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, 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.

CONTINUE READING ON phys.org