Scientists use nanoparticle-delivered gene therapy to inhibit blinding eye disease in rodents

In experiments in rats and mice, two Johns Hopkins scientists – an engineer and an ophthalmologist – report the successful use of nanoparticles to deliver gene therapy for blinding eye disease. A uniquely engineered large molecule allows researchers to compact large bundles of therapeutic DNA to be delivered into the cells of the eye.

The research provides evidence of the potential value of nanoparticle-delivered gene therapy to treat wet age-related macular degeneration – an eye disease characterized by abnormal blood vessel growth that damages the light-sensitive tissue in the back of the eye – as well as more rare, inherited blinding diseases of the retina.

"Some of the most prevalent inherited retinal degenerations are due to mutations in large genes that simply cannot fit into the most commonly used viral vector," says Peter Campochiaro, M.D., the Eccles Professor of Ophthalmology at the Johns Hopkins University School of Medicine, and a member of the Johns Hopkins Medicine Wilmer Eye Institute.

To overcome such limitations, Campochiaro and Jordan Green, Ph.D., developed a new approach involving a biodegradable polymer that surrounds and compacts long stretches of DNA, creating nanoparticles that can enter the cells. This technology allows the researchers to convert the cells of the eye into minifactories for a therapeutic protein.