A child undergoing experimental gene therapy with a new virus died shortly after the procedure, raising concerns about the safety of viral vectors used in brain gene therapies and potentially hindering progress in the field.
Researchers at Caltech have identified a previously unknown mechanism by which certain viral vectors can cross through the blood-brain barrier (BBB), which protects the brain from hazards in the bloodstream. The team discovered an enzyme called carbonic anhydrase IV (CA-IV) that enables a few different viral vectors to cross the BBB. This mechanistic insight may provide a new approach to designing viral vectors for research and therapeutic applications, and understanding this and other new mechanisms could also give insight into how the brain's defenses may be exploited by emergent pathogens, enabling researchers to prepare methods to block them.