CAG-Cre-T2A-GFP AAV (Serotype BR1)

Recent advances in the development of adeno-associated viruses (AAVs) have resulted in engineered capsids that are able to transduce specific cell populations in the central nervous system (CNS) more efficiently than naturally occurring capsids. As a rapid and flexible in vivo gene transfer platform, these vectors are expected to be a transformative catalyst for research, either in conjunction with or in place of existing mouse genetic tools. However, capsid development has primarily focused on vectors for transducing neurons or astrocytes. In contrast, relatively few vectors exist that specifically target other cell populations within the CNS, despite the growing recognition that a large number of non-neuronal cell types are essential for nervous system function.

Brain endothelial cells are a core component of the blood-brain barrier and are essential for CNS homeostasis. Therefore, an important research topic is to identify pathways that regulate brain endothelial cell function in both physiological and disease states. To facilitate this research, genetic tools for modulating brain endothelial cell function are currently being developed. One approach is to modify the capsid of adeno-associated viruses (AAVs) to enhance tropism for brain endothelial cells. Currently, AAV-BR1 and AAV-BI30 are the most advanced. AAV-BR1 was isolated from an AAV2 library and contains the amino acid sequence "NRGTEWD" in its capsid. AAV-BR1 is able to transduce brain microvascular endothelial cells with high specificity and has been successfully exploited by many research groups since its initial discovery.