中枢神经系统
生物
细胞生物学
转导(生物物理学)
内皮干细胞
神经科学
神经系统
转基因
内皮
体内
体外
基因
内分泌学
遗传学
生物化学
作者
Trevor Krolak,Ken Y. Chan,Luke Kaplan,Qin Huang,Jason Wu,Qingxia Zheng,Velina Kozareva,Thomas Beddow,Isabelle G. Tobey,Simon Pacouret,Albert T. Chen,Yujia A. Chan,Daniel Ryvkin,Chenghua Gu,Benjamin E. Deverman
标识
DOI:10.1038/s44161-022-00046-4
摘要
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. Finally, we demonstrate AAV-BI30's capacity to achieve efficient and endothelial-specific Cre-mediated gene manipulation in the central nervous system. This combination of attributes makes AAV-BI30 uniquely well-suited to address outstanding research questions in neurovascular biology and aid the development of therapeutics to remediate endothelial dysfunction in disease.
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