Spatial control of robust transgene expression in mouse artery endothelium under ultrasound guidance

Dear Editor,Dysfunctional vascular endothelial cells(ECs)contribute to the pathophysiology of several cardiovascular diseases,such as atherosclerosis and its life-threatening complications.1 Gene therapy can be a valuable approach to modulate endothelial cell function for the prevention of atherosclerosis.However,there is still a lack of method for transgene expression in vascular endothelium.2 Herein,an effective and specific strategy was established for noninvasive spatial control of transgene expression into the target region of the mouse artery without systemic spillover using an ultrasound and microbubble(MB)guided adenoassociated viral vector(UMGAAV)(Supplementary Fig.S1).To the best of our knowledge,this is the first report on noninvasive spatial control of transgene expression in arterial endothelium in vivo.