What is Gene Therapy?
Gene therapy is an experimental technique that uses genes
to treat or prevent disease. In the future, this technique may allow doctors to
treat a disorder by inserting a gene into a patient’s cells instead of using
drugs or surgery.(3) Although gene therapy is a promising treatment option for a number of diseases (including inherited disorders, some types of cancer, and certain viral infections), the technique remains risky and is still under study to make sure that it will be safe and effective.
Some testing approaches to gene therapy include:
Replacing a mutated gene that causes disease with a healthy copy of the
gene.
Inactivating, or “knocking out,” a mutated gene that is functioning
improperly.
Introducing a new gene into the body to help fight a disease.
Gene therapy uses a vector. A new gene is injected into a vector, which is used to introduce the modified DNA into a human cell. If the treatment is successful, the new gene will make a functional protein(4). One can see how this works by looking at the figure to the right.
The first gene therapy experiment occurred on September 14th, 1990, which means that this technique is quite new compared to other techniques.
Today, scientists are focused on diseases caused by single- gene defects. These diseases include cystic fibrosis, hemophilia, sickle cell anemia, muscular dystrophy, etc. Additionally, scientists are focused on cancer, and how gene therapy has the potential to cure it.