Gene therapy
For decades, scientists have been hopeful about the idea of editing our genes to treat difficult or seemingly incurable ailments. We’re now finally starting to see these efforts pay off. Since 2017, the FDA has approved at least two gene therapy treatments meant to correct or replace harmful mutations that are directly causing illness. A related area of research is CAR T-cell therapy, which edits a person’s T-cells in the lab to make them better at fighting off some cancers; the cells are then infused back into the body.
In December, a small trial using gene therapy to repair the misshapen red blood cells of people with sickle cell disease seems to have passed with flying colors. For up to three years after treatment, the cells of these volunteers still appeared to have their corrected shape, and, most importantly, the people no longer experienced the episodes of severe pain and other symptoms common with sickle cell. The results of this and other research are looking so impressive that this may genuinely represent a bona-fide cure for the genetic disorder, which is thought to affect about 100,000 Americans.
Other upcoming applications for gene therapy may include a painful skin disorder known as epidermolysis bullosa (also called “butterfly disease”), hemophilia, and more types of cancers.
