Endoglin and alk1 as therapeutic targets for hereditary hemorrhagic telangiectasia

Introduction: Hereditary Haemorrhagic Telangiectasia (HHT) is as an autosomal dominant trait characterized by frequent nose bleeds, mucocutaneous telangiectases, arteriovenous malformations (AVMs) of the lung, liver and brain, and gastrointestinal bleedings due to telangiectases. HHT is originated b...

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Detalles Bibliográficos
Autores: Ruiz-Llorente, Lidia, Gallardo-Vara, Eunate, Rossi, Elisa, Smadja, David M., Botella, Luisa María, Bernabéu, Carmelo
Tipo de recurso: artículo
Estado:Versión publicada
Fecha de publicación:2017
País:España
Institución:Consejo Superior de Investigaciones Científicas (CSIC)
Repositorio:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/155101
Acceso en línea:http://hdl.handle.net/10261/155101
Access Level:acceso abierto
Palabra clave:HHT
Endoglin
ALK1
Haploinsufficiency
Bleeding
Angiogenesis
AVM
Bevacizumab
Thalidomide
Descripción
Sumario:Introduction: Hereditary Haemorrhagic Telangiectasia (HHT) is as an autosomal dominant trait characterized by frequent nose bleeds, mucocutaneous telangiectases, arteriovenous malformations (AVMs) of the lung, liver and brain, and gastrointestinal bleedings due to telangiectases. HHT is originated by mutations in genes whose encoded proteins are involved in the transforming growth factor β (TGF-β) family signalling of vascular endothelial cells. In spite of the great advances in the diagnosis as well as in the molecular, cellular and animal models of HHT, the current treatments remain just at the palliative level.