Australia Establishes Pioneering Gene Therapy Lab for Inherited Retinal Diseases at CERA
September 17, 2025
A new gene therapy lab is being established at the Centre for Eye Research Australia (CERA), capable of developing viral vectors for treating inherited retinal diseases (IRDs), especially those involving large genes like MYO7A, which causes Usher syndrome.
Leading eye researcher Dr. Jiang-Hui Wang is relocating his advanced gene therapy lab from the United States back to Australia, specifically to CERA, to focus on innovative treatments for IRDs.
Dr. Wang is developing a novel, less invasive gene delivery method that involves splitting the MYO7A gene and reassembling it inside the retina with a specially engineered adeno-associated virus (AAV) vector, aiming to improve safety and effectiveness.
This approach has shown promising results in reversing retinal disease effects in mouse models, building on techniques learned under the mentorship of gene therapy expert Professor Guangping Gao in the US.
The new research platform is supported by CERA’s donors and funded by the DHB Foundation, enabling accelerated development of gene therapies for IRDs.
If successful, this method could lead to minimally invasive treatments for other inherited retinal diseases involving large genes and set a new standard in gene therapy.
Usher syndrome, primarily caused by mutations in the MYO7A gene affecting retinal photoreceptor cells, has traditionally been difficult to treat due to the gene's large size.
CERA plans to enhance its research programs in glaucoma and inherited retinal diseases with new appointments in 2026 to further advance eye health research in Australia.
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The National Tribune • Sep 17, 2025
Gene Therapies’ New Home