An international clinical trial conducted through the Perelman School of Medicine at the University of Pennsylvania has helped a patient suffering from childhood blindness gain vision after a single injection into the patient’s eye, The Hill reports.
The potentially groundbreaking research was published in a paper in Nature Medicine.
Patients received intraocular injections — shots into the eye — of a small RNA molecule that targets the eye’s cone cells, which are responsible for color vision.
The treatment is aimed at patients who suffer from Leber congenital amaurosis (LCA), a rare genetic eye disorder that affects the retina. Typically, patients with LCA are blind or have a severe visual impairment starting in infancy.
In this study, one of the patients was followed for 15 months after the single injection. The patient’s sight improved after one month, peaked in two months, and continued to hold on to the improvement even at 15 months following the single injection.