Abstract

Bridging the Translational Gap: A hiPSC Model of Marfan Syndrome for Drug Discovery

Sanjay Sinha, , University of Cambridge

Marfan syndrome is a connective tissue disorder, caused by mutations in FBN1, with pleiotropic manifestations including aortic aneurysms and dissection. Although the angiotensin II receptor blocker, losartan, was effective in treating the disease in mouse models, clinical trials have been much less successful. In this presentation, I will discuss the use of a hiPSC model of aortic disease in Marfan syndrome to understand the molecular basis of this disease and why the mouse studies may not have translated well to patients. I will also describe how this complex in vitro system can be used for novel drug screening for this devastating clinical disorder.