Many eye diseases that lead to blindness are caused by the death of light-sensing cells in the retina called photoreceptors. These retinal diseases affect over 196 million people worldwide and, until recently, there was no cure in sight.

Advances in gene therapy have now led to an approved treatment for inherited retinal disease (IRD) that works by replacing the individual genes that cause the disease with healthy copies. But this treatment is not effective for most people with retinitis pigmentosa (RP), one of the most common types of IRD.

Associate Professor Raymond Wong is investigating an alternative gene therapy approach. Instead of replacing the faulty gene that causes the loss of photoreceptors in RP, the treatment would reprogram retinal cells to become new photoreceptors. If successful, this cell reprogramming approach is expected to benefit all RP patients, regardless of the disease-causing genes or how much damage has occurred to the photoreceptors.

Associate Professor Wong and his team have already obtained promising data that their gene therapy can improve retinal responses in pre-clinical rodent models.

Through your support, the team has been able to purchase an essential piece of equipment – an OptoDrum – for the next stage of their pre-clinical research.

The results of this study will be used to predict how humans may respond to the treatment and is a crucial first step towards future clinical trials. Beyond this project, the OptoDrum will also allow CERA researchers to test the safety and effectiveness of upcoming stem cell and gene therapy technologies.

“By funding this new piece of equipment, CERA donors have made a critical difference to the trajectory of our current and future research, bringing us closer to a treatment that could potentially restore the sight of vision impaired patients,” says Associate Professor Wong.

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