Sydney, July 15 -- 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.
...