Research to develop a new gene therapy to restore the sight of people with blinding retinal diseases has received a major boost from the Medical Research Future Fund (MRFF).
CERA's Cellular Reprogramming Research, led by Associate Professor Raymond Wong, has received almost $600 000 in funding under the MRFF's Stem Cells Therapies Mission to build on promising pre-clinical research.
The funding will enable Associate Professor Wong to continue research aiming to 'reprogram' cells in the retina to become light-sensing photoreceptors.
After promising early results, the next phase of the research will further refine the use of technology on cells in the lab and test the use of a gene therapy to deliver the treatment to retinal cells in animal models.
About retinal disease
Millions of tiny light-sensing cells known as photoreceptors line our retina at the back of the eye, sending messages to our brain which enable us to see. When they are damaged or die, vision loss occurs.
An estimated 190 million people worldwide have retinal diseases - ranging from rare genetic conditions like retinitis pigmentosa and Stargardt's disease to more common diseases such as age-related macular degeneration.
Most of the research effort to treat inherited retinal diseases is focused on replacing single defective genes which cause vision loss, while those targeting more common diseases focus on introducing protective genes to stop future damage. Associate Professor Wong is taking a different approach.
"Our hope is that we can treat this using gene therapy that regenerates and replaces the cells that have died,'' he says.
"We want to stimulate stem cells in the retina using gene therapy to grow into new photoreceptor cells that work."
Reprogramming cells
"Cellular reprogramming is a technology which allows you to control the fate of cells using genes," Associate Professor Wong says. "Controlling genes can determine how cells behave."
The process aims to use genes to turn the stem cells in the retina - the Müller glia cells - into new photoreceptor cells to replace those damaged by retinal disease.
In chicken and fish, Müller glia cells can mobilise and repair the retina when retinal injury occurs.
"This ability for repair has been lost or suppressed in mammals including humans," Associate Professor Wong said. "We're working to unlock this regenerative ability and stimulate Müller glia cells into making new photoreceptor cells, which is quite exciting.
"This is a unique regenerative approach. For the past five years we have done a lot of work to get to this stage to try to regenerate photoreceptors.
"The MRFF funding is a critical next step in taking what we have learned in the lab and potentially translating it into a therapy that can be tested at clinical trial.''
A new start-up company aiming to develop a gene therapy to 'switch on sight' by restoring the retina's light-sensing cells has won vital funding to accelerate its research.
Our researchers use cell reprogramming and stem cell technologies to better understand and develop treatments for eye diseases, with the goal of regenerating cells in the retina.
