If the animal model can be cured by gene therapy, than a human clinical trial is in order. There are now numerous animal models of human retinal degeneration, all being pursued by different laboratories. If any model shows success from gene therapy, a clinical trial on human subjects will undoubtedly follow quickly. There are many animal models available but not enough labs are funded to move this ahead quicker. Clinical trials of gene therapy in human subjects face enormous scrutiny because of the Gelsinger tragedy at the University of Pennsylvania .
Current trials require much paper work and require an independent expert to check all data.
Nevertheless, trials are coming. In our laboratory, we are attempting to use a retroviral vector, lentivirus, to introduce genes into the retina. We take advantage of two major developments that greatly facilitate such experiments. We are able to see single retinal cells fluorescing within days after the virus is introduced into the eye of animals, telling us that the new gene is working. These new methods greatly facilitate testing gene therapy strategies.