Brain endothelial cells are critical for homeostasis of the central nervous system. Novel adeno-associated viruses (AAV) with brain endothelial cell tropism have been developed and are beginning to be employed in mechanistic and therapeutic research. Studies using AAVs can be involved in terms of cost, time and personnel, and many groups, including our own, are not experts on the technology. Therefore, it is important to report data using AAVs with the research community as a guide for ongoing and future studies. Here, we detail our initial experience with the two most prevalent AAVs with tropism for brain endothelial cells, AAV-BR1 and AAV-BI30. One of our long-term goals is to express key proteins in brain endothelial cells and determine the impact on brain function. For method development, we administered AAV-BR1 and AAV-BI30 with a CMV-driven fluorescent reporter (CMV-P2A-mCherry) to wild-type mice intravenously (retro-orbital) and measured expression in brain and peripheral tissues by RT-PCR and immunostaining. We found that AAV-BR1 transduces neurons and endothelial cells in the brain, and the lung and liver, whereas AAV-BI30 transduces brain endothelial cells and peripheral tissue. Our data highlights the importance of using the AAV best suited to the scientific question.
Keywords: AAV; BI30; BR1; brain endothelial cells.
Novel AAVs with brain endothelial cell tropism have been developed and are starting to be utilized by researchers for mechanistic and therapeutic studies. Therefore, it is important to report experience with the research community as a guide for future studies and we focused on the two most common AAVs with tropism for endothelial cells, AAV-BR1 and AAV-BI30. We found that AAV-BR1 transduces neurons and brain endothelial cells, and the lung and liver, whereas AAV-BI30 transduces brain endothelial cells, lung and liver. Collectively, our data highlight the importance of using the AAV that best matches the scientific question.