Gene Therapy Effective in Dogs With Cone-Rod Dystrophy

As reported October 29, 2013 in the journal Molecular Therapy, a French research team led by Fabienne Rolling, Ph.D., of INSERM has used gene therapy to restore vision in dogs affected by cone-rod dystrophy. CRD is caused by mutations in the gene RPGRIP1. In this study, retinal cone cell (central vision) function was significantly rescued in the the dogs, while rod cell (peripheral vision) function was preserved. The effect on vision persisted for the 2-year duration of the study. This is the first time RPGRIP1 gene therapy has been used successfully in a large-animal model of cone-rod dystrophy. Dr. Rolling and her team are now adapting the RPGRIP1 for human study.