Gene therapy refers to a technique that uses genes to treat, prevent or potentially cure certain diseases. The focus of gene therapy today is typically in diseases that are caused by variants in a single gene. In these conditions, a person’s own gene has a variant that affects the normal biological function of the protein made from this gene. Gene therapy contains a functioning gene that is designed to be added to the patient’s cells in effort to override the effects of a disease-causing variant.
Gene therapy is designed to work by using something called a vector (a kind of small, cellular package containing genetic information) to introduce, or transfer, a new gene into your body’s cells. The concept behind this is to add a working copy of the gene to overcome the effects of the genetic disease. The gene therapy is inserted into the body typically via an I.V. infusion.