Gene Therapy for Blinding Eye Conditions Improves with Engineered Virus
Unlike standard treatments, this new procedure – which takes as little 15-minutes – is surgically non-invasive, and can deliver normal functioning genes to difficult-to-access cells found throughout the entire retina. The study was published in Science Translational Medicine.
During the prior six years, several groups have successfully delivered treatment to individuals with a rare inherited eye disease by injecting a virus with a normal gene directly into the retina of an eye with a faulty gene. Despite the invasive delivery method, the virus with the normal gene was not capable of reaching all the retinal cells that were in need of repair.
According to Professor David Schaffer, sticking a needle through the retina and delivering the engineered virus behind the retina is a risky surgical procedure. However, by building upon 14 years of research, we have created a virus that you simply inject into the liquid inside the eye and the genes are able to reach a remote population of retinal cells. The 15 minute procedure is surgically non-invasive and safe, and you can likely go home the same day.
The engineered virus approach is far more effective than standard therapies in rodent models of two human degenerative eye diseases. It is capable of penetrating photoreceptor cells in the eyes of monkeys, which are similar in structure to those of humans.
Schaffer and colleagues are now collaborating with physicians to determine which patients are most likely to benefit most from this novel gene-delivery method and, following some preclinical development, hope to initiate clinical trials.
Additionally, Schaffer predicts that the virus can be employed not only for gene insertion that restores function to broken genes, but can also disrupt genes or block processes that are actively destroying retina cells, which may be the case in age-related macular degeneration.
He concludes by stating, "When I first got here 14 years ago, I really had the idea or the goal that I wanted to work on problems that would have direct impact on human health, and we are now getting there.”.