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JCI Targeting KIT prevents brain arteriovenous malformations driven by ALK1-deficient angiogenic endothelial cells
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JCI Targeting KIT prevents brain arteriovenous malformations driven by ALK1-deficient angiogenic endothelial cells
Hereditary hemorrhagic telangiectasia type 2 (HHT2), caused by mutations in ACVRL1 (also known as ALK1), is characterized by brain arteriovenous malformations (bAVMs), abnormal artery–vein connections for which treatment options remain limited. Despite evidence of endothelial cell (EC) heterogeneity, its role in bAVM pathogenesis remains poorly defined. Using EC-specific inducible Alk1-knockout mice (Alk1iECKO) and regionally resolved single-cel…
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