A Shot to the Eye Restored a Child’s Vision

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.