세미나 제목: Mechanism and therapy for arteriovenous malformation
연사성명: 오석
연사소속: University of Florida College of Medicine
강연날짜: 2013년 11월 20일 수요일 오후 4:00
강연장소: 녹지 생명과학관 535호
Hereditary hemorrhagic telangiectasia (HHT) is an autosomal dominant vascular disorder, characterized by spontaneous recurrent nosebleeds, mucocutaneous telangiectases, and arterio-venous malformations (AVMs) in the brain, lung, liver or GI tract. While reduced expression of either endoglin ( ENG ) or activin receptor-like kinase 1 ( ALK1 ) has shown to be associated HHT, the precise pathogenetic mechanisms underlying HHT remain elusive; and thus, while management options for HHT patients are well established, treatment options for this malady is currently lacking. The ultimate goal of my laboratory is to develop novel therapeutic reagents for treating HHT. To reach this goal we set out the following five stepwise goals: 1) development of mouse models that reproduce clinical features of vascular malformations found in HHT patients; 2) elucidation of pathogenetic mechanisms that underlie the vascular malformations using the animal model; 3) discovery of potential therapeutic target that can prevent or reverse the vascular malformations based on the mechanism; 4) preclinical validation of effects of the potential therapies using the animal models; 5) clinical trials of validated therapies through multi-HHT centers of excellence. I will present recent progress in the development of reliable animal models for HHT study. With this model, we unveiled novel pathogenetic mechanisms of disease. Potential therapeutic targets, preliminary data from preclinical studies, and future plans will be discussed.