Accession Number:



Understanding and Targeting Pulmonary Arteriovenous Malformations Using Repurposed Drugs

Descriptive Note:

[Technical Report, Annual Report]

Corporate Author:

Stanford University

Report Date:


Pagination or Media Count:



Hereditary hemorrhagic telangiectasia HHT is a genetic disease characterized by multiple arteriovenous malformationsAVMs which are direct connections between arteries and veins, bypassing the capillary bed. Severe epistaxis nosebleeds is the most common symptom, yet visceral AVMs in the brain 1-10, lung 15-45, liver and gastrointestinal tract cause significant morbidity and mortality due to embolic stroke, cerebral abscess, migraines, hemorrhagic stroke, seizures and life-threatening bleeding complications. In order to reduce the morbidity and mortality associated with HHT, we need a better understanding of HHT development and novel treatment approaches. Our aims are Aim 1 To understand the cellular and molecular mechanisms of PAVM development in mice and to identify the cell behaviors and populations that give rise to PAVMs. Aim 2 To identify and target pathological downstream signaling in endothelial cells derived from iPSCs iPSC-ECsfrom HHT patients with visceral AVMs. Aim 3 To target pathological downstream signaling with repurposed drugs to prevent and reverse PAVMs in the mouse model. The short-term impact will be a better understanding of how AVMs form in the lung and potentially in other organs. The long-term impact will be the identification of potential novel treatments for AVMs.

Subject Categories:

  • Medicine and Medical Research

Distribution Statement:

[A, Approved For Public Release]