Ocular gene therapy: the new era of blindness prevention

Summary In December 2017, the world’s first direct-to-human gene therapy was approved for an inherited retinal disease called Leber Congenital Amaurosis. This disease normally onsets in early childhood and causes severe vision loss and blindness. Excitingly, the new gene therapy treatment was able to halt the progression of this disease, saving vision for the study participants. This has opened the doors for a new era of medicine – one where blindness may be able to be stopped in its tracks with the use of gene therapy.

Project Details

In December 2017, the world’s first direct-to-human gene therapy was approved for an inherited retinal disease called Leber Congenital Amaurosis. This disease normally onsets in early childhood and causes severe vision loss and blindness. Excitingly, the new gene therapy treatment was able to halt the progression of this disease, saving vision for the study participants. This has opened the doors for a new era of medicine – one where blindness may be able to be stopped in its tracks with the use of gene therapy. Our research group has international expertise in vision restoration (gene therapy, bionic eyes) and visual function assessment. We are developing new gene therapy treatments for patients with inherited retinal degenerations. We are also aiming to develop novel vision tests to determine which patients will do best with gene therapy treatments, and to then monitor their performance when they are in a clinical trial. We have a number of projects available, from laboratory work (developing and testing new gene therapies – primary supervisor Dr Tom Edwards) to clinical studies (vision testing – primary supervisor Dr Lauren Ayton). We welcome applications from scientists and clinicians of all backgrounds.