What is gene therapy ?

Of all the possibilities of treatment for LCA, right now gene replacement therapy is at the top of the list. Researchers and scientists are focusing on the RPE65 gene. What gene therapy does is actually replace the defective gene that is causing the blindness. Essentially a virus cell containing the correct gene is placed into the retina via a very tiny needle. This is a way to deliver the gene that the individual is missing, and to the cells that need the gene. This sets off an array of processes, triggering a biochemical change in the retina, and allowing some vision to be restored. Corey Haas, the youngest in the world to have this procedure, had a very successful outcome. He had one eye treated, with great and promising results! You can see some video here of Corey, the Haas family, as well as a glimpse into the gene therapy treatment.