Large Gene Delivery via Adeno-Associated Viral Vector (rAAV Large Gene Delivery)

Large gene delivery via adeno-associated viral vector is a gene therapy approach used to deliver therapeutic genes larger than the standard AAV packaging capacity. It utilizes strategies such as dual or multi-vector systems to overcome size limitations and treat diseases caused by mutations in large genes, such as dystrophin in Duchenne muscular dystrophy.

Other names

AAV Large Gene DeliveryRecombinant AAV Large Gene DeliveryDual AAV Vector Gene TherapyMulti-AAV Vector Gene Therapy

AAV vectors deliver DNA sequences encoding therapeutic proteins into host cells. The delivered DNA typically remains episomal. After infection, the transgene is expressed by the host cell machinery using dual/multi-vector systems.