New Gene Therapy Corrects

New Gene Therapy Corrects a Form of Inherited Macular Degeneration in Canine Model

Philadelphia, PA (Scicasts) — Researchers from the University of Pennsylvania have developed a gene therapy that successfully treats a form of macular degeneration in a canine model.

The work sets the stage for translating the findings into a human therapy for an inherited disease that results in a progressive loss of central vision and which is currently untreatable.

The study, published today in Proceedings of the National Academy of Sciences, was led by Karina E. Guziewicz, a research assistant professor in Penn’s School of Veterinary Medicine, and Artur V. Cideciyan, a research professor of ophthalmology in Penn’s Perelman School of Medicine. The research is part of a long-standing partnership between Penn Vet and Penn Medicine scientists to push forward gene and other novel therapies for blinding disorders.

“In the eye, you have these two integral retinal cell layers that puzzle into one another and, like a zipper, they interweave your vision cells and the support cells,” Cideciyan says. “What this disease is doing is basically unzipping those layers, and what we’ve done is rezip them, bringing them together tightly.”

“With this research,” Guziewicz says, “we have demonstrated that there is a therapy that is working in a large animal model. Following safety studies, a human clinical trial could be less than two years away.”

Guziewicz and Cideciyan’s colleagues who collaborated on the work included vision scientists from Penn Vet’s Division of Experimental Retinal Therapies, Gustavo D. Aguirre, professor of medical genetics and ophthalmology, and William Beltran, professor of ophthalmology; and from Penn Medicine Samuel G. Jacobson, professor of ophthalmology.

Best disease, or vitelliform macular degeneration, is an inherited blinding disorder caused by mutations in the BEST1 gene. It often manifests in children and young adults, gradually robbing them of their central vision…..

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Source: Scicasts