specific proteins and enzymes that make muscles, bones, and blood, which in turn support most of our body’s functions, such as digestion, making energy, and growing.
Gene therapy offers the ability to permanently correct a disease at its most basic level, the genome and could offer cures for many conditions that are considered incurable at this time. It is a flexible treatment option and gene therapy has the potential to treat a variety of illnesses by:
Gene therapy offers patient alternatives to regular daily maintenance. Patients suffering from life-threatening disease are often forced to endure swallowing pills or daily/weekly injections to treat and/or monitor their disease. Gene therapy could ease the burden of enduring the disease by offering the promise of 1-2 treatment injections in a patient’s lifetime with no need for long-term follow-up.
With the remarkable progress in genetics, we believe gene therapy will prove to play an increasingly prominent and transformative role in medicine. Gene therapy has the potential to treat and maybe cure monogenic diseases. Most monogenic diseases are ones for which a protein is defective or not being expressed. As such, gene therapy has the potential to address many of these congenital disorders. There is also a broad opportunity to apply gene therapy for acquired diseases as diverse as HIV, heart failure, Alzheimer’s and more.