The picture above is of Layla a one-year-old girl with Leukemia who was recently put into remission through the use of gene-editing technology. This is only the second trial of gene-editing used as therapy the first trial was used in HIV patients. Layla's was not responding well from conventional treatment methods for Leukemia, so after a request she was given the new gene editing treatment.
This technique requires the healthy T cells from a donor. The T cells are then modified so that they are protected from the anti-cancer drugs that the patient has been taking. The T cells are also "edited" with a DNA-cutting enzyme called TALEN. This deactivates the immune cells from attacking the leukemia patient when injected. The patient then undergoes therapy in which their immune system in destroyed and replaced with the modified donor T cells. There are however many concerns when dealing with ex vivo and in vivo gene therapy. They can cause cut and mutations in other parts of the genome. Also for in vivo therapy the DNA-delievering vector can stay active in the body years after injection which raises concern for future immune reactions. There is definitely more research necessary for this treatment to become more conventional.
While this technique resulted in a cancer free Layla it is still an experimental stage and more research is being put into gene-editing therapy. In my opinion I believe that this can open many doors for treating immune system related diseases. Just the fact that a technique has been used for two diseases that have been found extremely difficult to treat shows where the research is going towards. To see new treatment techniques coming out out seemingly every year is amazing.
