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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Bibliographic Details
Authors: Ruiz-Llorente, Lidia, Gallardo-Vara, Eunate, Rossi, Elisa, Smadja, David M., Botella, Luisa María, Bernabéu, Carmelo
Format: article
Status:Published version
Publication Date:2017
Country:España
Institution:Consejo Superior de Investigaciones Científicas (CSIC)
Repository:DIGITAL.CSIC. Repositorio Institucional del CSIC
OAI Identifier:oai:digital.csic.es:10261/155101
Online Access:http://hdl.handle.net/10261/155101
Access Level:Open access
Keyword:HHT
Endoglin
ALK1
Haploinsufficiency
Bleeding
Angiogenesis
AVM
Bevacizumab
Thalidomide
Description
Summary: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.