FAST in collaboration with Apetura is exploring an investigational Adeno-Associated Virus gene therapy (AAV-GT) approach to treating Angelman syndrome using a specially engineered viral vector to potentially deliver a gene therapy through the blood.
A therapeutic approach that can potentially be given intravenously (by blood) and uses a specially engineered virus (non-infectious adeno-associated virus) that can cross the blood brain barrier to deliver a functional UBE3A gene to the brain.
