"Now that we've demonstrated this type of delivery can be accomplished, " said William A. Beltran, assistant professor of ophthalmology at Penn's School of Veterinary Medicine and director of the study, "the next step is to target common rod degenerations using canine models that mimic the human diseases. The delivery of a viral vector, coupled with a rod-specific promoter is likely to be the safest and most efficient approach to correct diseases such as X-linked and autosomal dominant retinitis pigmentosa, both of which lead to complete blindness."

A form of childhood blindness called RPE65-Leber's congenital amaurosis, LCA, is the first - and to this date the only - inherited retinal disease for which corrective gene therapy is currently being tested in three on-going clinical trials. This treatment was first developed and perfected in dogs by some of the scientists who participated in the present study before its use in human patients.

Source: University of Pennsylvania