ArticleStroke2017
Soluble FLT1 Gene Therapy Alleviates Brain Arteriovenous Malformation Severity.
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Article in Stroke, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.
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19citing papers in PubMed
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Who cites it
19 citing papers in PubMed, 28 citations in OpenAlex.
- VEGFR1 as a Target for Cardiovascular Gene Therapy.Journal of cardiovascular translational research · 2025Review
- Unraveling Etiological Indications and Therapeutic Implications of Familial Cerebral Cavernous Malformations in the Dawn of Gene Therapy for Monogenic Conditions.Current gene therapy · 2025Article
- Evaluation of AAV Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy.Translational stroke research · 2025Article
- Review
- Updates on the Pathogenesis and Therapeutic Approaches for Hereditary Hemorrhagic Telangiectasia.Current vascular pharmacology · 2025Review
- Evaluation of Aav Capsids and Delivery Approaches for Hereditary Hemorrhagic Telangiectasia Gene Therapy.Research square · 2024Article
- Potential Targets for the Treatment of Brain Arteriovenous Malformations.Translational stroke research · 2023Article
- Circulating Plasma miRNA Homologs in Mice and Humans Reflect Familial Cerebral Cavernous Malformation Disease.Translational stroke research · 2023Article
- A single-cell atlas of the normal and malformed human brain vasculature.Science (New York, N.Y.) · 2022Article
- Hepatic factor may not originate from hepatocytes.Frontiers in cardiovascular medicine · 2022Article
- Recent progress understanding pathophysiology and genesis of brain AVM-a narrative review.Neurosurgical review · 2021Review
- Review of treatment and therapeutic targets in brain arteriovenous malformation.Journal of cerebral blood flow and metabolism : official journal of the International Society of Cerebral Blood Flow and Metabolism · 2021Review
- sVEGFR1 Is Enriched in Hepatic Vein Blood-Evidence for a Provisional Hepatic Factor Candidate?Frontiers in pediatrics · 2021Article
- Recent Advances in Basic Research for Brain Arteriovenous Malformation.International journal of molecular sciences · 2019Review
- Risk factors for hemorrhage of brain arteriovenous malformation.CNS neuroscience & therapeutics · 2019Review
- Induction of Brain Arteriovenous Malformation Through CRISPR/Cas9-Mediated Somatic Alk1 Gene Mutations in Adult Mice.Translational stroke research · 2019Article
- Preeclampsia and Cerebrovascular Disease.Hypertension (Dallas, Tex. : 1979) · 2019Review
- Article
- The roles of endoglin gene in cerebrovascular diseases.Neuroimmunology and neuroinflammation · 2017Article
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Authors and funding
17 authors at 2 institutions in 1 country.
Wan ZhuFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Fanxia ShenFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Lei MaoFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Lei ZhanFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Shuai KangFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Zhengda SunFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Jeffrey NelsonFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Rui ZhangFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Dingquan ZouFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Cameron M McDougallFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Michael T LawtonFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Thiennu H VuFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Zhijian WuFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Abraham ScariaFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Peter ColosiFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
John ForsayethFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.).
Hua SuFrom the Center for Cerebrovascular Research, Department of Anesthesia and Perioperative Care (W.Z., F.S., L.Z., S.K., J.N., R.Z., D.Z., H.S.), Department of Neurological Surgery (L.M., C.M.M., M.T.L., J.F.), Department of Radiology (Z.S.), and Department of Medicine (T.H.V.), University of California, San Francisco; Ocular Gene Therapy Core, National Eye Institute, National Institutes of Health, Bethesda, MD (Z.W.); Sanofi-Genzyme R&D Center, Framingham, MA (A.S.); and BioMarin Pharmaceutical Inc, Novato, CA (P.C.). hua.su@ucsf.edu.
National Eye Institute · USNational Institutes of Health · US
Funding
Uncover the Pathogenesis of Brain Arteriovenous MalformationR01NS027713 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 1990 to 2023
$8.6MRetinal Gene TherapyZIAEY000443 · NEI · NATIONAL EYE INSTITUTE · PI LI, TIANSEN · 2009 to 2020
$8.4MCell type-specific influences on HHT pathogenesisR01HL122774 · NHLBI · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 2015 to 2018
$1.6MSoluble VEGF receptor therapy for brain arteriovenous malformationR21NS083788 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 2014 to 2015
$790kTargeted Gene Expression in Ischemic Brain by Intravenous DeliveryR21NS070153 · NINDS · UNIVERSITY OF CALIFORNIA, SAN FRANCISCO · PI SU, HUA · 2010 to 2011
$382kNHLBI NIH HHS R01 HL122774NINDS NIH HHS R01 NS027713NINDS NIH HHS R21 NS070153NINDS NIH HHS R21 NS083788
Abstract
background and purposeBrain arteriovenous malformation (bAVM) is an important risk factor for intracranial hemorrhage. Current therapies are associated with high morbidities. Excessive vascular endothelial growth factor has been implicated in bAVM pathophysiology. Because soluble FLT1 binds to vascular endothelial growth factor with high affinity, we tested intravenous delivery of an adeno-associated viral vector serotype-9 expressing soluble FLT1 (AAV9-sFLT1) to alleviate the bAVM phenotype.
methodsTwo mouse models were used. In model 1, bAVM was induced in R26CreER;
resultsAAV2-sFLT02 inhibited bAVM formation, and AAV9-sFLT1 reduced abnormal vessels in model 1 (GFP versus sFLT1: 3.66±1.58/200 vessels versus 1.98±1.29,
conclusionsBy applying a regulated mechanism to restrict sFLT1 expression to bAVM, AAV9-sFLT1 can potentially be developed into a safer therapy to reduce the bAVM severity.
Indexed as
Angiogenesis InhibitorsGenetic VectorsVascular Endothelial Growth Factor Receptor-1AnimalsArteriovenous FistulaDependovirusDisease Models, AnimalGenetic TherapyIntracranial Arteriovenous MalformationsMiceAngiogenesis InhibitorsVascular Endothelial Growth Factor Receptor-1angiogenesisanimalsbraingene therapy
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