Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model

Abstract Cerebral cavernous malformations (CCMs) are vascular abnormalities that primarily occur in adulthood and cause cerebral hemorrhage, stroke, and seizures. CCMs are thought to be initiated by endothelial cell (EC) loss of any one of the three Ccm genes: CCM1 (KRIT1), CCM2 (OSM), or CCM3 (PDCD...

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Main Authors: Huanjiao Jenny Zhou, Lingfeng Qin, Quan Jiang, Katie N. Murray, Haifeng Zhang, Busu Li, Qun Lin, Morven Graham, Xinran Liu, Jaime Grutzendler, Wang Min
Format: Article
Language:English
Published: Nature Portfolio 2021-01-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-020-20774-0
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author Huanjiao Jenny Zhou
Lingfeng Qin
Quan Jiang
Katie N. Murray
Haifeng Zhang
Busu Li
Qun Lin
Morven Graham
Xinran Liu
Jaime Grutzendler
Wang Min
author_facet Huanjiao Jenny Zhou
Lingfeng Qin
Quan Jiang
Katie N. Murray
Haifeng Zhang
Busu Li
Qun Lin
Morven Graham
Xinran Liu
Jaime Grutzendler
Wang Min
author_sort Huanjiao Jenny Zhou
collection DOAJ
description Abstract Cerebral cavernous malformations (CCMs) are vascular abnormalities that primarily occur in adulthood and cause cerebral hemorrhage, stroke, and seizures. CCMs are thought to be initiated by endothelial cell (EC) loss of any one of the three Ccm genes: CCM1 (KRIT1), CCM2 (OSM), or CCM3 (PDCD10). Here we report that mice with a brain EC-specific deletion of Pdcd10 (Pdcd10 BECKO) survive up to 6-12 months and develop bona fide CCM lesions in all regions of brain, allowing us to visualize the vascular dynamics of CCM lesions using transcranial two-photon microscopy. This approach reveals that CCMs initiate from protrusion at the level of capillary and post-capillary venules with gradual dissociation of pericytes. Microvascular beds in lesions are hyper-permeable, and these disorganized structures present endomucin-positive ECs and α-smooth muscle actin-positive pericytes. Caveolae in the endothelium of Pdcd10 BECKO lesions are drastically increased, enhancing Tie2 signaling in Ccm3-deficient ECs. Moreover, genetic deletion of caveolin-1 or pharmacological blockade of Tie2 signaling effectively normalizes microvascular structure and barrier function with attenuated EC-pericyte disassociation and CCM lesion formation in Pdcd10 BECKO mice. Our study establishes a chronic CCM model and uncovers a mechanism by which CCM3 mutation-induced caveolae-Tie2 signaling contributes to CCM pathogenesis.
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spelling doaj-art-fb06cd85440d49b9be758e802813c1932025-08-20T03:05:15ZengNature PortfolioNature Communications2041-17232021-01-0112112210.1038/s41467-020-20774-0Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse modelHuanjiao Jenny Zhou0Lingfeng Qin1Quan Jiang2Katie N. Murray3Haifeng Zhang4Busu Li5Qun Lin6Morven Graham7Xinran Liu8Jaime Grutzendler9Wang Min10Interdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineDepartment of Neurobiology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineDepartment of Cell Biology, Yale University School of MedicineDepartment of Cell Biology, Yale University School of MedicineDepartment of Neurobiology, Yale University School of MedicineInterdepartmental Program in Vascular Biology and Therapeutics, Department of Pathology, Yale University School of MedicineAbstract Cerebral cavernous malformations (CCMs) are vascular abnormalities that primarily occur in adulthood and cause cerebral hemorrhage, stroke, and seizures. CCMs are thought to be initiated by endothelial cell (EC) loss of any one of the three Ccm genes: CCM1 (KRIT1), CCM2 (OSM), or CCM3 (PDCD10). Here we report that mice with a brain EC-specific deletion of Pdcd10 (Pdcd10 BECKO) survive up to 6-12 months and develop bona fide CCM lesions in all regions of brain, allowing us to visualize the vascular dynamics of CCM lesions using transcranial two-photon microscopy. This approach reveals that CCMs initiate from protrusion at the level of capillary and post-capillary venules with gradual dissociation of pericytes. Microvascular beds in lesions are hyper-permeable, and these disorganized structures present endomucin-positive ECs and α-smooth muscle actin-positive pericytes. Caveolae in the endothelium of Pdcd10 BECKO lesions are drastically increased, enhancing Tie2 signaling in Ccm3-deficient ECs. Moreover, genetic deletion of caveolin-1 or pharmacological blockade of Tie2 signaling effectively normalizes microvascular structure and barrier function with attenuated EC-pericyte disassociation and CCM lesion formation in Pdcd10 BECKO mice. Our study establishes a chronic CCM model and uncovers a mechanism by which CCM3 mutation-induced caveolae-Tie2 signaling contributes to CCM pathogenesis.https://doi.org/10.1038/s41467-020-20774-0
spellingShingle Huanjiao Jenny Zhou
Lingfeng Qin
Quan Jiang
Katie N. Murray
Haifeng Zhang
Busu Li
Qun Lin
Morven Graham
Xinran Liu
Jaime Grutzendler
Wang Min
Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
Nature Communications
title Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
title_full Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
title_fullStr Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
title_full_unstemmed Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
title_short Caveolae-mediated Tie2 signaling contributes to CCM pathogenesis in a brain endothelial cell-specific Pdcd10-deficient mouse model
title_sort caveolae mediated tie2 signaling contributes to ccm pathogenesis in a brain endothelial cell specific pdcd10 deficient mouse model
url https://doi.org/10.1038/s41467-020-20774-0
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