Update on Viral Gene Therapy Clinical Trials for Retinal Diseases

In 2001, the first large animal was successfully treated with a gene therapy that restored its vision. Lancelot, the Briard dog that was treated, suffered from a human childhood blindness called Leber's congenital amaurosis type 2. Sixteen years later, the gene therapy was approved by the U.S. Food and Drug Administration. The success of this gene therapy in dogs led to a fast expansion of the ocular gene therapy field. By now every class of inherited retinal dystrophy has been treated in at least one animal model and many clinical trials have been initiated in humans. In this study, we review the status of viral gene therapies for the retina, with a focus on ongoing human clinical trials. It is likely that in the next decade we will see several new viral gene therapies approved.

Automated summary

Gene therapy successfully restored vision in a dog with a childhood blindness disease, leading to FDA approval and further development in ocular gene therapy. Clinical trials have been initiated in humans, with the potential for approval of new viral gene therapies in the next decade.