Researchers see eye to eye on gene therapy

Ithaca, N.Y.-A new treatment that applies gene therapy to dogs blinded by an inheritedretinal degenerative disease has proven successful, according to CornellUniversity researchers.

The treatment restored vision to the dogs by applying genes from healthydogs to the blind, the Cornell College of Veterinary Medicine reports. Genetreatment may someday lead to such therapy for humans.

"We have shown that gene therapy can restore vision in dogs withone of the most clinically severe retinal degenerations," Dr. GregoryM. Acland, a research veterinarian at Cornell's James A. Baker Institutefor Animal Research, says. Ultimately, he says, the therapy "couldcorrect defects in humans with RPE65 mutations."

The RPE cell layer (the gene associated with the retinal pigment epithelium)in humans and dogs supports the retina by sustaining it and removing wastewhile transferring vitamin A to the photoreceptors. Puppies with defectiveRPE65 genes generate a mutant type of RPE65 protein, which leads to visionloss and eventual blindness later in life. The condition has only been foundin the Briard dog breed.

The advancement was reported in a recent issue of the Nature Geneticsjournal.