A team of scientists in Australia has identified a previously unknown gateway into human cells that could drastically improve the safety and effectiveness of gene therapy.
Scientists have discovered a new cellular receptor, AAVR2, that could allow therapeutic viruses to enter cells through an alternative pathway, enabling gene therapy using lower doses of the virus, researchers at the University of Sydney's Centenary Institute.
The discovery reduces the risk of severe immune reactions and extends the safety and effectiveness of the treatment for serious genetic conditions, such as Duchenne muscular dystrophy, Pompe disease and hemophilia, the Centenary Institute said in a statement.
These findings reveal that the newly discovered receptor acts as an alternative entry point for adeno-associated viruses (AAV), a viral vector commonly used to deliver therapeutic genes.
"We found that certain AAV strains can use this newly identified receptor, AAVR2, to enter cells, providing an alternative to previously known entry routes," said Bijay Dhungel, lead author and a researcher at the Centenary Institute.
The research team showed that engineering a miniaturized version of AAVR2 significantly increased the uptake of the gene therapy in human cells and tissues.
The discovery paves the way for safer, more precise, and more cost-effective gene therapies, while deepening our understanding of how therapeutic viruses interact with human cells, which is crucial for advancing next-generation treatments, the study, published in the journal Cell, said.
