A single injection reverses blindness in patient with rare genetic disorder
A Penn Medicine patient with a genetic form of childhood blindness gained vision, which lasted more than a year, after receiving a single injection of an experimental RNA therapy into the eye. The clinical trial was conducted by researchers at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. Results of the case, detailed in a paper published today in Nature Medicine, show that the treatment led to marked changes at the fovea, the most important locus of human central vision.
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