Overview of Retinal Gene Therapy: Current Status and Future Challenges.

Abstract

The success of the first Food and Drug Administration (FDA)- and European Medicines Agency (EMA)-approved gene therapy for genetic disease, voretigene neparovovec-rzyl, (Luxturna) has helped pave the way for development of retinal gene therapies to target other genetic and acquired forms of blindness. Gene therapy trials are now taking place in multiple continents and numerous countries, they use several different gene transfer reagents ("vectors"), studies have used several different routes of administration, and different strategies are being tested in interventional studies with promising results. The future has never been brighter for individuals with retinal degeneration. Here and in the literature cited below, we summarize the state-of-the-art of retinal gene therapy and consider some of the questions and challenges that lie ahead.