Annual Report 2001 - Cornell University

01 Restoring vision through gene therapy

Gustavo D. Aguirre, Veterinary Medicine, Gregory M. Acland, and colleagues in the Baker Institute for Animal Health restored the vision of dogs (the Briard breed), who suffered from an inherited retinal degenerative disease, by treating them with genes from healthy dogs. This is the first successful gene therapy for blindness in a large animal. Mutations in the gene associated with the retinal pigment epithelium, RPE65, cause early vision loss, degeneration of the retinas, and near-total blindness later in life in humans, dogs, and other mammals. The treatment, given to young dogs with congenital stationary night blindness, offers hope for treating a similar childhood disease—Leber congenital amaurosis. Although many safety and efficacy studies still must be done before clinical trials for the gene therapy can begin for human patients, this is an excellent example of how research for animals benefits both animal and human patients.

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© 2002 by the Office of the Vice Provost for Research [OVPR], Cornell University.