Source: Xinhua
Editor: huaxia
2025-07-15 11:25:16
SYDNEY, July 15 (Xinhua) -- Scientists in Australia have identified a previously unknown gateway into human cells that could dramatically improve the safety and effectiveness of gene therapies.
Scientists have discovered a new cellular receptor, AAVR2, that enables therapeutic viruses to enter cells through an alternative pathway, which could allow gene therapies to use lower viral doses, researchers at the Centenary Institute, affiliated to the University of Sydney, said on Tuesday.
The discovery reduces the risk of severe immune reactions and expands the safety and effectiveness of these treatments for serious genetic conditions such as Duchenne muscular dystrophy, Pompe disease and haemophilia, according to a Centenary Institute statement.
The findings reveal that this newly discovered receptor acts as an alternative entry point for adeno-associated viruses (AAVs) -- the viral vectors commonly used 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 Bijay Dhungel, lead author and researcher at the Centenary Institute.
The research team demonstrated that engineering a miniature version of AAVR2 significantly boosts the uptake of gene therapies in human cells and tissues.
The discovery paves the way for safer, more precise, and cost-effective gene therapies, while deepening understanding of how therapeutic viruses interact with human cells, crucial for advancing next-generation treatments, said the study published in the journal Cell. ■