15/07/2025
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that advances scientific understanding of how therapeutic viruses interact with human cells
Published in the leading journal Cell Press, the study identifies a previously unknown gateway into human cells, a receptor called AAVR2, that gene therapy viruses use to deliver therapeutic genes.
“We found that certain AAV types can use this newly identified receptor, AAVR2, to enter cells, providing an alternative to the previously known entry route,” said Dr Bijay Dhungel, lead author of the study and researcher at the Centenary Institute’s Centre for Rare Diseases & Gene Therapy and the University of Sydney.
“We not only identified this new receptor AAVR2 but also discovered how it binds to the viruses that deliver the genes,” said co-senior author Dr Charles (Chuck) Bailey, Head of the Centre for Rare Diseases & Gene Therapy at the Centenary Institute and researcher at the University of Sydney.
The researchers say the findings have important implications for the future of gene therapy, offering new strategies to tailor treatments, lower required doses and potentially avoid immune-related complications that have limited some current approaches.
NSW Health, National Health and Medical Research Council - NHMRC Tour de Cure Australia Sydney Health
Scientists from the Centenary Institute and the University of Sydney have made a landmark discovery that could lead to safer and more effective gene therapies for a range of serious genetic disorders including Duchenne muscular dystrophy, Pompe disease and haemophilia.