Engineered Virus Crosses Blood-Brain Barrier, Delivers Therapeutic Genes in Mice

Researchers from the Broad Institute of MIT and Harvard have taken a significant stride toward more effective gene therapies for brain diseases by engineering a gene-delivery vehicle that efficiently crosses the blood-brain barrier in mice. The vehicle, an adeno-associated virus (AAV), uses a human protein to navigate this formidable obstacle, offering hope for future therapies in patients.

The blood-brain barrier, a highly selective membrane separating the blood from the brain, has long...