WHAT ARE CELLS AND GENES? HOW DO THEY INTERACT?

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.

WHAT IS GENE THERAPY? HOW DOES IT WORK?

The genes in your 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 reprogram a patient’s own cells to attack a deadly cancer, and the most recently approved product targets a disease caused by mutations in a specific gene.

Gene therapy... can be performed both inside and outside the body. This infographic illustrates in simple terms how gene therapy works inside the body.

HOW GENE THERAPY WORKS

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, and this sometimes contributes to health problems or to disease.

In gene therapy, scientists can do one of several things depending on what is required. They can replace a gene that causes a medical problem with one that doesn’t, add genes to help the body to fight or treat disease, or turn off genes that are causing problems.

In order to insert new genes directly into cells, scientists use a a “vector” , that is, a vehicle that has been genetically engineered to deliver the gene.

Viruses, for example, have a natural ability to deliver genetic material into cells, and can therefore be used as vectors. Before a virus is made to carry therapeutic genes into human cells, however, it must be modified in such a way that it can no longer 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.

When done outside the body, blood, bone marrow, or another tissue is removed 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 then injected back into the patient, where they continue to multiply, and eventually produce the desired effect.