Recombinant adeno-associated viruses (AAVs) are ideal gene delivery vehicles that have the potential to treat a variety of diseases. Because they do not replicate without a helper virus, AAVs alone elicit low immune responses in mammalian cells. In addition, AAV vectors have high gene transfer efficiency, stable long-term expression, and they only infect specific cell types. These features make recombinant AAV the vector of choice for viral-based gene targeting strategies in both basic and clinical research settings.
As AAV vectors became a popular viral gene delivery system, scientists developed tools and strategies to refine their production processes. While the field has come a long way, scientists still struggle to scale their experiments and consistently produce the large quantities of viral vectors required for preclinical and clinical gene therapy studies.1
The inability to properly scale viral production is in part due to the lack of a complete viral production system that ...
