Overview

• The gene therapy was developed by the Institute of Genetics and Medicine of Pozzuoli and delivered at the University Luigi Vanvitelli ophthalmology clinic in Campania, Italy.
• Treatment involves subretinal injections under general anesthesia of two viral vectors, each carrying half of the large Usher syndrome type 1B gene to overcome size constraints.
• Within two weeks, the 38-year-old patient showed visual improvement and by one month could navigate dim environments and read television subtitles clearly.
• Preliminary results from seven additional trial participants confirm a favorable safety profile, with no serious adverse events and only limited inflammation resolved with corticosteroids.
• Researchers plan to expand clinical trials and explore applications of the double-vector method for other inherited retinal disorders with oversized gene defects.