New gene therapy platform uses the brain's own transport system to target glial cells

For decades, gene therapy for neurological disease has been constrained by two problems: getting therapeutic genes into the brain and ensuring they reach the cells that matter. The blood-brain barrier excludes most treatments delivered through the bloodstream, while viral vectors that do enter the central nervous system often lack the precision needed to selectively target disease-relevant cell populations.Researchers at the University of Rochester have now published a strategy that tackles both challenges simultaneously. The team developed engineered adeno-associated virus (AAV) vectors designed to preferentially infect human glial cells and paired them with a delivery approach that exploits the brain's own glymphatic transport system.