With the help of directed evolution, scientists inch closer to developing viral vectors that can cross the human blood-brain barrier to deliver gene therapy.
The Scientists speaks with Beverly Davidson, a scientist at UPenn and Viviana Gradinaru, a neuroscientist at the California Institute of Technology (Caltech). Gradinaru’s work focuses on adeno-associated virus (AAV) vectors as delivery vehicle for gene therapy. AAVs is considered the leader in vivo gene therapy delivery platform due to their unmatched targeting capabilities, lasting expression in nondividing cells, and lack of pathogenicity in humans.