CERA

Science and Research

Gene therapy research

CERA is leading world-first research into gene therapies that could halt or even reverse vision loss in patients with conditions including inherited retinal diseases and glaucoma.

Overview

CERA is at the forefront of gene therapy research for eye disease and is striving to establish a Melbourne Centre for Excellence in Ocular Gene and Cell Therapy to continue advancing this exciting field.

Our scientists are devoted to investigating gene therapies that could halt the progression of specific inherited retinal diseases, or even partially reverse the damage. This is done by identifying a defective gene which causes vision loss, producing a correct copy of the gene in the lab and reintroducing this copy back into the retinal cells, using a specially-engineered virus.

In the glaucoma research unit, our researchers are investigating a number of strategies including gene therapy to enhance optic nerve regeneration.

A gene therapy for glaucoma previously developed by Professor Keith Martin is currently in advanced pre-clinical development prior to planned human clinical trials.

Why this research is important

Millions of working-age people worldwide are affected by inherited retinal diseases like retinitis pigmentosa, macular dystrophy, or a range of rarer genetic conditions. More than 200 genes are known to be associated with retinal diseases.

These conditions are currently considered untreatable. Gene therapy brings new hope that we may be able to stop the progression of vision loss for some patients, or even one day restore some sight.

As research progresses, we may also develop techniques that can be applied to more common eye diseases with a genetic basis such as age-related macular degeneration.

Key research questions

  • Can we find novel therapies gene therapies to find a cure for inherited retinal degeneration?
  • Can we halt progression of specific inherited retinal diseases with specific novel therapies, e.g. gene therapy?
  • Can gene therapy be used to treat patients with glaucoma whose vision is deteriorating despite conventional treatment to lower the eye pressure?
  • Can we regenerate the damaged optic nerve to achieve useful restoration of vision?

Gene therapy explained

In this video, learn about CERA’s world-first research into gene therapies that could halt or even reverse vision loss in patients with conditions such as inherited retinal diseases and glaucoma.