Nature Medicine doi:10.1038/nm.2025 (2009)

Viruses can be modified to deliver therapeutic genes directly to the blood vessels feeding diseased brains, according to research by Beverly Davidson and her colleagues at the University of Iowa in Iowa City. To identify address labels for the diseased blood vessels, the team used the technique of 'phage panning'. Millions of particles of a bacterial virus — phage — displaying different mouse proteins on their surface were injected into the blood of mice with a lysosomal storage disease. Phages that stuck to diseased-brain blood vessels were isolated and the protein sequences responsible for binding were identified.

When these sequences were engineered into the outer shell of an adeno-associated virus, AAV2, they enabled AAV2 to target brain blood-vessel cells in the diseased mice and deliver a gene coding for an enzyme that helps to reverse the effects of the storage defect.