Nature Biotechnol. doi: 10.1038/nbt1515 (2008)

A virus has been found that can shuttle genes across the blood–brain barrier. The discovery raises hopes that gene therapy could one day be used to treat neurodegenerative diseases.

Although a variety of viruses have been harnessed as vectors to carry genes into cells, none has been able to enter the brain from the bloodstream. Brian Kaspar of Nationwide Children's Hospital in Columbus, Ohio, and his co-workers have found that adeno-associated-virus 9 (AAV9) carrying a fluorescent 'reporter' gene enters neurons in the brain and spinal cord when injected intravenously into newborn mouse pups, in which the blood–brain barrier is not fully formed.

AAV9 also entered the central nervous system of adult mice, but primarily targeted support cells called astrocytes.