A High-Efficiency AAV for Endothelial Cell Transduction Throughout the Central Nervous System.

ABSTRACT: Endothelial cells have a crucial role in nervous system function, and mounting evidence points to endothelial impairment as a major contributor to a wide range of neurological diseases. However, tools to genetically interrogate these cells in vivo remain limited. Here, we describe AAV-BI30, a capsid that specifically and efficiently transduces endothelial cells throughout the central nervous system. At relatively low systemic doses, this vector transduces the majority of arterial, capillary, and venous endothelial cells in the brain, retina, and spinal cord vasculature of adult C57BL/6 mice. Furthermore, we show that AAV-BI30 robustly transduces endothelial cells in multiple mouse strains and rats in vivo and human brain microvascular endothelial cells in vitro. Finall

SUBMITTER: Krolak T 

PROVIDER: S-EPMC9103166 | biostudies-literature | 2022 Apr

REPOSITORIES: biostudies-literature