Journal article

Vascular gene transfer driven by endoglin and ICAM-2 endothelial-specific promoters

B Velasco, JR Ramirez, M Relloso, C Li, S Kumar, JP Lopez-Bote, F Perez-Barriocanal, JM Lopez-Novoa, PJ Cowan, AJF d'Apice, C Bernabeu

GENE THERAPY | NATURE PUBLISHING GROUP | Published : 2001

Abstract

The involvement of the vascular endothelium in a large number of diseases supports the importance of vascular-specific gene delivery for their treatment. The hereditary hemorrhagic telangiectasia type 1 is an example of a vascular inherited disease (OMIM 187300). This is an autosomal dominant vascular disorder originated by mutations in the endoglin gene and associated with frequent epistaxis, telangiectases, gastrointestinal bleedings, and arteriovenous malformations in brain, lung and liver. Here, we address for the first time the possibility of using in vivo gene transfer to target endoglin expression to the vasculature. The promoter of the endothelial gene, ICAM-2, was used to generate t..

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