In December, the FDA approved the first gene therapy to treat an inherited genetic disease. This new approval will be used to combat a type of retinal dystrophy, a rare vision disorder that involves mutations to genes critical to retinal function. Retinal cells are responsible for converting light into electrical signals in the brain, which then get translated into the images we see. This new gene therapy is called voretigene neparvovec-rzyl or Luxturna; it was developed by Spark therapeutics and it works by delivering normal copies of the aberrant gene into retinal cells. These genes are delivered using a modified adenovirus, similar to those that cause common respiratory infections. NPR reported that people who were treated in the trail for the gene therapy were able to read, ride bicycles and see faces; things they were not able to do prior to the procedure. However, the results of gene therapy have not always been promising, the technique suffered a blow to its public image when a boy died in 1999, and shortly after the university testing gene editing on him for his liver closed its program. Despite our better understanding of genetics and improved technology since then, the long term effects of gene editing are still being debated.
I think this is incredible and has an amazing potential to be used for a wide variety of disorders. I am so glad to be alive during a time where gene therapy can begin to thrive, I think its by far the best way to combat so many different types of disorders because it literally "rewrites" the mistakes in genetic code. As of now the gene therapies available for individuals are far too costly to be a realistic option for many but hopefully as time goes on technology will further increase and allow this to become more of a possibility for everyone that needs it. Hopefully with the decreased price more and more trials could also be done in order to learn more about the long term effects associated with the procedures.