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, both leading international clinical trials through Cerulea Clinical Trials and developing our own novel treatments in our laboratories.
Our scientists are devoted to investigating gene therapies that could halt the progression of specific inherited retinal diseases (IRDs), or even partially reverse the damage.
Gene therapy can be done in a number of ways:
-
- Gene augmentation 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.
- Gene editing (both DNA and RNA) is possible using special enzymes to cut and paste the genetic code in our eye cells.
- Gene therapy can be used in a more general form to provide protective factors to the eye. That is, not directly correcting a defect in the genetic code but supporting general eye health.
The Inherited Retinal Disease Research and Retinal Gene Therapy Research teams are conducting several gene therapy trials for IRDs and age-related macular degeneration (AMD). They currently have several gene therapy treatments for retinal disease in pre-clinical development.
In our Glaucoma Research team, 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 IRDs like retinitis pigmentosa, macular dystrophy or a range of rarer genetic conditions. More than 300 genes are known to be associated with IRDs.
IRDs are an ideal target for gene therapy only one faulty gene needs to be addressed. More complex eye conditions, like AMD and glaucoma, may also be treated with gene therapy, but this will likely be more difficult.
Many eye conditions, including IRDs, 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.
Key research questions
- Can we halt progression of eye diseases with specific novel therapies, e.g. gene therapy?
- Can we regenerate the damaged retina or optic nerve to achieve useful restoration of vision?
- Are these therapies safe and effective in people of all ages?
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.