Promising and delivering gene therapies for vision loss

- Retinal gene therapy can restore visual function.
- Current clinical trials have shown safety and efficacy of AAV-based gene therapies.
- Preclinical studies indicate several candidate genes for future clinical trials.
- Disadvantages of AAV vectors include a restricted genome size packaging capacity.
- Retinal gene therapy has a promising future but several aspects require improvement.

The maturity in our understanding of the genetics and the pathogenesis of disease in degenerative retinal disorders has intersected in past years with a novel treatment paradigm in which a genetic intervention may lead to sustained therapeutic benefit, and in some cases even restoration of vision. Here, we review this prospect of retinal gene therapy, discuss the enabling technologies that have led to first-in-human demonstrations of efficacy and safety, and the road that led to this exciting point in time.