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Researchers use nanoparticles to deliver gene therapy for blinding eye disease

July 7, 2020

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 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.

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