What Are the Pros and Cons Associated With Gene Therapy?

Gene therapy is a treatment that revolves around altering the genes inside cells to stop disease, according to Mayo Clinic. The pros include helping people fight disease or even cure their disease, while some cons are unwanted immune reactions, infections, tumors and damaging healthy cells.

Researchers are looking at several ways to use gene therapy, including replacing or fixing mutated genes and making diseased cells easier targets for the immune system, explains Mayo Clinic. Replacing genes that don't work correctly or no longer work at all may help treat certain diseases. Mutated genes could be turned off so they don't promote disease, and healthy genes could be turned on so they inhibit disease. Gene therapy could be used to train the immune system to better recognize cells that are a threat.

Because the new genes are delivered using a carrier that is usually a virus, the immune system might attack them and cause inflammation or organ failure, notes Mayo Clinic. It's possible the viruses may infect additional cells, and if this happens healthy cells could be damaged, causing other diseases, including cancer. Although they're altered before insertion, the viruses might recover their ability to cause disease once introduced into the body. If new genes get inserted in the wrong spot it could lead to tumor formation.