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


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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, described July 3 in Science Advances, provides evidence of the potential value of nanoparticle-delivered  to treat wet age-related macular degeneration—an  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.

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