Sunday, November 8, 2015

New Gene-Editing Therapy Technique Effective in Leukemia Patient

          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.


  1. It is amazing how far we have come in treating very difficult autoimmune diseases. I find this article hopeful as not all cancers should be treated with the traditional chemotherapy. My high school biology teacher described chemotherapy as "trying to shoot a squirrel with a shotgun in a mall", and that makes a lot of sense to me; why treat such a specific cancer with a treatment that is better equipped for a cancer that is tumorous? Very good post.

  2. Wow that is amazing. Although this technique for therapy is at its very early stages it sounds like it has a lot of potential in helping people with many different kind of diseases. And even if it is only found to be useful in some cases, some is better than none, therefore I think this is great progress and I hope it proceeds successfully in the future.

  3. I find personal interest in this topic, as I have had family members pass due to leukemia. This is a tremendous advancement made in cancer treatment. Good article overall, Chris.