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A Shot to the Eye Restored a Child’s Vision

A single shot of experimental RNA therapy restored vision for more than 15 months.

There’s no magic cure for blindness, but it’s an attractive concept for medical innovators. A recent Interesting Engineering article discussed an experimental treatment that successfully restored vision in a child born with a genetic form of childhood blindness. A single shot of RNA therapy administered directly to the eye restored the child’s eyesight for more than a year.

The treatment was developed for patients pre-diagnosed with Leber congenital amaurosis (LCA), which is an eye disorder affecting the retina. People with the gene mutation for LCA usually experience severely impaired vision beginning in infancy. In a clinical trial, researchers at University of Pennsylvania’s Scheie Eye Institute, participants were injected with an antisense oligonucleotide. This short RNA molecule increases specific protein levels in the photoreceptors to improve retinal function and perception in daylight. 

While the strategy isn’t a panacea for blindness, it’s a major step closer to creating new techniques to improve vision in those genetically predisposed to suffer from eye diseases. 

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