Towards novel treatments for genetic causes of blindness

This project aims to save the sight of children with rare genetic defects that slowly cause blindness. This would ultimately involve modifying the mutation within the patient’s eye using gene editing tools. The safety and effectiveness of this approach must be tested before moving to clinical trial. It is possible to build a model of a human eye from patient stem cells. Using this approach, researchers will carry out a unique human-based approach to determining whether this treatment is likely to work. The team includes clinical ophthalmologists with experience in taking novel treatments into eye and the charity Genetic Cures Australia. If successful, this innovative technology will have widespread application in many genetic and congenital diseases that affect millions of people.

For more information on this project contact Linda Cox on linda.cox@mcri.edu.au or +61 418 583 986.

Investigators from the University of Melbourne

  • Professor Alice P├ębay (University of Melbourne)
  • Lisa Kearns (University of Melbourne and Centre for Eye Research Australia)

Investigators from other institutes