ArticleGene therapy2001
Vascular gene transfer driven by endoglin and ICAM-2 endothelial-specific promoters.
Article in Gene therapy, 2001. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 12 papers.
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Who cites it
12 citing papers in PubMed, 46 citations in OpenAlex.
- Neu5Gc and beyond: sialylation at the innate immune and thromboinflammatory interface of xenotransplantation.Frontiers in transplantation · 2026Review
- Article
- Hereditary haemorrhagic telangiectasia.Nature reviews. Disease primers · 2025Review
- Construction of a novel expression cassette for increasing transgene expression in vivo in endothelial cells of large blood vessels.Gene therapy · 2011Article
- Review
- New vectors and strategies for cardiovascular gene therapy.Current gene therapy · 2007Review
- Hereditary hemorrhagic telangiectasia, a vascular dysplasia affecting the TGF-beta signaling pathway.Clinical medicine & research · 2006Review
- Article
- Highlights on endoglin (CD105): from basic findings towards clinical applications in human cancer.Journal of translational medicine · 2004Article
- Endoglin (CD105): a powerful therapeutic target on tumor-associated angiogenetic blood vessels.Oncogene · 2003Review
- Expression of the TGF-beta coreceptor endoglin in epidermal keratinocytes and its dual role in multistage mouse skin carcinogenesis.Oncogene · 2003Article
- Transcriptional Targeting in Cancer Gene Therapy.Journal of biomedicine & biotechnology · 2003Article
Corrections and comments
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Authors and funding
11 authors at 6 institutions in 3 countries.
Funding
No grant is acknowledged in the PubMed record.
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 transgenic animals which demonstrated endothelial expression of endoglin. Next, the promoters of the human endothelial genes, endoglin and ICAM-2, were inserted upstream of the human endoglin cDNA, and the resulting constructs were systemically or locally delivered, demonstrating endoglin expression in the vessel walls of liver, lung and skin. These gene transfer experiments represent an initial step in the treatment of the hereditary hemorrhagic telangiectasia type 1 by gene therapy, and suggest that endoglin and ICAM-2 promoters can be used to deliver other genes to the endothelium specifically.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.