Assessing the efficacy of gene therapy in Rpe65^-/- mice using photoentrainment of circadian rhythm

Gene therapy (GT) can be described as the in vivo transfer of DNA for therapeutic purposes. In the case of congenital retinal dystrophies (RD), GT can only be considered a successful treatment option if the gene transfer results in the restoration of vision at some level. Thus, a critical component of developing GT treatments for RDs is assessing the amount of functioning vision that is produced. In mouse models of RD, visual function after gene therapy is tested using techniques such as electroretinograms (ERGs) and retinoid analysis. A potentially useful addition to these tests would be a murine behavior-based technique, like those used with the RPE65 dog model, to demonstrate effective GT-induced visual recovery.