Leber congenital amaurosis (LCA) is one of the most common causes of childhood blindness. The findings of a small clinical study published recently in Nature Medicine highlights the results of a new therapy that improved the vision of children living with LCA. Study participants were treated with an intraocular injection of an oligonucleotide, an RNA molecule.

“It was very dramatic to see one of the patients improve from only being able to differentiate light or dark to reading many letters on an eye chart at two months following the first injection,” noted lead study investigator Artur Cideciyan, Ph.D., a research professor of ophthalmology at the Scheie Eye Institute in the Perelman School of Medicine at the University of Pennsylvania. “So, we performed a thorough interim analysis of all results from all patients.”

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“Injectable RNA Therapy Dramatically Improves Vision in Child Blindness”